Open Label, Single-center Pilot Study to Investigate Alglucosidase Alfa (20 mg/kg) Frequency Reduction From 2 to 4 Weeks in a Subgroup of Elderly Patients With Late-onset Pompe Disease (TRIPOD-Study)

The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mg/kg once every 2 weeks to once every 4 weeks is safe and does not lead to increased progression of disease in a selected group of patients with late-onset Pompe disease.

Start Date01 Oct 2024

Sponsor / Collaborator

Erasmus MC

CTIS2024-514255-15-01

/ Not yet recruitingPhase 4

Open label, single-center pilot study to investigate enzyme replacement therapy (ERT) frequency reduction from 2 to 4 weeks in a subgroup of elderly patients with late-onset Pompe disease

Start Date01 Oct 2024

Sponsor / Collaborator

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

NCT06121011

/ RecruitingNot Applicable

A Global Prospective Observational Registry of Patients With Pompe Disease

This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate.
The objectives of the registry are:
* To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/serious adverse events (SAEs) occurring in Pompe disease patients
* To evaluate the long-term real-world effectiveness of Pompe disease treatments
* To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and patient-reported outcomes (PROs)
* To describe the natural history of untreated Pompe disease

Start Date16 Feb 2024

Sponsor / Collaborator

Amicus Therapeutics, Inc.

100 Clinical Results associated with Alglucosidase Alfa

100 Translational Medicine associated with Alglucosidase Alfa

100 Patents (Medical) associated with Alglucosidase Alfa

308

Literatures (Medical) associated with Alglucosidase Alfa

01 Apr 2026·CURRENT OPINION IN IMMUNOLOGY

A comprehensive study on the effect of alglucosidase alpha and immunomodulation on survival, motor and cardiac outcome, creatine kinase and antibody titers in classic infantile Pompe disease: the Monza experience

Review

Author: Tedesco, Lucia M ; Gasperini, Serena ; Boffi, Maria L ; Pretese, Roberta ; van den Hout, Johanna Mp ; Serafini, Marta ; Bonanomi, Sonia ; Pozzi, Katia ; Crescitelli, Viola ; Faraguna, Martha C ; van der Ploeg, Ans T ; Kullmann, Gaia A ; Risca, Giulia ; Balduzzi, Adriana C

We report on 13 classic infantile Pompe patients, including four cross-reactive immunological material negative (31%), treated with alglucosidase alpha (rhGAA) at Fondazione IRCCS San Gerardo, Monza, between 2003 and 2024. Median age at rhGAA initiation was 3.3 months, with nine patients (69%) starting on doses > 20 mg/kg/every other week. With a median follow-up of 6.9 years, the 5- and 10-year survival was 92%. Four patients died, and three became ventilator-dependent. Hypertrophic cardiomyopathy normalized in all patients, though electrocardiogram abnormalities persisted in 36%. Walking was achieved by 10 (77%). Ten patients received immune tolerance induction (77%, five primary, two secondary, three both), and seven received long-term sirolimus. Nine developed anti-rhGAA, of whom five (38%) had high-sustained antibody titers (HSAT). All had elevated creatine kinase at diagnosis; creatine phosphokinase normalized over time in four patients on 40 mg/kg/week since start without HSAT. This study offers real-world insight into managing classic infantile Pompe disease and compares the cohort's outcome to international experiences.

01 Mar 2026·NEUROMUSCULAR DISORDERS

Clinically important improvements in 6-minute walk distance and forced vital capacity in adults with late-onset Pompe disease switching from alglucosidase alfa to cipaglucosidase alfa plus miglustat in the PROPEL study

Article

Author: Peceny, Markus ; Gloeckner, Birgit ; Jain, Vipul ; Hummel, Noemi ; Schoser, Benedikt ; Fox, Brian ; Wenninger, Stephan ; Holdbrook, Fred ; Claeys, Kristl G ; Kushlaf, Hani

We determined the proportion of adults with late-onset Pompe disease (LOPD) with clinically important changes in 6-minute walk distance (6MWD) and/or forced vital capacity (FVC) after switching from alglucosidase alfa (alg) to cipaglucosidase alfa plus miglustat (cipa+mig) in PROPEL (NCT03729362). This post hoc analysis (95 patients) used published anchor- and distribution-based minimal clinically important differences for 6MWD (in meters for anchor-based; % predicted for distribution-based) and a 3 % threshold for FVC (% predicted). For 6MWD, a higher percentage of patients improved after switching to cipa+mig versus alg plus placebo (alg+pbo) (anchor-based: 29.2 % versus 13.3 %; distribution-based: 33.8 % versus 13.3 %), fewer patients worsened (anchor-based: 12.3 % versus 26.7 %; distribution-based: 7.7 % versus 13.3 %). For FVC, 27.7 % versus 0.0 % improved for cipa+mig versus alg+pbo, 27.7 % versus 53.3 % worsened. Overall, 50.8 % versus 13.3 % of patients experienced improvements in 6MWD (% predicted) and/or FVC with cipa+mig versus alg+pbo, 30.8 % versus 56.7 % worsened. For combined responses from 6MWD (% predicted) and FVC (% predicted), the odds of a better versus a lower response category (improvement > stability > worsening) were 4.05 (95 % confidence interval 1.73-9.51) times higher for cipa+mig than alg+pbo (P = 0.0013). Adults with LOPD switching from alg to cipa+mig have greater chances of clinically relevant motor and lung function improvements than those remaining on alg.

01 Jan 2026·MOLECULAR GENETICS AND METABOLISM

Focused ultrasound delivery of enzyme replacement therapy to the brain of Gaa−/− Pompe disease mice

Article

Author: Dai, Jian ; Chrisholm, Amanda ; Zhang, Yongzhi ; Zhang, Haoyue ; Owusu-Yaw, Bernie Simone ; Nowlin, Paige ; Bali, Deeksha ; Upadhyay, Jaymin ; Todd, Nick ; Young, Sarah P

Clinically used enzyme replacements therapies (ERTs) have been successful in mitigating peripheral tissue pathology in patients with infantile onset (IOPD) and late onset (LOPD) Pompe disease (PD). However, none of the approved therapies are known to cross the blood brain barrier (BBB) and patients with PD have progressive central nervous system (CNS)-associated impairments due to lysosomal glycogen accumulation in the CNS. Here we investigate the use of focused ultrasound (FUS) to temporarily open the BBB as a treatment strategy for achieving delivery of intravenously administered recombinant human acid alpha glucosidase enzyme (rhGAA) into targeted regions of the mouse brain. We investigated GAA delivery and glycogen accumulation in 5-month-old Gaa-/- knockout mice following administration of two clinically available rhGAA ERTS (alglucosidase alfa and avalglucosidase alfa) at different dosages with and without FUS-BBB opening over four biweekly treatment sessions. BBB opening was targeted specifically to the striatum and cortex bilaterally in the FUS groups. Diphenhydramine was administered intraperitoneally 10 min before ERT to avoid anaphylactic response. Mice were sacrificed 24 h after the last treatment session. One hemisphere was used for histological analysis based on periodic acid Schiff (PAS) and H&E stained sections. The other hemisphere was used for biochemical assays to measure GAA enzyme activity and glycogen content. Contrast MRI showed consistent BBB opening over all mice treated with FUS with no significant differences in extent of BBB opening between hemispheres or treatment session. We observed significant reductions in the level of PAS staining for FUS + ERT treated mice compared to No FUS + ERT treated mice, indicative of successful ERT delivery across the BBB resulting in glycogen clearance. Biochemical analysis supported these results, showing an increase in GAA enzyme activity and reduction in glycogen content for FUS + ERT treated mice compared to No FUS + ERT groups. Future work will determine if this promising treatment paradigm can rescue disease phenotypes that are downstream of glycogen accumulation and work towards clinical translation.

News (Medical) associated with Alglucosidase Alfa

08 Sep 2025

·www.globenewswire.com

New 4-Year Data for Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) Presented at ICIEM

Long-Term Data Adds to the Growing Body of Evidence Supporting the Compelling Clinical Profile of Pombiliti® + Opfolda®PRINCETON, N.J., Sept. 08, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced the presentation of new 4-year muscle function, muscle strength and biomarker endpoints from the PROPEL open-label extension (OLE) study of cipaglucosidase alfa-atga + miglustat (cipa+mig) in adults with late-onset Pompe disease (LOPD) at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan. The new analysis, “208-week efficacy and safety of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: muscle function and biomarkers,” was conducted on the 82 patients (62 ERT-experienced and 20 ERT-naïve) who were randomized to cipa+mig in PROPEL and who enrolled in the open label extension (OLE). Pombiliti + Opfolda is indicated in the U.S. for the treatment of adult patients with LOPD weighing ≥40 kg and who are not improving on their current ERT. In the U.S., Pombiliti + Opfolda is not indicated for patients naïve to ERT treatment. The data summarized below are from the ERT-experienced group in the OLE. Pulmonary function data from the PROPEL OLE will be presented separately at an upcoming conference. The mean age was 48.8 years and the median duration of prior ERT in the ERT-experienced cohort at PROPEL baseline was 7.5 years. In the ERT-experienced group, the measures of muscle function, muscle strength and biomarkers improved and/or were sustained out to 208 weeks. Mean change from PROPEL baseline to week 208 were as follows: Percent predicted six-minute walk distance (6MWD), +2.3%;Lower extremity manual muscle test (MMT) score, +1.6 points;Gait-Stairs-Gowers-Chair (GSGC) score, +0.1 points;PROMIS® physical function score, +1.2 points;Serum creatine kinase (CK) levels, -160.0 serum CK; and,Urine hexose tetrasaccharide (Hex4), -1.9 mmol/mol creatinine. No new safety signals were identified. Up to week 208, 41 patients experienced treatment-related treatment-emergent adverse events (TEAEs), leading to four cipa+mig discontinuations, and two patients experienced serious treatment-related TEAEs, leading to two cipa+mig discontinuations. One patient experienced a TEAE (worsening of Lewy body disease) unrelated to study treatment, which led to death; the patient had discontinued from the study prior to death. “Durability of effect is important for evaluating the impact of treatments for individuals with LOPD. In this new analysis assessing the long-term outcomes from the open label extension of PROPEL, the improvements in muscle function, muscle strength, and biomarkers observed in patients receiving Pombiliti and Opfolda were maintained for four years,” said Priya Kishnani, MD, Chief, Division of Medical Genetics, Chen Family Distinguished Professor of Pediatrics, Chief, Division of Medical Genetics, Duke University Medical Center. “This new analysis adds to the growing body of evidence supporting the compelling clinical profile of Pombiliti and Opfolda in ERT-experienced adults,” said Jeff Castelli, PhD, Chief Development Officer, Amicus Therapeutics, Inc. “To observe these sustained improvements and stability in the patients in the open label extension at four years is very encouraging given the progressive nature of LOPD. We look forward to presenting long-term extension data across pulmonary measures soon and will continue to closely study Pombiliti and Opfolda as part of our commitment to make a meaningful difference in the lives of people living with late-onset Pompe disease.” Other abstracts at ICIEMIn addition to this analysis, the following abstracts related to Pombiliti + Opfolda based on Amicus studies were also presented at ICIEM: Abstract Title: Cipaglucosidase alfa and alglucosidase alfa enzymes have similar stability at neutral pH and can be stabilized with miglustat Presenter: Filip Cosmanescu, Amicus Therapeutics Inc., Princeton, NJ, U.S.A. Abstract Title: Integrating clinical evidence on cipaglucosidase alfa plus miglustat and alglucosidase alfa via a multi-level network meta-regression Presenter: William Kerr, Amicus Therapeutics Ltd, Marlow, U.K. Abstract Title: PROPEL Japan subpopulation: efficacy and safety of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in patients with late-onset Pompe disease Presenter: Hiroshi Kobayashi, The Jikei University School of Medicine, Tokyo, Japan Abstract Title: ATB200-19: an open-label, expanded access study of the safety and effectiveness of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease in Japan Presenter: Aya Narita, ISEIKAI International General Hospital, Osaka, Japan Abstract Title: PROPEL Australia subpopulation: efficacy and safety of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in patients with late-onset Pompe disease Presenter: Michel Tchan, Westmead Hospital, Westmead, NSW, Australia The following abstract related to Pombiliti + Opfolda from a non-Amicus study was also presented at ICIEM: Abstract Title: Real World Study: Assessing the impact of switching from alpha-glucosidase to cipaglucosidase alfa with miglustat on disease progression in adults with Pompe disease Lead author: Sara Lucas Del Pozo, National Hospital for Neurology and Neurosurgery, UCLH, London, U.K.Presenter: Robin Lachmann, National Hospital for Neurology and Neurosurgery, UCLH, London, U.K. About the PROPEL StudyPROPEL was a 52-week, double-blind randomized global study designed to assess the efficacy, safety, and tolerability of cipaglucosidase alfa-atga + miglustat compared to non-U.S. approved alglucosidase alfa + placebo (the comparator). The study enrolled 123 adult LOPD patients who still had the ability to walk and to breathe without mechanical ventilation. Patients enrolled in PROPEL were randomized 2:1 so that for every two patients randomized to be treated with cipaglucosidase alfa-atga + miglustat, one was randomized to be treated with the comparator. Of the patients enrolled in PROPEL, 77% were being treated with alglucosidase alfa (n=95) for at least 2 years at study entry and 23% had never been treated with any ERT (n=28). 117 of the 123 patients (>95%) completed the PROPEL study. Efficacy endpoints of the study included primary endpoint of change from baseline to week 52 in 6-minute walk distance (6MWD) for comparison of superiority and key secondary endpoint of change from baseline to week 52 in forced vital capacity (FVC). PROPEL did not achieve statistical significance for the primary endpoint of superiority in change from baseline to week 52 in 6MWD in the overall population. After 52 weeks, ERT-experienced patients treated with cipaglucosidase alfa-atga + miglustat (n=61) walked an estimated 17 meters (95% CI, 0.2, 33) farther than the comparator group (n=29). After 52 weeks, ERT-experienced patients treated with cipaglucosidase alfa-atga + miglustat (n=55) showed an estimated treatment difference of 3.5% (95% CI, 1.0, 6.0) in FVC compared with the comparator group (n=29). About the PROPEL Open-label Extension StudyThe Phase 3 open-label extension (OLE) study (ATB200-07) is a multicenter, international study designed to evaluate the long-term safety and efficacy of cipaglucosidase alfa-atga + miglustat in adult subjects with LOPD who completed the Phase 3 PROPEL Study (ATB200-03). Of the 118 patients treated in the OLE, 81 continued cipa+mig treatment from PROPEL (cipa+mig group; 61 ERT-experienced prior to PROPEL; 20 ERT-naïve) and 37 switched from alg+pbo to cipa+mig (switch group; 29 ERT-experienced; 8 ERT-naive). Efficacy endpoints of the study included primary endpoint of change from PROPEL baseline to approximately 4 years in 6-minute walk distance (6MWD), forced vital capacity (FVC), creatine kinase (CK) and hexose tetrasaccharide (Hex4) levels, patient-reported outcomes and safety. About Pombiliti + OpfoldaPombiliti® + Opfolda®, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood. U.S. INDICATIONS AND USAGEPOMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). SAFETY INFORMATION HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full PRESCRIBING INFORMATION for OPFOLDA (miglustat) LINK. About Late-Onset Pompe DiseaseLate-onset Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Late-onset Pompe disease can be severe and debilitating with progressive muscle weakness throughout the body that worsens over time, particularly skeletal muscles and muscles that control breathing. About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on X and LinkedIn. NP-AT-ALL-00010825 CONTACTS: Investors: Amicus Therapeutics Andrew Faughnan Vice President, Investor Relations afaughnan@amicusrx.com(609) 662-3809 Media: Amicus Therapeutics Diana Moore Vice President, Corporate Communications dmoore@amicusrx.com(609) 662-5079 FOLD-G

Clinical ResultPhase 3Drug Approval

03 Sep 2025

·pharma.economictimes.indiatimes.com

GST waiver for 36 drugs; formulations under 5% slab

New Delhi: Executing the much-awaited overhaul of the Goods and Services Tax (GST) regime, the GST Council has brought a major relief for patients by reducing the indirect tax on medicines to 5 per cent, along with exempting as many as 36 life-saving medicines and other critical drugs. The GST council—a statutory body highest decision making body on indirect taxes led by the Union Finance Ministry at its 56th meeting in the national capital has recommended to reduce the GST on all drugs and medicines (finished formulations) from 12 per cent to 5 per cent under the new two-tier tax structure of 5 and 18 per cent slabs to be effective from September 22. Additionally the body has also recommended to exempt GST on 33 lifesaving drugs and on 3 other medicines used for treatment of cancer, rare diseases and other severe chronic diseases. Speaking at a press briefing after the council 10.5-hour long marathon meet, Finance Minister Nirmala Sitharaman said, “all recommendations have been cleared with zero reservations and have corrected various inverted duty structure problems, classification related issues and that this rationalization exercise will bring predictability and stability of GST.” “These reforms have been carried out with a focus on the common man, and after a rigorous evaluation process in most sectors including healthcare, the rates have come down drastically, she added. Previously, medicines in India were taxed at 5 per cent for life-saving and essential drugs, and at 12 per cent for others but following the announcement all medicines will attract a standard duty of 5 per cent with extensions for several medicines specified at the end of this article. Welcoming the move, Sudarshan Jain, Secretary General, IPA said, “the decision to exempt life-saving and cancer medicines from GST, is a step that will bring direct relief to patients and their families. and, the reduction in GST on a wide range of medicines from 12 per cent to 5 per cent will further help to ease the overall treatment burden and make essential therapies more affordable.” Meanwhile, for companies, the clarity on a 5 per cent slab and exemptions removes long-standing ambiguity, enabling transparent pricing and better market planning. The move will expand access in semi-urban and rural markets, ease litigation, and free up resources for innovation, said Manoj Mishra, Partner and Tax Controversy Management Leader, Grant Thornton Bharat LLP. Notably, on the issue of inverted duty involving APIs and key starting materials—which are currently taxed at 18 per cent against 12 or 5 per cent for finished formulations—further clarity is awaited from the industry. ETPharma earlier reported that, in a letter to the Finance Minister, the national chemist body, the All India Organisation of Chemists and Druggists (AIOCD) had urged, “to bring all medicines including vitamins, probiotics, nutritional, food supplements under the 5 per cent GST slab. The rate rationalization comes days after Prime Minister Narendra Modi had pitched for introducing next-gen reforms across sectors to “increase ease of living for the common man and strengthen the economy.” However, this rationalization brings a revenue shortfall challenge for governments with states likely to bear a greater share of the burden, as they receive over 40 percent of the inter-state inflows in addition to their own SGST collections. Responding to a media query at the media briefing Arvind Shrivastava, Revenue Secretary said that, based on the consumption of FY23-24 the ministry has estimated that the net revenue implication of this proposal will be around Rs 48,000 crore. “However the exercise will trigger buoyancy effects and will have a positive impact on consumer and compliance. And therefore we believe this proposal will be fiscally sustainable both for centers and the states,” Shrivastava noted. ' S.no. Drug Indication Brand name and (Company) 1 Onasemnogene abeparvovec SMA (One-time gene therapy) Zolgensma (Novartis) 2 Asciminib Blood Cancer Scemblix (Novartis) 3 Mepolizumab Asthma Nucala (GSK) 4 Pegylated Liposomal Irinotecan Pancreatic Cancer Onivyde (Ipsen) 5 Daratumumab Cancer Darzalex (J&J subsidiary) 6 Daratumumab subcutaneous Cancer Darzalex Faspro (J&J subsidiary) 7 Teclistamab Blood Cancer Tecvayli (J&J subsidiary) 8 Amivantamab Lung Cancer Rybrevant (J&J subsidiary) 9 Alectinib Lung Cancer Alecensa (Roche) 10 Risdiplam SMA Evrysdi (Roche) 11 Obinutuzumab Blood Cancer Gazyva (Roche) 12 Polatuzumab vedotin Blood Cancer Polivy (Roche) 13 Entrectinib Cancer Rozlytrek (Roche) 14 Atezolizumab Cancer (PD-L1 immunotherapy) Tecentriq (Roche) 15 Spesolimab GPP flares Spevigo (Boehringer Ingelheim) 16 Velaglucerase Alpha Gaucher Vpriv (Takeda) 17 Agalsidase Alfa Fabry disease Replagal (Takeda) 18 Rurioctocog Alpha Pegol Hemophilia Adynovate (Takeda) 19 Idursulphatase Type II Mucopolysaccharidosis Elaprase (Takeda) 20 Alglucosidase Alfa Pompe Myozyme (Sanofi) 21 Laronidase Type I Mucopolysaccharidosis Aldurazyme (Sanofi) 22 Olipudase Alfa ASMD Xenpozyme (Sanofi) 23 Tepotinib Lung Cancer Tepmetko (Maerck KGaA) 24 Avelumab Cancer (PDL-1 immunotherapy) Bavencio (Pfizer) 25 Emicizumab Hemophilia Hemlibra (Roche subsidiary) 26 Belumosudil cGVHD - 27 Miglustat Gaucher Zavesca (J&J subsidiary) 28 Velmanase Alfa Alpha-mannosidosis - 29 Alirocumab Cholesterol lowering drug (LDL-C) Praluent (Sanofi) 30 Evolocumab Cholesterol lowering drug (LDL-C) Repatha (Amgen) 31 Cystamine Bitartrate Cystagon - 32 CI-Inhibitor injection Hereditary Angioedema - 33 Inclisiran Cholesterol lowering drug (LDL-C) Leqvio (Novartis) 34 Agalsidase Beta Fabry disease Fabrazyme (Sanofi) 35 Imiglucerase Gaucher Cerezyme (Sanofi) 36 Eptacog alfa activated recombinant coagulation factor VIIa Hemophilia NovoSeven Note: Above is the list of medicines exempted from GST tax recommended by the 56th GST Council Meet/ Source: PIB By Abhijeet Singh ,

Gene Therapy

31 Jul 2025

·www.globenewswire.com

Amicus Therapeutics Announces Second Quarter 2025 Financial Results and Corporate Updates

Q2 2025 Total Revenue of $154.7M, up 18% at CER Galafold® Q2 Revenue of $128.9M, up 12% at CER Pombiliti® + Opfolda® Q2 Revenue of $25.8M, up 58% at CER Reiterating 2025 Financial Guidance including GAAP Profitability During H2 2025 Conference Call and Webcast Today at 8:30 a.m. ET PRINCETON, N.J., July 31, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced financial results for the quarter ended June 30, 2025. "We delivered strong second quarter growth of 18%, marking the seventeenth consecutive quarter of double-digit gains at CER and reflecting excellent commercial execution for both Galafold and Pombiliti + Opfolda. Looking ahead, we expect Galafold to continue its growth trajectory, fueled by underlying patient demand, and Pombiliti + Opfolda to gain momentum with new patient starts across the U.S. and other key markets. Our collaboration with Dimerix on DMX-200 for FSGS is also progressing well, with the pivotal Phase 3 study expected to complete enrollment by year-end. Building on the performance of our two commercial therapies, we are firmly positioned to reach GAAP profitability in the second half of 2025 and to drive continued growth and patient benefit well into the future," said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. “With our unique, leverageable capabilities, the underlying momentum in our business and the significant remaining unmet patient need across markets in Fabry and Pompe diseases, Amicus anticipates exceeding $1 billion in total revenue in 2028 and furthering our position as a leading rare disease focused biotechnology company.” Second Quarter 2025 Financial Highlights: Total revenues for the second quarter 2025 were $154.7 million, reflecting strong operational growth measured at constant exchange rates (CER)1 of 18% and a currency tailwind of $5 million or 4%. (in thousands)Three Months Ended June 30, Year over Year % Growth Six Months Ended June 30, Year over Year % Growth 2025 2024 Reported at CER1 2025 2024 Reported at CER1Galafold®$128,872 $110,817 16% 12% $233,116 $210,176 11% 9%Pombiliti® + Opfolda®$25,816 $15,852 63% 58% $46,821 $26,896 74% 72%Net Product Revenues$154,688 $126,669 22% 18% $279,937 $237,072 18% 16% Galafold (migalastat) net product sales for the second quarter 2025 were $128.9 million, representing a year-over-year increase of 16%, or 12% at CER1, driven by continued commercial execution in all markets, net new patient starts, and strong compliance.Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales for the second quarter 2025 were $25.8 million, representing a year-over-year increase of 63%, or 58% at CER1, driven by high commercial demand and new launches in five countries.Total GAAP operating expenses, which include the upfront payment of $30 million for the U.S. licensing agreement of DMX-200, of $148.9 million for the second quarter 2025 increased by 48% as compared to $100.4 million for the second quarter 2024. Total non-GAAP operating expenses2, which also include the upfront payment of $30 million for the U.S. licensing agreement of DMX-200, were up 56% to $127.8 million for the second quarter 2025 as compared to $82.1 million for the second quarter 2024.GAAP net loss was $24.4 million, or $0.08 loss per share basic and diluted, for the second quarter 2025, compared to a net loss of $15.7 million, or $0.05 per share basic and diluted, for the second quarter 2024. Non-GAAP net income2,3 was $1.9 million, or $0.01 per share basic and diluted, for the second quarter 2025, compared to non-GAAP net income of $18.5 million, or $0.06 per share basic and diluted, for the second quarter 2024.Cash, cash equivalents, and marketable securities totaled $231.0 million at June 30, 2025 as compared to $249.9 million at December 31, 2024. Current cash position reflects the $30 million upfront payment in the second quarter for the U.S. licensing agreement of DMX-200. Corporate Updates: Pombiliti + Opfolda regulatory approval granted in Japan for adult LOPD patients in June. The Company also continues to anticipate additional reimbursement agreements throughout the year and remains on track for up to 10 new launch countries in 2025.New analysis of data from the ERT-experienced cohort of the PROPEL study of cipaglucosidase alfa + miglustat published in Muscle and Nerve. As previously announced, in this new publication, based on a within group effect-size analysis, subjects who switched from alglucosidase alfa to cipaglucosidase alfa + miglustat achieved improvements or stability in most of the outcomes measured.ACTION3 Study of DMX-200 is on track for full enrollment by end of year. As announced in May, Amicus entered into a licensing agreement with Dimerix for exclusive rights for the U.S. commercialization of their Phase 3 asset, DMX-200. DMX-200 is a first in class treatment for FSGS, a rare and fatal kidney disease with no approved therapies and significant market potential. The ACTION3 study, being funded and executed by Dimerix, is on track for full enrollment by end of the year. 2025 Financial Guidance Amicus reiterates its financial guidance for 2025, as follows: Total Revenue Growth115% to 22% Galafold Revenue Growth110% to 15% Pombiliti + Opfolda Revenue Growth150% to 65% Gross MarginMid 80% Non-GAAP Operating Expenses4$380M to $400M(Incl. $30M Upfront License Payment)GAAP Net IncomePositive during H2 2025 1 In order to illustrate underlying performance, Amicus discusses its results in terms of constant exchange rate (CER) growth. This represents growth calculated as if the exchange rates had remained unchanged from those used in the comparative period. 2 Full reconciliation of GAAP results to the Company’s non-GAAP adjusted measures for all reporting periods appear in the tables to this press release.3 Amicus defines non-GAAP Net (Loss) Income as GAAP Net (Loss) Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, loss on impairment of assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, restructuring charges and income taxes.4 A reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure is not available without unreasonable effort due to high variability, complexity, and low visibility as to the items that would be excluded from the GAAP measure. Conference Call and WebcastAmicus Therapeutics will host a conference call and audio webcast today, July 31, 2025, at 8:30 a.m. ET to discuss the second quarter 2025 financial results and corporate updates. Participants and investors interested in accessing the call by phone will need to register using the online registration form. After registering, all phone participants will receive a dial-in number along with a personal PIN to access the event. A live audio webcast and related presentation materials can also be accessed via the Investors section of the Amicus Therapeutics corporate website at ir.amicusrx.com. Web participants are encouraged to register on the website 15 minutes prior to the start of the call. An archived webcast and accompanying slides will be available on the Company's website shortly after the conclusion of the live event. About Galafold Galafold® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of people living with Fabry disease may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan. U.S. INDICATIONS AND USAGEGalafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data. This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. U.S. IMPORTANT SAFETY INFORMATION ADVERSE REACTIONS: The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. USE IN SPECIFIC POPULATIONS: There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus. It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition. Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis. The safety and effectiveness of Galafold have not been established in pediatric patients. To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. About Pombiliti + OpfoldaPombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood. U.S. INDICATIONS AND USAGEPOMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). SAFETY INFORMATION HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full PRESCRIBING INFORMATION for OPFOLDA (miglustat) LINK. About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on X and LinkedIn. Non-GAAP Financial Measures In addition to financial information prepared in accordance with U.S. GAAP, this press release also contains adjusted financial measures that we believe provide investors and management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses and profitability on a forward-looking basis, a reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results. Forward Looking StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to the prospects and timing of the potential regulatory and pricing approval of our products, commercialization plans, manufacturing and supply plans, financing plans, the collaboration with Dimerix, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities and pricing and reimbursement authorities, are based on current information. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that required regulatory inspections may be delayed or not be successful and delay or prevent product approval; the potential that we may not be successful in negotiations with pricing and reimbursement authorities; the potential that we may not be successful in commercializing Galafold and/or Pombiliti and Opfolda in Europe, the UK, the US and other geographies; the potential that the Dimerix license agreement for of DMX-200 may not be successful, including without limitation expectations of the timing of Phase 3 clinical trial evaluating DMX-200; the likelihood of success of such clinical trial; the prospects for FDA approval of DMX-200 for FSGS or other indications; the estimated prevalence of FSGS; the achievement of any milestone and timing of any payments associated with milestones and the success of any efforts to commercialize DMX-200, including any projections of future financial performance or payments; the potential that we may not be able to manufacture or supply sufficient commercial products; and the potential that we will need additional funding to complete the manufacturing and commercialization of our products. With respect to statements regarding corporate financial guidance and financial goals and the expected attainment of such goals and projections of the Company's revenue, non-GAAP profitability and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2024 and our Quarterly Report on Form 10-Q to be filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof. CONTACT: Investors: Amicus Therapeutics Andrew FaughnanVice President, Investor Relationsafaughnan@amicusrx.com(609) 662-3809 Media: Amicus Therapeutics Diana Moore Head of Global Corporate Affairs and Communicationsdmoore@amicusrx.com(609) 662-5079 FOLD-G TABLE 1 Amicus Therapeutics, Inc.Consolidated Statements of Operations(Unaudited)(in thousands, except share and per share amounts) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 Net product sales$154,688 $126,669 $279,937 $237,072 Cost of goods sold 15,217 11,261 26,915 24,828 Gross profit 139,471 115,408 253,022 212,244 Operating expenses: Research and development 60,848 24,683 88,687 53,012 Selling, general, and administrative 84,543 73,576 176,370 161,605 Restructuring charges — — — 6,045 Loss on impairment of assets 1,702 — 1,702 — Depreciation and amortization 1,852 2,182 3,689 4,336 Total operating expenses 148,945 100,441 270,448 224,998 Loss from operations (9,474) 14,967 (17,426) (12,754)Other expense: Interest income 843 1,370 1,655 2,910 Interest expense (11,565) (12,512) (23,020) (24,948)Other income (expense) 1,015 (3,717) 1,565 (8,683)Loss before income tax (19,181) 108 (37,226) (43,475)Income tax expense (5,239) (15,805) (8,880) (20,641)Net loss attributable to common stockholders$(24,420) $(15,697) $(46,106) $(64,116)Net loss attributable to common stockholders per common share — basic and diluted$(0.08) $(0.05) $(0.15) $(0.21)Weighted-average common shares outstanding — basic and diluted 308,254,256 303,773,922 307,972,054 303,336,787 TABLE 2 Amicus Therapeutics, Inc.Consolidated Balance Sheets(in thousands, except share and per share amounts) June 30, 2025 December 31, 2024Assets Current assets: Cash and cash equivalents$158,702 $213,752 Investments in marketable securities 72,296 36,194 Accounts receivable 105,849 101,099 Inventories 154,875 118,782 Prepaid expenses and other current assets 46,285 34,909 Total current assets 538,007 504,736 Operating lease right-of-use assets, net 21,988 22,278 Property and equipment, less accumulated depreciation of $30,991 and $28,775 at June 30, 2025 and December 31, 2024, respectively 28,570 29,383 Intangible assets, less accumulated amortization of $7,430 and $5,802 at June 30, 2025 and December 31, 2024, respectively 15,570 17,198 Goodwill 197,797 197,797 Other non-current assets 13,371 13,641 Total Assets$815,303 $785,033 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable$13,893 $12,947 Accrued expenses and other current liabilities 145,144 127,300 Operating lease liabilities 8,610 8,455 Total current liabilities 167,647 148,702 Long-term debt 391,322 390,111 Operating lease liabilities 43,383 45,078 Other non-current liabilities 8,647 7,097 Total liabilities 610,999 590,988 Commitments and contingencies Stockholders’ equity: Common stock, $0.01 par value, 500,000,000 shares authorized, 308,064,329 and 299,041,653 shares issued and outstanding at June 30, 2025 and December 31, 2024, respectively 3,016 2,944 Common stock in treasury, at cost; 7,390 shares as of June 30, 2025 (71) — Additional paid-in capital 2,956,839 2,926,115 Accumulated other comprehensive income (loss): Foreign currency translation adjustment 30,935 5,302 Unrealized loss on available-for-sale securities (129) (207)Warrants — 71 Accumulated deficit (2,786,286) (2,740,180)Total stockholders’ equity 204,304 194,045 Total Liabilities and Stockholders’ Equity$815,303 $785,033 TABLE 3 Amicus Therapeutics, Inc.Reconciliation of Non-GAAP Financial Measures(in thousands)(Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024Total operating expenses - as reported GAAP$ 148,945 $ 100,441 $ 270,448 $ 224,998Research and development: Share-based compensation 2,393 3,061 6,397 7,932Selling, general and administrative: Share-based compensation 15,166 13,136 36,334 39,068Loss on impairment of assets 1,702 — 1,702 —Restructuring Charges — — — 6,045Depreciation and amortization 1,852 2,182 3,689 4,336Total operating expense adjustments to reported GAAP 21,113 18,379 48,122 57,381Total operating expenses - as adjusted$127,832 $82,062 $222,326 $167,617 TABLE 4 Amicus Therapeutics, Inc.Reconciliation of Non-GAAP Financial Measures(in thousands, except share and per share amounts)(Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 GAAP net loss $(24,420) $(15,697) $(46,106) $(64,116)Share-based compensation 17,559 16,197 42,731 47,000 Depreciation and amortization 1,852 2,182 3,689 4,336 Loss on impairment of assets 1,702 — 1,702 — Restructuring charges — — — 6,045 Income tax expense 5,239 15,805 8,880 20,641 Non-GAAP net income $1,932 $18,487 $10,896 $13,906 Non-GAAP net income attributable to common stockholders per common share — basic and diluted $0.01 $0.06 $0.04 $0.05 Weighted-average common shares outstanding — basic 308,254,256 303,773,922 307,972,054 303,336,787 Weighted-average common shares outstanding — diluted 309,651,693 307,022,626 309,520,474 307,598,542

License out/inFinancial StatementDrug ApprovalPhase 3Accelerated Approval

100 Deals associated with Alglucosidase Alfa

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KEGG	Wiki	ATC	Drug Bank
D03207	Alglucosidase Alfa	A16AB07	DB01272

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Glycogen Storage Disease Type II	European Union	Sanofi	28 Mar 2006
Glycogen Storage Disease Type II	Iceland	Sanofi	28 Mar 2006
Glycogen Storage Disease Type II	Liechtenstein	Sanofi	28 Mar 2006
Glycogen Storage Disease Type II	Norway	Sanofi	28 Mar 2006

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01526785 (ADVANCE, Pubmed \| J Neuromuscul Dis)	Phase 4	Glycogen Storage Disease Type II	90	4000L alglucosidase alfa	wrhnjddfyq(jpbriiweua) = glwgakuihv tlemrivdsk (felujinitk )	-	01 Sep 2022
NCT00486889 (CTgov)	Phase 4	Glycogen Storage Disease Type II	12	alglucosidase alfa	jzcarrbwgn = jzrfvwuwbr jttxqxrdcv (oakkxcnuet, dbhwyrkhte - hvenghtvcy) View more	-	26 Aug 2022
NCT03687333 (APOLLO-IOPD, PRNewswire) Manual	Phase 4	Glycogen Storage Disease Type II	10	Alglucosidase Alfa	xcyljmihmi(muijhvpxob) = cmqqgbjzpn wkztdbxvff (rjtncnaxqg, 39.93) View more	Positive	05 Aug 2022
NCT03687333 (APOLLO-IOPD, Pubmed \| Front Pharmacol) Manual	Phase 4	Glycogen Storage Disease Type II	10	Alglucosidase Alfa	dtzjvexigf(bmhsedgfjb) = jhhrysyvpx whttbncula (zfwsmwrlrk, 155.99) View more	Positive	27 Jun 2022
NCT03019406 (Mini-COMET, CTgov)	Phase 2	Glycogen Storage Disease Type II	22	Avalglucosidase Alfa (PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg)	wrdmmwapxf = lpiezobqrb wmwcmukdim (kfhnqnwtgz, viihwbhjkt - uhtnhzyqlt) View more	-	22 Nov 2021
				Avalglucosidase Alfa (PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg)	wrdmmwapxf = ndxwkdeeif wmwcmukdim (kfhnqnwtgz, gikekuttvw - cpgpqucuup) View more
NCT01410890 (PAPAYA, CTgov)	Phase 4	Glycogen Storage Disease Type II	21	Alglucosidase Alfa (Alglucosidase Alfa: <18 Years)	awddcctidw(djdbjubobo) = avdnjqgfxn zjhfdobjta (aozkmcbctp, 94600) View more	-	11 Jun 2021
				Alglucosidase Alfa (Alglucosidase Alfa: >=18 Years)	awddcctidw(djdbjubobo) = daydisvqfj zjhfdobjta (aozkmcbctp, 143000) View more
NCT00701701 (CTgov)	Phase 4	Glycogen Storage Disease Type II	4	Myozyme (Regimen A: Alglucosidase Alfa and Cyclophosphamide)	sqrcrhofkd = qmkxhtjfxs acbhbvjaei (frifxklkes, lskmsbpduy - tjjqhufycm) View more	-	12 Oct 2020
				Myozyme (Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate)	sqrcrhofkd = fybryzhlhj acbhbvjaei (frifxklkes, xacawkoyjm - jbcqextzdh) View more
NCT01526785 (Pubmed) Manual	Phase 4	Glycogen Storage Disease Type II	113	Alglucosidase alfa (IOPD)	oeasvzaxcs(icicjxbefy) = uahzyldxae mjmtgadism (fpwwskcyow ) View more	Positive	01 Oct 2018
				Alglucosidase alfa (LOPD)	oeasvzaxcs(icicjxbefy) = soxaaozjei mjmtgadism (fpwwskcyow ) View more
NCT00158600 \| NCT00455195 (LOTS, AAN2017)	Not Applicable	Glycogen Storage Disease Type II	68	Alglucosidase alfa	mrgwznqtdc(unhipjkvqu) = fhejacocdr zclngbpzmg (rrzidvhccc ) View more	Positive	18 Apr 2017
NCT01597596 (CTgov)	Phase 4	Glycogen Storage Disease Type II	4	alglucosidase alfa (Alglucosidase Alfa 4000 L Material (Non-US Participants))	nabujwuhbv = oldwpkyznb oriwawlnox (mvpiexuqth, zfwouuirsc - jnavnxwmlt) View more	-	18 Jan 2016
				alglucosidase alfa (Alglucosidase Alfa 160 L Material (US Participants))	qnqdsykmto(vsnuewvwaw) = ordrrficjg llqrbdisce (scmyazlrtr, 1.103) View more

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