RegulationOrphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Orphan Drug (South Korea), Orphan Drug (Australia), Fast Track (United States)

Structure/Sequence

Molecular FormulaC15H9FN2O3

InChIKeyOOUGLTULBSNHNF-UHFFFAOYSA-N

CAS Registry775304-57-9

Clinical Trials associated with Ataluren

JPRN-jRCT2071220020

/ CompletedPhase 1IIT

A Single-Dose Study to Assess the Food Effect on the Pharmacokinetics and Tolerability of Ataluren (PTC124) in Healthy Japanese Subjects

Start Date24 Jun 2022

Sponsor / Collaborator-

NCT04336826

/ CompletedPhase 2

An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy

This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).

Start Date29 Dec 2021

Sponsor / Collaborator

PTC Therapeutics, Inc.

CTR20211795

/ CompletedPhase 3

一项 ATALUREN (PTC124) 在无义突变型杜兴氏肌营养不良患者中的长期安全性、疗效和耐受性的开放性研究

[Translation] An open-label study of the long-term safety, efficacy, and tolerability of ATALUREN (PTC124) in patients with nonsense mutations in Duchenne muscular dystrophy

本研究的目标在于评估长期 Ataluren 10 mg/kg 、10 mg/kg 和 20 mg/kg 给药在完成 III 期研究 (PTC124-GD-041-DMD )后希望继续接受治疗的 nmDMD 受试者中的安全性、疗效和耐受性。将根据所有不良事件 (AE)、实验室检查异常、生命体征、心电图 (ECG)、超声心动图和体格检查结果的类型、频率、严重程度、时间以及与 Ataluren 的关系来描述 Ataluren 的安全性特征。Ataluren 的疗效将根据上肢功能 (PUL)、DMD 上肢患者报告结局指标 (PROM)、肺量测定、行走能力丧失 (LoA)和 6 分钟步行试验 (6MWT)较基线的变化来评估。

[Translation]

The objective of this study was to evaluate the safety, efficacy, and tolerability of long-term dosing of Ataluren 10 mg/kg, 10 mg/kg, and 20 mg/kg in nmDMD subjects who wished to continue treatment after completing a Phase III study (PTC124-GD-041-DMD). The safety profile of ataluren will be described based on the type, frequency, severity, timing, and relationship to ataluren of all adverse events (AEs), laboratory abnormalities, vital signs, electrocardiogram (ECG), echocardiogram, and physical examination findings. The efficacy of ataluren will be evaluated based on change from baseline in upper limb function (PUL), DMD upper limb patient-reported outcome measures (PROMs), spirometry, loss of ambulation ability (LoA), and 6-minute walk test (6MWT).

Start Date14 Sep 2021

Sponsor / Collaborator

ROVI Pharma Industrial Services SA

[+2]

100 Clinical Results associated with Ataluren

100 Translational Medicine associated with Ataluren

100 Patents (Medical) associated with Ataluren

336

Literatures (Medical) associated with Ataluren

01 Mar 2026·NEUROLOGICAL SCIENCES

Patient reported outcome measures in spinal muscular atrophy and duchenne muscular dystrophy: review of instruments and their inclusion in clinical and regulatory processes

Review

Author: Spataro, Clarissa ; D'amico, Adele ; Scopinaro, Annalisa ; Sansone, Valeria ; Meregaglia, Michela ; Malandrini, Francesco ; Ciani, Oriana

Abstract:

Objectives:

This study aims to analyze the use of patient-reported outcomes measures (PROMs), observer-reported outcome measures (ObsROMs), and caregiver-reported outcome measures (CROMs) in spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). The objectives are twofold: (1) to identify and characterize available instruments to be used in research and clinical practice, and (2) to assess their inclusion in drug development and regulatory assessment processes.

Methods:

A systematic search was conducted using PubMed, Google Scholar, Scopus, and the ePROVIDE database to identify PROMs, ObsROMs, and CROMs for SMA and DMD. The identified instruments were analyzed for validation, psychometric properties, Minimal Clinically Important Difference (MCID), and recall period. Additionally, clinical trial protocols, relative study publications, European Public Assessment Report (EPAR), and Italian Medicines Agency (AIFA) reports for innovativeness recognition on medicines for SMA and DMD (i.e., nusinersen, onasemnogene abeparvovec, risdiplam, and ataluren) were reviewed to evaluate the inclusion of these measures in drug development and regulatory assessment.

Results:

The initial search identified 50 questionnaires, including 40 PROMs, 5 ObsROMs, and 5 CROMs. Of these, 15 (30.0%) instruments were included in pivotal clinical trial protocols, with none designated as primary endpoints. Only 6 (12.0%) instruments were mentioned in EPARs, and MCID determination was reported for 6 (12.0%) of the instruments. Generic instruments like the PedsQL were frequently used but criticized for limited specificity.

Conclusions:

Despite the availability of PROMs, ObsROMs, and CROMs for SMA and DMD, their use in clinical trials and regulatory documents is limited and inconsistent. Greater standardization and systematic inclusion of these measures are needed to support patient-centered drug development and evaluation.

01 Mar 2026·SPECTROCHIMICA ACTA PART A-MOLECULAR AND BIOMOLECULAR SPECTROSCOPY

Unraveling the interaction mechanism between the genetic disease drug Ataluren and eukaryotic codon recognition factor 1/ human serum albumin through spectroscopic and nuclear magnetic resonance approaches

Article

Author: Tian, Hong ; Duan, Ran ; Yu, Juping ; Guo, Hanlin ; Peng, Zhengxiong ; Yu, Xinyu ; Liu, Wei

PTC124 (Ataluren) is a small molecule drug for the treatment of genetic diseases that has been shown to promote early termination codon read-through. In this study, the binding model of PTC124 to human serum albumin and eukaryotic stop codon recognition factor (eRF1) was established in vitro. In the cell model, the read-through effect of PTC124 on the stop codon was confirmed. Fluorescence analysis showed that PTC124 could spontaneously bind eRF1 and HSA, the binding constants were 3.132 ± 0.643 × 10⁴ and 2.963 ± 0.218 × 10⁵ L/mol (298 K), respectively. Thermodynamic analysis indicated that the binding to eRF1 was driven mainly by hydrophobic interactions, hydrogen bonding, and van der Waals forces, whereas the binding to HSA was driven primarily by hydrogen bonding and van der Waals forces. Structural analysis revealed perturbations in the secondary structure of both HSA and eRF1 induced by PTC124, while the overall native folds of the proteins were maintained. In addition, a binding model of the interaction between PTC124 and eRF1/HSA was established by molecular simulation and STD-NMR. The binding model described in this paper explains the interaction mechanism between PTC124 and its utility and transport targets at the molecular level, which is helpful for the design and synthesis of new molecules related to PTC124 structure and based on its mechanism of action.

01 Mar 2026·Molecular Therapy-Nucleic Acids

Beyond the stop: Oxadiazole TRIDs restore LRBA protein expression in nonsense-driven primary immunodeficiency

Article

Author: Lentini, Laura ; Moutschen, Michel ; Pibiri, Ivana ; Meier-Menches, Samuel M ; Pace, Andrea ; Zito, Antonino ; Ricci, Davide ; Vitale, Emanuele ; Borutzki, Yasmin ; Fiduccia, Ignazio ; Bileck, Andrea ; Colige, Alain ; Varrica, Riccardo ; Marino, Sefora

Nonsense mutations are among the genetic causes of LRBA (lipopolysaccharide-responsive beige-like anchor) deficiency, a rare autosomal-recessive immunodeficiency disorder. These mutations introduce premature stop codons, leading to the loss of LRBA protein expression. Following the recent market withdrawal of ataluren, the only approved translational readthrough-inducing drug (TRID), there is an urgent need for alternative therapeutic options. In this study, we investigated the efficacy of three 1,2,4-oxadiazole-based TRIDs-NV848, NV914, and NV930-using primary fibroblasts from a patient homozygous for the R1683X nonsense mutation. All compounds restored full-length LRBA protein with correct cytoplasmic localization, as confirmed by western blot and immunofluorescence, outperforming ataluren in readthrough efficiency. NV848 exhibited the strongest activity and uniquely increased LRBA mRNA levels, suggesting transcript stabilization. In contrast, NV930 and NV914 induced readthrough without stabilizing mRNA. Global proteomic profiling revealed minimal off-target effects for NV848, limited protein modulation by NV914, and widespread variations of 828 proteins by NV930, affecting pathways related to vesicular transport and mRNA splicing. However, network analysis revealed poor connectivity among differentially expressed proteins, with LRBA unrelated to any regulated cluster. These findings highlight the reported molecules as promising candidates for precision therapy in LRBA deficiency and shed light on the broader cellular impact of TRIDs.

News (Medical) associated with Ataluren

10 Mar 2026

·www.fiercebiotech.com

Republican senator slams FDA rare disease rebuffs, pledges to investigate recent actions: Bloomberg

Wisconsin Sen. Ron Johnson told Bloomberg that he may call on top FDA leaders to testify before the Senate’s Permanent Subcommittee on Investigations.\n As tensions around the FDA continue to escalate, a Republican U.S. senator has announced an investigation into recent approval rejections for rare disease candidates, according to Bloomberg News. Wisconsin Sen. Ron Johnson is asking to see several of the complete response letters that have been delivered of late and also told Bloomberg that he may call on top FDA leaders to testify before the Senate’s Permanent Subcommittee on Investigations, which Johnson chairs.The probe comes on the heels of heightened interactions between gene therapy developer uniQure and the FDA as an unnamed senior agency official—widely believed to be the now-departing Vinay Prasad, M.D.—defended the FDA’s demand that the biotech launch another sham-controlled trial for its Huntington’s disease candidate. The one-time uniQure therapy is given directly into the brain through small holes in the skull. Now, Johnson is categorizing the request for a new trial as “bureaucratic idiocy,” according to Bloomberg. “You’re expecting people to go through sham surgeries where they get holes drilled in their heads?” Johnson said to the outlet. “That’s just unbelievable.” On March 2, uniQure leaders argued that a sham-controlled study “could impose significant risks and burden to patients—some might even consider this trial design to be unethical.” A few days later, the anonymous FDA official denied that the control group would require that patients get holes drilled in their heads. “We asked uniQure to take their product and randomize patients to getting the treatment the way they give the treatment, which requires a skin incision, a hole in the skull and an intracranial injection of the product, versus a control arm, where all they do is anesthetize the patient and put one to three nicks in the scalp,” the unknown official said on March 5. “We did not ask them to compare against a partial sham burr hole.”One day later, news broke that Prasad would be departing his role as the FDA’s top regulator of vaccines and cell and gene therapies at the end of April—the second time the leader has left the agency. A search is currently underway for Prasad’s successor.“The stories are so outrageous,” Johnson said to Bloomberg. “It just appears that they’re looking for excuses to say no.”In response, Department of Health and Human Services spokesperson Andrew Nixon told Fierce that the FDA “stands by the scientists evaluating these applications and each rationale is explained in detail through CRL’s, which, for the first time, are made available to the public.” Under the second Trump administration, the drug regulator pledged to publicly release CRLs at the same time they are issued to sponsoring companies, part of what the FDA calls a push toward “radical transparency.” The agency has released batches of certain rejection letters, but it has not been consistent in what is published and when. “The number of FDA approvals and rejections under this administration are consistent with historical data over the last decade. In fact, CBER had a record number of approvals in December,” HHS’ Nixon said.The agency did not provide the referenced data behind this claim when asked.When asked about the intensifying pushback from the rare disease community, the HHS spokesperson said, “Our obligation is to patients and public health, and we remain committed to a regulatory approach that brings safe, effective individualized treatments to patients who urgently need them.”He also cited the agency’s plausible mechanism framework, which is draft guidance for an approval pathway that could see custom CRISPR therapies formally embraced by the agency. “This FDA is doing more than any other time in history to help speed up the development of ultra-rare disease cures for the American people,” the spokesperson said. In late February, a panel of physicians, biotech leaders and patient advocates took aim at the FDA during a Senate hearing chaired by Sen. Rick Scott, R-Fla., on how the agency’s bureaucracy affects innovation.Sen. Johnson spoke during that hearing, which included a discussion of the FDA’s November rejection of Biohaven’s experimental drug, troriluzole, for spinocerebellar ataxia. “I wrote six letters to FDA leadership between 2023 and 2025, cosigned by 17 ataxia colleagues asking FDA to review the application again and work with Biohaven to make the drug available [and], if necessary, performing post-marketing studies. I never heard back,” Jeremy Schmahmann, M.D., a neurology professor at Harvard Medical School and founding director of Massachusetts General Hospital’s Ataxia Center, said during the hearing.Johnson told Bloomberg that he is also upset about the spinocerebellar ataxia rebuff, adding that he doesn’t understand why FDA officials aren’t listening to specialists who say rare disease candidates are helping their patients.The senator will be holding a press conference alongside patients and families who are losing access to ataluren, also known as Translarna, a treatment designed to treat Duchenne muscular dystrophy (DMD). In mid-February, drugmaker PTC Therapeutics revealed that it withdrew its FDA application for Translarna in nonsense mutation DMD after receiving feedback from the regulator. As of publication, Johnson’s office has not responded to Fierce Biotech’s inquiries for additional comment.

19 Feb 2026

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results

– Full-year 2025 product and royalty revenue of $831M, exceeding guidance – – Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of $92M and $111M, respectively – – Cash of $1.95B as of December 31, 2025 – WARREN, N.J., Feb. 19, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2025. "We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs, and strong financial position, we look forward to continued success as we approach cash flow breakeven." Key Corporate Highlights Full-year 2025 product and royalty revenue of $831 million, exceeding guidance Global launch of Sephience off to strong start Q4 2025 revenue of $92 million, including $81 million revenue in the US and $11 million revenue ex-US Sephience total net revenue of $111 million in 2025 since launch 946 total patients on commercial therapy worldwide as of December 31, 2025 1,134 patient start forms received in the US as of December 31, 2025 Approval in Japan in December 2025; approval in Brazil in February 2026 Sephience global footprint expected to increase to 20 to 30 countries by end of 2026 In December 2025, PTC sold the remainder of its Evrysdi® (risdiplam) royalty to Royalty Pharma for $240 million upfront and up to $60 million in sales-based milestones; PTC maintains the right to receive a $150 million milestone from Roche based on single-year Evrysdi sales of $2.5 billion End-of-Phase 2 meeting with FDA held in Q4 2025 to discuss the votoplam Huntington's disease (HD) program Alignment reached on design of global Phase 3 trial, INVEST-HD, which is planned to initiate in 1H 2026 FDA confirmed openness for potential Accelerated Approval pathway given significant unmet need Type C meeting with FDA held in December 2025 to discuss the vatiquinone Friedreich's ataxia program; FDA indicated that an additional study would be necessary to support NDA resubmission and meeting minutes stated that this could be an open-label study with a natural history control group Fourth Quarter and Full Year 2025 Financial Highlights Total revenues were $164.7 million for the fourth quarter of 2025, compared to $213.2 million for the fourth quarter of 2024. Total revenues were $1,730.7 million for full year 2025, compared to $806.8 million for full year 2024. Included in total revenues is collaboration and license revenue of $998.4 million for the full year 2025, related to the votoplam license and collaboration agreement with Novartis, which closed in January 2025. Total net product revenues were $184.0 million for the fourth quarter of 2025, compared to $150.1 million for the fourth quarter of 2024. Total net product revenues were $586.7 million for full year 2025, compared to $582.1 million for full year 2024. Translarna™ (ataluren) net product revenues were $39.0 million for the fourth quarter of 2025, compared to $89.1 million for the fourth quarter of 2024. Translarna net product revenues were $235.3 million for full year 2025, compared to $321.1 million for full year 2024. Emflaza® (deflazacort) net product revenues were $27.1 million for the fourth quarter of 2025, compared to $50.5 million for the fourth quarter of 2024. Emflaza net product revenues were $146.4 million for full year 2025, compared to $207.2 million for full year 2024. Roche reported Evrysdi full year 2025 sales of approximately 1,757 CHF million, resulting in royalty revenue of $244.2 million to PTC for full year 2025, compared to $203.9 million to PTC for full year 2024. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $133.1 million for the fourth quarter of 2025, compared to $124.8 million for the fourth quarter of 2024. GAAP R&D expenses were $455.2 million for full year 2025, compared to $534.5 million for full year 2024. Non-GAAP R&D expenses were $124.3 million for the fourth quarter of 2025, excluding $8.8 million in non-cash, stock-based compensation expense, compared to $116.0 million for the fourth quarter of 2024, excluding $8.8 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $419.6 million for full year 2025, excluding $35.7 million in non-cash, stock-based compensation expense, compared to $497.9 million for full year 2024, excluding $36.6 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $96.9 million for the fourth quarter of 2025, compared to $84.7 million for the fourth quarter of 2024. GAAP SG&A expenses were $347.1 million for full year 2025, compared to $300.9 million for full year 2024. Non-GAAP SG&A expenses were $87.2 million for the fourth quarter of 2025, excluding $9.7 million in non-cash, stock-based compensation expense, compared to $76.3 million for the fourth quarter of 2024, excluding $8.4 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $308.3 million for full year 2025, excluding $38.9 million in non-cash, stock-based compensation expense, compared to $262.9 million for full year 2024, excluding $38.0 million in non-cash, stock-based compensation expense. Net loss was $135.0 million for the fourth quarter of 2025, compared to net loss of $65.9 million for the fourth quarter of 2024. Net income was $682.6 million for full year 2025, compared to net loss of $363.3 million for full year 2024. Cash, cash equivalents, and marketable securities were $1,945.4 million on December 31, 2025, compared to $1,139.7 million on December 31, 2024. Shares issued and outstanding as of December 31, 2025, were 81,474,366. Full Year 2026 Financial Guidance Total product revenue of $700 to $800 million, representing a 19 to 36% increase from 2025, with the majority from Sephience GAAP R&D and SG&A expense of $775 to $815 million Non-GAAP R&D and SG&A expense of $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the call by phone, please click here to register and you will be provided with dial-in details. The webcast conference call can be accessed on the Investors section of the PTC website at . A replay of the call will be available after completion of the call and will be archived on the company's website. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Instagram and Facebook. For more Information Investors Ellen Cavaleri +1 (615) 618-6228 [email protected] Media Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "Full Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the U.S. on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

Financial StatementPhase 3License out/inDrug ApprovalNDA

16 Feb 2026

·www.fiercepharma.com

PTC shuts down FDA approval bid for troubled Duchenne med Translarna

With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna. Late Thursday, PTC revealed that it withdrew its FDA application for Translarna in nonsense mutation DMD after receiving feedback on its filing from the regulator. "FDA shared that based on its review to date, the data in the [New Drug Application] submission are unlikely to meet the Agency's threshold of substantial evidence of effectiveness to support approval of Translarna," Matthew Klein, M.D., PTC's chief executive, explained in a statement. PTC is "disappointed that FDA approval cannot be achieved," Klein said, citing the decades of development work that have gone into the company's protein restoration therapy. In a letter to the U.S. DMD community, PTC cited unresolvable "differences in data interpretation" with the FDA, Pharmaphorum reports. Translarna has a checkered past with the FDA and, despite scoring a thumbs-up in Europe back in 2014, recently met a major marketing setback there, as well. Stateside, the FDA initially declined to review PTC's Translarna application in 2016, telling the company its application was incomplete. About a year and a half later, the FDA formally rejected the drug, citing the need for PTC to run an additional trial "at a minimum" to better validate Translarna's effectiveness. The FDA also flagged other "nonclinical and [chemistry, manufacturing and controls] matters" in the approval bid. PTC announced it would make another attempt at U.S. approval in early 2024, pointing to "recent feedback" from the FDA. Now, that effort is ending without success. The drug has also suffered recent regulatory setbacks in the EU and, despite some pushback from European officials, the European Medicines Agency's human medicines committee ultimately decided not to renew the drug's conditional marketing authorization in late 2024. The European Commission, which has final say on approvals in the bloc, made the same call last March. European regulators blessed the med with a conditional nod in 2014 on results from a phase 2b study that favored Translarna over placebo on a six-minute walk test after 48 weeks of treatment. But that study and a subsequent trial did not produce statistically significant results on the metric. Back in the U.S., PTC also faced FDA friction around its Friedreich's ataxia candidate vatiquinone last year when the regulator denied approval of the candidate over a lack of "substantial evidence of efficacy" in the company's filing. Translarna, which goes by the generic moniker ataluren, is a protein restoration therapy that is designed to allow the formation of a functioning protein in patients with genetic disorders caused by nonsense mutations. PTC describes that type of mutation as an alteration in the genetic code that prematurely halts the synthesis of an essential protein. In the case of DMD, that protein would be dystrophin. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultDrug Approval

100 Deals associated with Ataluren

External Link

KEGG	Wiki	ATC	Drug Bank
D09323	Ataluren	M09AX03	DB05016

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	European Union	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	Iceland	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	Liechtenstein	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	Norway	PTC Therapeutics International Ltd.	31 Jul 2014

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cystic Fibrosis	NDA/BLA	European Union	PTC Therapeutics, Inc.	-
Becker Muscular Dystrophy	Phase 3	Australia	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Belgium	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Canada	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	France	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Germany	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Israel	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Italy	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Spain	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Sweden	PTC Therapeutics, Inc.	20 May 2012

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03179631 (Study 041, CTgov)	Phase 3	Muscular Dystrophy, Duchenne	360	Ataluren (Ataluren)	ybmlujnwvk(hpipfsegwc) = aworfojatt uevhbuaydb (ixewnfbawp, 6.461) View more	-	10 Mar 2026
NCT03179631 (Study 041, CTgov)	Phase 3	Muscular Dystrophy, Duchenne	360	PLACEBO (Placebo)	ybmlujnwvk(hpipfsegwc) = jbegmexfhz uevhbuaydb (ixewnfbawp, 6.377) View more	-	10 Mar 2026
NCT03179631 \| NCT01826487 \| NCT00592553 (Study 041 \| Study 007 \| ACT DMD, AAN2024)	Phase 2/3	Muscular Dystrophy, Duchenne	-	Ataluren	sumpkhmqjw(haaxbclbzv) = xrnqzpqbks tdugkwlbpv (gvdugoehei ) View more	Positive	09 Apr 2024
NCT04336826 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	pemqbdngoq = kocvnenrrq ndbwdrifly (yhbueemyqm, fxurrohhmb - ffpjfgmcsi) View more	-	01 Apr 2024
NCT03179631 (Study 041, AAN2023)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation (nm) DMD	-	Ataluren	mtdwerbhmp(nfdlkerimv) = crmxdynkco xprgjkeckp (pfjqvehayf ) View more	Positive	25 Apr 2023
NCT03179631 (Study 041, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	-	Ataluren	duszsoobru(erwhobbrto) = kiilskksjb icfxrjhwbw (eerjxmqcek ) View more	Positive	19 Mar 2023
NCT03179631 (Study 041, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	701	Ataluren	abgvbhnvyy(iiilbhemux) = digoqlttzu ldgtlfhoop (bjnzvitspa ) View more	Positive	19 Mar 2023
NCT00803205 \| NCT02139306 \| NCT02107859 (Pubmed \| Cochrane Database Syst Rev)	Not Applicable	Cystic Fibrosis	517	Ataluren and similar compounds	kmhwklvtca(xvuoixwzxl) = Ataluren was associated with a higher rate of episodes of renal impairment (risk ratio 12.81, 95% confidence interval 2.46 to 66.65; P = 0.002; I2 = 0%; 2 trials, 517 participants) rkgsamundr (ukgqkdawmy ) View more	-	03 Mar 2023
	Not Applicable	Cystic Fibrosis	517	Placebo		-	03 Mar 2023
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Ataluren (Ataluren)	rsrixpzahe(licsknbtum) = gssbvblsyt vpzwvrhrty (vfxagjusac, 7.425) View more	-	27 May 2022
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Placebo+Ataluren (Placebo)	rsrixpzahe(licsknbtum) = ojtmwjvema vpzwvrhrty (vfxagjusac, 11.809) View more	-	27 May 2022
NCT03796637 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	mquiugvhrn(gmzoptstfk) = loxgroovxb eremioervk (irxpxlxtws, 0.05874) View more	-	05 Apr 2022
NCT03648827 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	20	Ataluren	tpzlcetcvu(gszkrebasu) = gaxpylpwhm ditbujjutz (imisjbinto, ixozwqrpmb - qujtkopvxv) View more	-	05 Apr 2022

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