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

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PTC Therapeutics (NASDAQ: PTCT) announced that the European Commission (EC) has adopted the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) opinion to not renew the authorization of Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy.

This decision effectively removes the drug's conditional marketing authorization in the European Economic Area. However, the EC indicated that individual EU countries can use Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued Translarna use. PTC Therapeutics plans to work on a country-by-country basis to provide commercial drug where possible.

PTC Therapeutics (NASDAQ: PTCT) ha annunciato che la Commissione Europea (CE) ha adottato il parere del Comitato per i Medicinali per Uso Umano (CHMP) dell'Agenzia Europea dei Medicinali, di non rinnovare l'autorizzazione per Translarna™ (ataluren) per il trattamento della distrofia muscolare di Duchenne causata da mutazioni senza senso.

Questa decisione rimuove di fatto l'autorizzazione all'immissione in commercio condizionata del farmaco nell'Area Economica Europea. Tuttavia, la CE ha indicato che i singoli paesi dell'UE possono utilizzare gli Articoli 117(3) e 5(1) della Direttiva UE 2001/83 per consentire l'uso continuato di Translarna. PTC Therapeutics prevede di lavorare su base nazionale per fornire il farmaco commerciale dove possibile.

PTC Therapeutics (NASDAQ: PTCT) anunció que la Comisión Europea (CE) ha adoptado la opinión del Comité de Medicamentos para Uso Humano (CHMP) de la Agencia Europea de Medicamentos de no renovar la autorización de Translarna™ (ataluren) para el tratamiento de la distrofia muscular de Duchenne por mutaciones sin sentido.

Esta decisión elimina efectivamente la autorización condicional de comercialización del medicamento en el Espacio Económico Europeo. Sin embargo, la CE indicó que los países individuales de la UE pueden utilizar los Artículos 117(3) y 5(1) de la Directiva UE 2001/83 para permitir el uso continuado de Translarna. PTC Therapeutics planea trabajar país por país para proporcionar el medicamento comercial donde sea posible.

PTC Therapeutics (NASDAQ: PTCT)는 유럽연합 집행위원회(EC)가 유럽 의약품청(EMA)의 의약품 인체 사용 위원회(CHMP)가 Translarna™ (ataluren)의 허가 갱신을 하지 않겠다는 의견을 채택했다고 발표했습니다.

이 결정은 유럽 경제 지역 내에서 약물의 조건부 마케팅 승인을 사실상 제거합니다. 그러나 EC는 개별 EU 국가들이 EU 지침 2001/83의 제117(3)조 및 제5(1)조를 사용하여 Translarna의 지속적인 사용을 허용할 수 있다고 밝혔습니다. PTC Therapeutics는 가능한 경우 상업적 약물을 제공하기 위해 국가별로 작업할 계획입니다.

PTC Therapeutics (NASDAQ: PTCT) a annoncé que la Commission Européenne (CE) a adopté l'avis du Comité des Médicaments à Usage Humain (CHMP) de l'Agence Européenne des Médicaments de ne pas renouveler l'autorisation de Translarna™ (ataluren) pour le traitement de la dystrophie musculaire de Duchenne due à des mutations sans sens.

Cette décision supprime effectivement l'autorisation de mise sur le marché conditionnelle du médicament dans l'Espace Économique Européen. Cependant, la CE a indiqué que les pays de l'UE peuvent utiliser les Articles 117(3) et 5(1) de la Directive UE 2001/83 pour permettre l'utilisation continue de Translarna. PTC Therapeutics prévoit de travailler pays par pays pour fournir le médicament commercial lorsque cela est possible.

PTC Therapeutics (NASDAQ: PTCT) gab bekannt, dass die Europäische Kommission (EK) die Stellungnahme des Ausschusses für Humanarzneimittel (CHMP) der Europäischen Arzneimittel-Agentur angenommen hat, die Genehmigung von Translarna™ (ataluren) zur Behandlung der Duchenne-Muskeldystrophie aufgrund von Stop-Mutationen nicht zu verlängern.

Diese Entscheidung hebt effektiv die bedingte Marktzulassung des Medikaments im Europäischen Wirtschaftsraum auf. Die EK wies jedoch darauf hin, dass einzelne EU-Länder die Artikel 117(3) und 5(1) der EU-Richtlinie 2001/83 nutzen können, um die fortgesetzte Verwendung von Translarna zu ermöglichen. PTC Therapeutics plant, landesweise zu arbeiten, um das Medikament kommerziell anzubieten, wo dies möglich ist.

Positive
  • Individual EU countries can still allow Translarna use through specific regulatory mechanisms
  • Company can pursue country-specific approvals for commercial distribution
Negative
  • Loss of European-wide conditional marketing authorization for Translarna
  • Negative CHMP opinion adopted by European Commission
  • Potential reduction in European market access and sales

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 www.ptcbio.com and follow us on Facebook, X, and LinkedIn.

For More Information: 

Investors: 

Ellen Cavaleri
+1 (615) 618-6228
ecavaleri@ptcbio.com 

Media: 

Jeanine Clemente 
+1 (908) 912-9406 
jclemente@ptcbio.com  

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.

Cision View original content:https://www.prnewswire.com/news-releases/ptc-therapeutics-provides-regulatory-update-on-translarna-ataluren-in-europe-302414288.html

SOURCE PTC Therapeutics, Inc.

FAQ

What is the impact of EC's decision on PTC Therapeutics' Translarna in Europe?

The EC's decision removes Translarna's conditional marketing authorization in the European Economic Area, though individual EU countries can still allow its use through specific regulations.

Can Translarna still be sold in European countries after losing EC authorization?

Yes, individual EU countries can allow Translarna's use through Articles 117(3) and 5(1) of EU Directive 2001/83, requiring country-by-country approval.

What was the European Commission's decision on PTCT's Translarna in March 2025?

The EC adopted CHMP's negative opinion, deciding not to renew the authorization of Translarna for nonsense mutation Duchenne muscular dystrophy treatment.

How will PTCT continue Translarna distribution in Europe after the EC decision?

PTC Therapeutics will work on a country-by-country basis to provide commercial drug where possible through specific EU member state mechanisms.
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