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

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.

CTIS2024-518919-19-00

/ RecruitingPhase 2IIT

A single patient trial with Ataluren in one case of severe common variable immunodeficiency with autoimmunity due to homozygous stop codon mutations of LRBA - LRBA01

Start Date28 Jul 2021

Sponsor / Collaborator

Centre Hospitalier Universitaire de Liege

100 Clinical Results associated with Ataluren

100 Translational Medicine associated with Ataluren

100 Patents (Medical) associated with Ataluren

392

Literatures (Medical) associated with Ataluren

01 May 2025·Recenti progressi in medicina

[Administrative healthcare data to identify and describe patients with rare diseases: the case of Duchenne muscular dystrophy.]

Article

Author: Dondi, Leonardo ; Esposito, Immacolata ; Calabria, Silvia ; Marcellusi, Andrea ; Addesi, Alice ; Martini, Nello ; Ronconi, Giulia ; Dondi, Letizia ; Dell'Anno, Irene ; Bello, Luca ; Piccinni, Carlo

INTRODUCTION:

Duchenne muscular dystrophy (DMD) is a rare disease that causes a progressive loss of muscle function in males, presenting at the age of two years, and involving respiratory and heart function starting from teenage years. This retrospective observational study has identified patients potentially affected by DMD and described their utilization of healthcare resources and direct healthcare costs charged to the Italian National Healthcare Service (INHS).

METHODS:

From the Foundation Ricerca e Salute (ReS), through a specific algorithm based only on administrative healthcare data of 5.4 million inhabitants in 2021 (index date), male patients aged <30 years potentially affected by DMD were identified. Comorbidities at baseline, and utilization of healthcare resources and direct healthcare costs charged to the INHS during the year following index date, were described.

RESULTS:

In 2021, 120 male patients aged <30 years were identified as potentially affected with DMD (2.2/100,000 inhabitants; 16.1/100,000 males aged <30 years). Chronic airway disease and cardiomyopathy were found in 19.2% and 15.0% of patients, respectively. During follow-up: 41.7% of patients were treated with deflazacort, 2.5% with ataluren and about one third with cardiac drugs; 29.2% and 42.5% were admitted to overnight and day hospitalization, respectively, mainly due to neurological, cardiac, and respiratory diseases; 12.5% accessed the emergency department, mainly for traumatisms and fractures; 70.8% received local outpatient specialist care, half of which were specialist visits, and about 15% cardiac diagnostics. On average, the per capita annual total cost charged to the -INHS was € 6713; ataluren accounted for more than half of this expenditure. After having excluded the dispensation of ataluren during follow-up, the mean per capita total cost was € 2548, more than half of which due to hospitalizations.

CONCLUSIONS:

This study of administrative data has identified patients potentially affected by DMD, a rare disease, from a large sample of INHS beneficiaries, and assessed their healthcare pathway. This is useful for regulatory purposes and for improved access to emerging innovative therapies.

01 Apr 2025·ADVANCED SYNTHESIS & CATALYSIS

Photoredox Hydroacylative Dearomatization of Indoles with Aromatic Carboxylic Acids

Author: Zhu, Lin ; Zhang, Si‐Si ; Chen, Shan‐Shan ; Peng, Hong‐Bo ; Qu, Chuan‐Hua ; Song, Gui‐Ting

Abstract:

Herein we reported for the first time a photocatalytic dearomative hydroacylation reaction of electron‐deficient indoles with readily available aromatic acids through a phosphoranyl radical‐assisted deoxygenative process. A range of racemic and optically active 2‐acylindoline derivatives were obtained in good yields (up to 94%) with good diastereoselectivity. The synthetic robustness is highlighted by the enantioselective dearomatization of indoles and the late‐stage modification of natural products and pharmaceutical molecules. This protocol features broad substrate scope and mild reaction conditions, providing a reliable entry to developing deoxygenative strategies in dearomatization reactions. Preliminary mechanistic studies and DFT calculations suggest that a Giese‐type radical addition pathway should be involved.

20 Mar 2025·NUCLEIC ACIDS RESEARCH

Mechanism-based approach in designing patient-specific combination therapies for nonsense mutation diseases

Article

Author: Bhat, Saleem Y ; Cui, Xiaonan ; Bhattacharya, Arpan ; Li, Hong ; Cooperman, Barry S ; Goldman, Yale E ; Lueck, John D

Abstract:

Premature termination codon (PTC) diseases account for ∼12% of all human disease mutations. Although there are no FDA approved treatments for increasing PTC readthrough, one readthrough inducing drug, ataluren, has conditional approval for treatment of Duchenne muscular dystrophy elsewhere. Ataluren displays low toxicity in clinical trials for treatment of PTC diseases, but its therapeutic effects are inconsistent. The messenger RNA (mRNA) sequence context of a PTC is a major determinant of PTC readthrough efficiency. We have shown that ataluren stimulates readthrough exclusively by competitively inhibiting release factor complex (RFC) catalysis of translation termination. Here, using an in vitro reconstituted system, we demonstrate that PTC identity and the immediately adjacent mRNA sequence contexts modulate RFC activity in terminating peptide elongation. Such modulation largely determines the effectiveness of ataluren in stimulating readthrough, whether added alone or in combination with either the aminoglycoside G418 or an anticodon edited aa-tRNA, which stimulate readthrough by mechanisms orthogonal to that of ataluren. Our results suggest a potential rationale for the variability of ataluren effectiveness in stimulating readthrough. We hypothesize that patients harboring a PTC mutation within a sequence context promoting strong interaction with RFC will be resistant to ataluren, but that ataluren treatment will be more effective for patient sequences conferring weaker interaction with RFC.

News (Medical) associated with Ataluren

06 May 2025

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports First Quarter 2025 Financial Results

– Strong revenue performance of $190 million – – Positive CHMP opinion for Sephience™ (sepiapterin) received in April 2025, NDA review remains on track for July 29, 2025 PDUFA date – – Global Sephience launch activities progressing well – – Strong cash position of over $2.0 billion as of March 31, 2025 – WARREN, N.J., May 6, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2025. "Following a year of outstanding execution across every part of the Company, we have built on this positive momentum with solid revenue performance in the first quarter, allowing us to narrow our full-year revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "Our strong cash balance supports all of our planned commercial and R&D activities and provides the ability to reach cashflow breakeven without raising additional capital. The positive CHMP opinion for Sephience kickstarts our anticipated global launch for what we see as a significant revenue opportunity." Key Corporate Updates: First quarter 2025 total net product and royalty revenue of $190 million. First quarter 2025 revenue for the DMD franchise of $134 million, including net product revenue for Translarna™ of $86 million and for Emflaza® of $48 million. Key Clinical and Regulatory Milestones: Sephience Positive CHMP opinion on Sephience MAA for adult and pediatric PKU patients received on April 25, 2025; the opinion includes a broad label inclusive of all ages and disease severities. PTC expects the EC to adopt the opinion in approximately two months NDA currently under review by the FDA, with a target regulatory action date of July 29, 2025 Japan regulatory submission under review and a decision is expected in Q4 2025 Vatiquinone NDA for pediatric and adult patients with Friedreich's ataxia accepted and granted Priority Review by the FDA, with a target regulatory action date of August 19, 2025 Translarna NDA currently under review by the FDA PTC518 Phase 2 PIVOT-HD study results announced on May 5, 2025: Met primary endpoint of dose-dependent blood HTT lowering at Week 12 Dose-dependent trends of clinical benefit in Stage 2 patients at Month 12 Signals of dose-dependent clinical benefit relative to matched natural history cohort and dose-dependent lowering of NfL in Stage 2 subjects at Month 24 Continued favorable safety and tolerability with no treatment-related NfL spikes Plans to complete additional analyses and discuss next development and regulatory steps, including potential for accelerated approval First Quarter 2025 Financial Highlights: Total net product and royalty revenue was $189.9 million for the first quarter of 2025, compared to $208.8 million for the first quarter of 2024. Total revenue includes net product revenue across the commercial portfolio of $153.4 million for the first quarter of 2025, compared to $177.6 million for the first quarter of 2024. Total revenue also includes royalty, collaboration and license, and manufacturing revenue of $1,022.7 million for the first quarter of 2025, compared to $32.5 million for the first quarter of 2024. The first quarter of 2025 collaboration and license revenue includes $986.2 million, related to the PTC518 license and collaboration agreement with Novartis, which closed in January 2025. Translarna net product revenues were $86.2 million for the first quarter of 2025, compared to $103.6 million for the first quarter of 2024. Emflaza net product revenues were $47.8 million for the first quarter of 2025, compared to $57.5 million for the first quarter of 2024. Roche reported Evrysdi® full year 2025 sales of approximately 420 CHF million, resulting in royalty revenue of $36.4 million to PTC for first quarter 2025, as compared to $31.2 million for first quarter 2024. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $109.0 million for the first quarter of 2025, compared to $116.1 million for the first quarter of 2024. Non-GAAP R&D expenses were $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense, compared to $107.2 million for the first quarter of 2024, excluding $9.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $81.0 million for the first quarter of 2025, compared to $73.3 million for the first quarter of 2024. Non-GAAP SG&A expenses were $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $63.9 million for the first quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense. Net income was $866.6 million for the first quarter of 2025, compared to net loss of $91.6 million for the first quarter of 2024. Cash, cash equivalents, and marketable securities were $2,027.2 million as of March 31, 2025, compared to $1,139.7 million as of December 31, 2024. Shares issued and outstanding as of March 31, 2025 were 79,225,276. PTC Updates Full-Year 2025 Financial Guidance: PTC now anticipates full-year 2025 revenue to be between $650 million and $800 million, which includes in-line products, potential new product launches, and royalty revenue from Evrysdi. PTC anticipates full-year 2025 GAAP R&D and SG&A expense to be between $805 and $835 million. PTC anticipates full-year 2025 non-GAAP R&D and SG&A expense to be between $730 and $760 million, excluding estimated non-cash, stock-based compensation expense of $75 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 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) CHMP: Committee for Medicinal Products for Human Use DMD: Duchenne Muscular Dystrophy EC: European Commission FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's Disease HTT: Huntingtin protein MAA: Marketing Authorization Application NDA: New Drug Application NfL: Neurofilament light chain nmDMD: Nonsense mutation Duchenne muscular dystrophy PDUFA: Prescription Drug User Fee Act 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. To avoid delays, we recommend participants dial in for the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investors section of the PTC website at . A replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit and follow on Facebook, X, and LinkedIn. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [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 "PTC Updates Full Year 2025 Financial Guidance", including with respect to (i) 2025 total revenue guidance and (ii) 2025 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, 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," 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; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia and other regions; the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna on other regulatory bodies; PTC's ability to use the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis; 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 Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of regulatory and sales milestones and contingent payments that PTC may be obligated to make; 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 most recent 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 Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi, Waylivra, Sephience or vatiquinone. 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. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultFinancial StatementLicense out/inNDAPhase 2

28 Mar 2025

·pipelinereview.com

PTC Therapeutics Provides Regulatory Update on Translarna™ (ataluren) in Europe

WARREN, NJ, USA I March 28, 2025 I PTC Therapeutics, Inc. (NASDAQ: PTCT ) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy. While this action effectively removes the drug’s conditional marketing authorization in the European Economic Area, the EC indicated that individual countries within the European Union can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued use of Translarna. “We are of course disappointed that after this prolonged period of review the European Commission has decided to adopt the CHMP negative opinion on Translarna,” said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. “The EC’s indication that European Union member states have a mechanism to maintain treatment speaks to the safety, benefit and lack of alternative therapies for boys and young men with nonsense mutation Duchenne muscular dystrophy. We look forward to working on a country-by-country basis to provide commercial drug where possible.” About Translarna™ (ataluren) Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States. About Duchenne Muscular Dystrophy (Duchenne) Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20’s due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company’s strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, X, and LinkedIn. SOURCE: PTC Therapeutics

Drug Approval

28 Mar 2025

·www.prnewswire.com

PTC Therapeutics Provides Regulatory Update on Translarna™ (ataluren) in Europe

WARREN, N.J., March 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy. While this action effectively removes the drug's conditional marketing authorization in the European Economic Area, the EC indicated that individual countries within the European Union can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued use of Translarna. "We are of course disappointed that after this prolonged period of review the European Commission has decided to adopt the CHMP negative opinion on Translarna," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "The EC's indication that European Union member states have a mechanism to maintain treatment speaks to the safety, benefit and lack of alternative therapies for boys and young men with nonsense mutation Duchenne muscular dystrophy. We look forward to working on a country-by-country basis to provide commercial drug where possible." About Translarna™ (ataluren) Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States. About Duchenne Muscular Dystrophy (Duchenne) Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20's due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at and follow us on Facebook, X, and LinkedIn. For More Information: Investors: Ellen Cavaleri +1 (615) 618-6228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statement 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 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, commercialization and other matters with respect to its products and product candidates; the clinical utility and potential advantages of Translarna (ataluren); PTC's strategy, future operations, future financial position, future revenues, projected costs; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; 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," 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; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia and other regions; PTC's ability to identify other potential mechanisms by which it may provide Translarna to nmDMD patients in the EEA; whether healthcare professionals agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from PTC's trials in Translarna; 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; and the factors discussed in the "Risk Factors" section of PTC's most recent 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 Translarna. 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. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug 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 \| NCT01826487 \| NCT00592553 (Study 041 \| Study 007 \| ACT DMD, AAN2024)	Phase 2/3	Muscular Dystrophy, Duchenne	-	Ataluren	vmupbdxmyi(fcxqpzxwjl) = bfvtcwshzu sdpclnmdxq (ijlpuvvpns ) View more	Positive	09 Apr 2024
NCT04336826 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	oggntxizxm = agjauidqkv umwsierfcs (bscwbbuzro, nzpfbfjjxs - crweezquyq) View more	-	01 Apr 2024
NCT03179631 (Study 041, AAN2023)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation (nm) DMD	-	Ataluren	degekjjkeq(tjeljydemt) = megpxnmitu siewkjxvre (hweofngvwk ) View more	Positive	25 Apr 2023
NCT03179631 (Study 041, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	-	Ataluren	znqawjimtk(wfhggsikkj) = chzwrvsdyb egcbmifagc (irtgfwyfoz ) View more	Positive	19 Mar 2023
NCT03179631 (Study 041, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	701	Ataluren	qnmeekakyt(kwcgiszddp) = mejkejxyjm yalarcnuyp (zvjzivpuql ) View more	Positive	19 Mar 2023
NCT00803205 \| NCT02139306 \| NCT02107859 (Pubmed \| Cochrane Database Syst Rev)	Not Applicable	Cystic Fibrosis	517	Ataluren and similar compounds	swvuqvcovk(yqavyimnni) = 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) ibdbpdzwdr (xvbvmzmulj ) View more	-	03 Mar 2023
	Not Applicable	Cystic Fibrosis	517	Placebo		-	03 Mar 2023
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Ataluren (Ataluren)	cojylogevo(jzquxisrkd) = huzwgiagpl amvscslutk (ntjuztksbf, 7.425) View more	-	27 May 2022
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Placebo+Ataluren (Placebo)	cojylogevo(jzquxisrkd) = fvdfivpxee amvscslutk (ntjuztksbf, 11.809) View more	-	27 May 2022
NCT03796637 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	majadjtexf(sujtidizmf) = urccpfsrjq txllzskloj (vyywdxhlli, 0.05874) View more	-	05 Apr 2022
NCT03648827 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	20	Ataluren	izpobsqzkv(wywbozgpcq) = ohtiyawnbv wwhvkhlzxj (bgcrcsieuz, sbautqzsfo - enqjrqecdn) View more	-	05 Apr 2022
NCT01557400 (Study 019, MDA2021)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation	94	Ataluren 10, 10, 20 mg/kg	skidojtzth(izugfdmddc) = a trend in delay in decline to predicted FVC <50% by ~1 year jemormntim (kizxlytuzx )	Positive	01 Mar 2021
NCT01557400 (Study 019, MDA2021)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation	94	Ataluren		Positive	01 Mar 2021

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