RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (South Korea), Fast Track (United States)

Structure/Sequence

Sequence Code 1216633

Source: *****

Clinical Trials associated with Sebelipase Alfa

NCT02112994

/ CompletedPhase 2

A Multi-Center, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency

This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).

Start Date24 Jun 2014

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

NCT02193867

/ TerminatedPhase 2

A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.

Start Date06 Jun 2014

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

NCT01757184

/ CompletedPhase 3

A Multicenter, Randomized, Placebo-controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency

This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid lipase deficiency (LAL-D) (cholesteryl ester storage disease [CESD]).
Late-onset LAL-D is an underappreciated cause of cirrhosis, liver failure and dyslipidemia. There is currently no standard treatment for LAL-D other than supportive care. Enzyme replacement therapy may be a potential new treatment option for LAL-D participants.

Start Date22 Jan 2013

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

100 Clinical Results associated with Sebelipase Alfa

100 Translational Medicine associated with Sebelipase Alfa

100 Patents (Medical) associated with Sebelipase Alfa

Literatures (Medical) associated with Sebelipase Alfa

01 May 2026·PROTEIN EXPRESSION AND PURIFICATION

Pharmaceutical protein production by transgenic chickens: several viewpoints towards the next stage

Review

Author: Okuzaki, Yuya ; Nishijima, Ken-Ichi

Several pharmaceuticals produced by transgenic chickens, Kanuma and Epovet, are already in commercial use, which indicates the potential cost competitiveness of this approach as an "animal bioreactor." Since the establishment of reliable cultivation and genetic modification methods for chicken primordial germ cells, the robustness of this system has increased significantly. Moreover, even proteins that might otherwise be deleterious if expressed systemically may be produced when their expression is restricted to the oviduct through the use of tissue-specific promoters. In this review, we discuss several critical issues that must be addressed in order to advance chicken bioreactor systems to the next stage. First, glycosylation profiles should be optimized according to the intended application. Although chickens generally exhibit mammalian-like glycosylation patterns, egg white proteins are known to carry relatively short glycans that lack terminal galactose and sialic acid residues. Therefore, technologies enabling the introduction of appropriate glycan structures need to be developed. Second, the screening process for establishing transgenic lines should be minimized to reduce overall production costs. Recent advances in site-specific genome modification technology are promising in this regard, provided that patent disputes can be resolved. The integration of these emerging strategies into transgenic chicken bioreactor platforms could further enhance their competitiveness as systems for recombinant protein production.

01 Jan 2026·JOURNAL OF INHERITED METABOLIC DISEASE

Natural‐History Mapping of Lysosomal Storage Disorders ( LSDs ): Gaucher Disease as a Model for Precision Care

Review

Author: Vaishnaw, Maya ; Mistry, Pramod. K. ; Ain, Noor Ul

ABSTRACT:

Natural‐history datasets have become pivotal for drug development and for shaping clinical‐practice guidelines in rare diseases, yet many lysosomal storage disorders would benefit from deep phenotyping and modern analytic methods. Our objective was to integrate the past decade of genomic, cellular, treatment‐outcome, and regulatory advances into a practical framework for capturing and interpreting natural‐history data, using Gaucher disease (GD) as a paradigm. We reviewed more than 300 peer‐reviewed articles (2005–2025), FDA guidance documents, and output from large real‐world registries. Particular attention was paid to long‐read GBA sequencing, biomarkers such as lyso‐Gb1, emerging newborn screening programs, and preliminary observational work that points toward multi‐state disease modeling. Key observations include: (i) Whole‐gene sequencing has expanded genotype–phenotype maps, revealing more than 70 recombinant GBA alleles that confound panel tests; (ii) registry trajectories suggest that formal multi‐state models could capture treatment‐modified courses and silent endpoints—monoclonal gammopathy, malignancy, Parkinson's disease, pulmonary arterial hypertension—better than current summary statistics; (iii) lyso‐Gb1 outperforms legacy biomarkers and now serves as a second‐tier newborn‐screening marker; (iv) Robust natural‐history evidence has already underpinned regulatory approvals across several lysosomal disorders—including olipudase alfa for ASMD, cerliponase alfa for CLN2, vestronidase alfa for MPS VII, and sebelipase alfa for infantile‐onset LAL‐D—demonstrating that well‐curated registries can serve as viable external controls for future LSD submissions. The convergence of deep phenotyping, genotype‐aware analytics, and systematic biomarker capture promises to transform natural‐history registries from descriptive archives into predictive engines. Gaucher disease offers a working template that, when extended across the LSD spectrum, can accelerate precision care and the development of next‐generation therapeutics.Trial Registration:ClinicalTrials.gov identifier: NCT00358943, NCT03291223.

01 Dec 2025·MOLECULAR GENETICS AND METABOLISM

Survival, growth, and safety findings in patients with rapidly progressive, infantile-onset LAL-D: Results from the international LAL-D registry

Article

Author: Lacaille, Florence ; Vijay, Suresh ; Heras, Javier de Las ; Evans, Jennifer ; Abel, Florian

In symptomatic infants, lysosomal acid lipase deficiency (LAL-D; historically Wolman disease) is characterized by a rapidly progressive disease course of hepatosplenomegaly and liver disease. This course includes liver failure, malabsorption and growth failure, and systemic inflammation, such as hemophagocytic lymphohistiocytosis, typically leading to death by 6 months of age if untreated. Sebelipase alfa (KANUMA®; Alexion, AstraZeneca Rare Disease, Boston, MA) is a recombinant human LAL (a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme) approved for the treatment of LAL-D in infants with rapidly progressive disease as well as in children and adults with LAL-D. Previous studies showed that enzyme replacement therapy (ERT) with sebelipase alfa led to survival with improved growth and development, hematologic parameters, and liver parameters and was well tolerated. We report long-term outcomes in 29 patients with rapidly progressive LAL-D who were symptomatic in infancy using patient data from the International LAL-D Registry, an ongoing observational, multicenter, international registry (NCT01633489) that collects data on patients with LAL-D. Among these 29 patients treated with sebelipase alfa, 41 % were male, and 28 % had participated in clinical trials with sebelipase alfa. Median age (Q1, Q3) was 2.3 months (1.8, 3.1) at the start of ERT. Patients received a starting ERT dose of ≤1 mg/kg or ≥3 mg/kg per week. In patients who had participated in clinical trials of sebelipase alfa, the starting dose was driven by the clinical trial protocol and was between 0.2 and 1.0 mg/kg per week. Overall, 27 of 29 (93 %) patients survived during the median observation time (Q1, Q3) of 6.2 years (3.5, 8.4). At baseline, 11 patients had abnormally low weight-for-age z scores (<-2); for 5 of these patients, weight stabilized after 6 to 12 months of treatment (z scores between -2 and 2). Adverse events occurred in 23 (79 %) patients. Eleven (38 %) patients experienced adverse events potentially related to sebelipase alfa, which were generally not severe and most resolved. Four patients among 7 tested developed antidrug antibodies, and 3 had positive results for neutralizing antidrug antibodies. These results confirmed the dramatic survival and metabolic benefit associated with sebelipase alfa ERT in patients with symptomatic, rapidly progressive LAL-D.

News (Medical) associated with Sebelipase Alfa

29 Feb 2024

·www.pharmaceutical-technology.com

NHS expedites availability of Belumosudil for chronic GVHD

The once-daily tablet hinders proteins that trigger the severe inflammatory response linked to chronic GVHD. Credit: fizkes / Shutterstock.com. The National Health Service (NHS) in the UK has expedited the availability of the new drug, Belumosudil (Rezurock), for cancer patients with chronic graft versus host disease (GVHD). The decision provides a treatment that can be administered in the at-home setting, reducing hospital visits. Administered as a once-daily tablet, Belumosudil hinders proteins that trigger the severe inflammatory response linked to chronic GVHD. Clinical trials have demonstrated that up to three-quarters of the subjects responded to the therapy, with significant effectiveness in treating symptoms affecting the mouth, intestine or joints. The treatment is accessible through the NHS England’s Innovative Medicines Fund, which fast-tracks the delivery of the most promising non-cancer drugs to patients. See Also: AbbVie licenses OSE’s chronic inflammation therapy for $48m upfront DNTH-103 by Dianthus Therapeutics for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): Likelihood of Approval Chronic GVHD is a rare but serious condition that can occur after a life-saving stem cell transplant. White blood cells in the donated tissue attack the patient’s organs and weaken their immune system. Approximately one-third of patients who receive stem cell or bone marrow transplants for blood cancers such as leukaemia and lymphoma can develop chronic GVHD. Around 1,200 patients in England undergo such transplants each year. Around 400 go on to develop the condition, which is sometimes mild but for one in ten cases is fatal. For half of patients with chronic GVHD, existing therapies such as pentostatin, rituximab and imatinib are ineffective. Belumosudil will now be made available for 200 severely affected patients who have not responded to two previous therapies. This development comes after the NHS decided to fast-track the funding for the drug based on clinical guidance from the UK National Institute of Health and Care Excellence ( Nice ). Belumosudil is the fourth therapy offered to patients through the Innovative Medicines Fund. In September 2023, the NHS used the fund to roll out Sebelipase alfa, a therapy for treating Wolman’s disease, a rare genetic ailment. NHS England specialised services medical director James Palmer stated: “This rollout is a real breakthrough for patients with graft versus host disease and it will help people living with this debilitating condition effectively manage their symptoms and enhance their quality of life and daily functioning. “It’s great news that the NHS has been able to fast-track funding for a treatment which will have a significant impact for hundreds of patients over the coming years.”

Clinical Result

27 Nov 2023

·www.pmlive.com

AstraZeneca's rare disease unit receives NICE recommendation for Wolman disease therapy in infants

AstraZeneca's rare disease unit has received a recommendation from the National Institute for Health and Care Excellence (NICE) for the use of its enzyme replacement therapy in infants with Wolman disease. Alexion’s Kanuma (sebelipase alfa), which has been specifically recommended for use in patients who are aged two years or younger when administration begins, will now become the first treatment available on the NHS for the rapidly-progressive rare genetic disease. Occurring in around one in 350,000 births, Wolman disease causes a build-up of fat in cells in the liver, heart, blood vessels and digestive system. Symptoms in infants include enlarged liver and spleen, poor weight gain, low muscle tone, jaundice, vomiting, diarrhoea, developmental delay and anaemia. Until now, standard care for the disease has been palliative and limited to managing symptoms, with patients normally not surviving past the age of one without treatment. Administered as weekly intravenous infusions which can be given at home alongside a restricted low-fat diet, Kanuma works by replacing an enzyme missing in the body. Some patients may also have a blood and marrow/stem cell transplant. NHS chief executive, Amanda Pritchard, said: “I am delighted the NHS can now, for the first time, offer a life-changing treatment to families facing this enormously difficult condition. “Where previously there were no treatments available for infants facing this debilitating disease, this new therapy could save families from facing indescribable grief and allow more children… to grow up, go to school and live normal lives.” The therapy will now be fast-tracked via NHS England’s Innovative Medicines Fund to enable routine patient access up to five months earlier than would otherwise be the case. Sean Richardson, vice president and general manager, Alexion, UK, described NICE’s decision as a “milestone moment for infants born with Wolman Disease and their families”. He continued: “The recommendation is the result of continued constructive collaboration between Alexion, NICE, NHS England, patient groups and the medical community, to ensure babies born with this life-threatening disease have a treatment available to them.”

Drug Approval

02 Oct 2023

·www.prnewswire.com

Global Enzyme Replacement Therapy (ERT) Markets, 2017-2022, 2022-2027F, 2032F: Lamzede's FDA Approval Paves the Way for a Brighter Future in Enzyme Replacement Therapies

DUBLIN, Oct. 2, 2023 /PRNewswire/ -- The "Enzyme Replacement Therapy Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering. The global enzyme replacement therapy market is expected to grow from $10.03 billion in 2022 to $10.85 billion in 2023 at a compound annual growth rate (CAGR) of 8.1%. The market is expected to reach $14.79 billion in 2027 at a CAGR of 8.1%. In the forefront of medical advancement, the enzyme replacement therapy (ERT) market stands tall, offering vital treatment avenues for individuals grappling with chronic disorders arising from enzyme deficiencies. The research report delves deep into the nuances of the ERT sector, highlighting market statistics, regional shares, and key players commanding significant market share. ERT's pivotal role in normalizing urinary GAG levels and promoting liver and spleen health is becoming increasingly recognized. Primary enzymes like agalsidase alfa - a critical tool in the combat against Fabry's disease - are reshaping the therapeutic landscape. Our comprehensive report also categorizes other prominent enzymes used, shedding light on their applications in conditions such as Gaucher disease, Pompe disease, and more. A spotlight trend in the ERT realm is the surge in product approvals. For instance, the recent FDA nod to Lamzede (velmanase alfa) in February 2023 marked a significant milestone. As the inaugural enzyme replacement therapy greenlit in the U.S for tackling the non-central neurological manifestations of alpha-mannosidosis, Lamzede offers a promising beacon of hope. Moreover, with the escalating prevalence of rare, chronic, and genetic disorders, the ERT market trajectory looks set for robust growth. The U.S National Library of Medicine projects a near-doubling of individuals aged 50 and above with a chronic ailment by 2050, further emphasizing the critical need for effective enzyme replacement therapies. The report also touches upon the market revenues, detailing contributions from key treatments like Sebelipase alfa, Elosulfase alfa, and more. Our exhaustive coverage ensures businesses and stakeholders are well-armed with insights to navigate the dynamic ERT ecosystem. In a geographical context, while North America reigned supreme in the ERT domain in 2022, the forecasted period paints a promising picture for the Asia-Pacific region. Our study covers a broad spectrum of nations from Australia to the USA, offering a global perspective on this burgeoning market. Harness the power of data-driven insights, industry trends, and strategic opportunities with our comprehensive report, and be poised at the cusp of ERT innovations and advancements. Major players in the enzyme replacement therapy market are Biomarin Pharmaceutical Inc. Leadiant Biosciences Inc. Pfizer Inc. Sanofi S.A. AbbVie Inc. Takeda Pharmaceutical Company Limited Protalix BioTherapeutics Inc. Amicus Therapeutics Inc. Johnson & Johnson Services Inc. Digestive Care Inc. Nestle Health Science Alexion Pharmaceuticals Inc. Allergan Plc. Actelion Pharmaceuticals Ltd. CANbridge Life Sciences Ltd. Chiesi Farmaceutici S.P.A. Astrazeneca Plc. Genzyme Corporation JCR Pharmaceutical Co. Ltd. TEIJIN LIMITED Horizon Therapeutics Plc. Perseo Pharma AG SmartPharm Therapeutics Inc. Key Topics Covered: 1. Executive Summary 2. Enzyme Replacement Therapy Market Characteristics 3. Enzyme Replacement Therapy Market Trends And Strategies 4. Enzyme Replacement Therapy Market - Macro Economic Scenario 4.1. COVID-19 Impact On Enzyme Replacement Therapy Market 4.2. Ukraine-Russia War Impact On Enzyme Replacement Therapy Market 4.3. Impact Of High Inflation On Enzyme Replacement Therapy Market 5. Enzyme Replacement Therapy Market Size And Growth 5.1. Global Enzyme Replacement Therapy Historic Market, 2017-2022, $ Billion 5.1.1. Drivers Of The Market 5.1.2. Restraints On The Market 5.2. Global Enzyme Replacement Therapy Forecast Market, 2022-2027F, 2032F, $ Billion 5.2.1. Drivers Of The Market 5.2.2. Restraints On the Market 6. Enzyme Replacement Therapy Market Segmentation 6.1. Global Enzyme Replacement Therapy Market, Segmentation By Enzyme Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Agalsidase Alfa Agalsidase Beta Galsulfase Other Enzyme Types 6.2. Global Enzyme Replacement Therapy Market, Segmentation By Application, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Gaucher Disease Pompe Disease Fabry Disease Other Applications 6.3. Global Enzyme Replacement Therapy Market, Segmentation By Route Of Administration, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Oral Parenteral 6.4. Global Enzyme Replacement Therapy Market, Segmentation By End-Users, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Hospitals Infusion Centers Other End-Users 7. Enzyme Replacement Therapy Market Regional And Country Analysis 7.1. Global Enzyme Replacement Therapy Market, Split By Region, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion 7.2. Global Enzyme Replacement Therapy Market, Split By Country, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1904 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

100 Deals associated with Sebelipase Alfa

External Link

KEGG	Wiki	ATC	Drug Bank
D10377	Sebelipase Alfa	A16AB14	DB11563

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Cholesterol Ester Storage Disease	United Kingdom	AstraZeneca PLC	31 Jan 2016
Wolman Disease	European Union	Alexion Europe SAS	28 Aug 2015
Wolman Disease	Iceland	Alexion Europe SAS	28 Aug 2015
Wolman Disease	Liechtenstein	Alexion Europe SAS	28 Aug 2015
Wolman Disease	Norway	Alexion Europe SAS	28 Aug 2015

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Failure to Thrive	Phase 3	United States	Alexion Pharmaceuticals, Inc.	04 May 2011
Failure to Thrive	Phase 3	Egypt	Alexion Pharmaceuticals, Inc.	04 May 2011
Failure to Thrive	Phase 3	France	Alexion Pharmaceuticals, Inc.	04 May 2011
Failure to Thrive	Phase 3	Ireland	Alexion Pharmaceuticals, Inc.	04 May 2011
Failure to Thrive	Phase 3	United Kingdom	Alexion Pharmaceuticals, Inc.	04 May 2011
Myocarditis	Phase 2	United States	Alexion Pharmaceuticals, Inc.	12 Dec 2011
Myocarditis	Phase 2	Canada	Alexion Pharmaceuticals, Inc.	12 Dec 2011
Myocarditis	Phase 2	Czechia	Alexion Pharmaceuticals, Inc.	12 Dec 2011
Myocarditis	Phase 2	France	Alexion Pharmaceuticals, Inc.	12 Dec 2011
Myocarditis	Phase 2	United Kingdom	Alexion Pharmaceuticals, Inc.	12 Dec 2011

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02112994 (Pubmed \| J Pediatr Gastroenterol Nutr) Manual	Phase 2	Wolman Disease	31	Sebelipase Alfa	rshtvzsyki(qtsztdoyjp) = positive 2 pts tkjevvgfre (eencbwebho ) View more	Positive	20 Apr 2022
				Sebelipase alfa 3.0 mg/kg qow and 3.0 mg/kg weekly
NCT01757184 (ARISE, Pubmed \| J Hepatol) Manual	Phase 3	Wolman Disease	59	Sebelipase Alfa	zhceeeidpr(bvyljqaibx) = ooqthgeafw xochgjxgyt (immujunndm ) View more	Positive	01 Mar 2022
NCT01488097 (LAL-CL04, Pubmed \| Liver Int) Manual	Phase 2	Wolman Disease	8	Sebelipase alfa	cvcxszizfz(puxlhpoozh) = fixvwjxono ealoojawoc (foebonschv ) View more	Positive	01 Sep 2020
				Sebelipase alfa 3.0 mg/kg	-
NCT02193867 (CTgov)	Phase 2	Wolman Disease	10	Sebelipase Alfa	kecdjlrcej = zoqgbdjtwh iaejuhdrcf (fopqklulxm, gxcltmezvs - nyvnujjpfq) View more	-	18 Nov 2019
SSIEM2019	Not Applicable	Wolman Disease	19	Sebelipase alfa	cxvlpplrst(brnwciwffq) = Of 17 patients tested for ADAs, 10 had detectable titers and 8 developed neutralizing antibodies cudotaagjg (amvvsqsgvg ) View more	-	25 Aug 2019
EASL2019	Not Applicable	Wolman Disease	31	Sebelipase alfa 1.0 mg/kg	dzcbmkmlhc(qskllfzikd) = ircglmqrij tylpauyolj (gtdykrvfpx ) View more	Positive	13 Apr 2019
				Sebelipase alfa 3.0 mg/kg	dzcbmkmlhc(qskllfzikd) = qdwivfbvur tylpauyolj (gtdykrvfpx ) View more
NCT02112994 (CTgov)	Phase 2	Wolman Disease	31	Sebelipase Alfa	pwbfpnrisg = bgczouiulw ttilvltkxs (wnpvxckgdz, kaqnwyquho - hlyhkmhnlu) View more	-	15 Jan 2019
NCT01307098 (CTgov)	Phase 1/2	Wolman Disease	9	Sebelipase alfa 0.35 mg/kg (Sebelipase Alfa 0.35 mg/kg)	aguquahlcc = lhnsjnxcjc jeuxgmndgj (jxzskkdxwt, bdfxqqdmmt - qfjtctvmao) View more	-	11 Dec 2018
				Sebelipase alfa 1 mg/kg (Sebelipase Alfa 1 mg/kg)	aguquahlcc = ylhtjejucn jeuxgmndgj (jxzskkdxwt, hbgopqgbol - lszqyzrhrh) View more
NCT01371825 (VITAL, Pubmed \| Orphanet J Rare Dis) Manual	Phase 2/3	Wolman Disease	30	sebelipase Alfa	yxgqmyphlb(foldzitwzk) = mcudgkcvlv tprejotlfd (gttyrzrptr, 30 - 93) View more	Positive	08 Feb 2017
				historical control	yxgqmyphlb(foldzitwzk) = acnwnpknpl tprejotlfd (gttyrzrptr, 0 - 16)
NCT01488097 (LAL-CL04, CTgov)	Phase 2	Wolman Disease	8	sebelipase alfa	myobxhhvhj = hxawnbliwj rhedfqqxgz (tlcflbcumj, kvgpyjxumw - sxfumrxdfb) View more	-	09 May 2016

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