CHMP Maintains Negative Opinion on Translarna™ Reexamination
PTC Therapeutics announced that the CHMP of the European Medicines Agency has maintained its negative opinion on renewing the conditional marketing authorization for Translarna™ (ataluren) for treating nonsense mutation Duchenne Muscular Dystrophy (nmDMD) after re-examination. The opinion will be reviewed by the European Commission, with a decision expected in about 67 days.
PTC's CEO, Matthew B. Klein, M.D., expressed disappointment, stating that the CHMP based its decision on the primary analysis subpopulation of Study 041 instead of considering the totality of evidence, including data from three placebo-controlled trials and the STRIDE registry. He emphasized that the decision goes against the wishes of physicians, patients, and families in Europe.
Translarna will remain authorized in Europe pending the EC review. PTC will continue to make Translarna available to patients and ensure the EC has all possible evidence to support continued authorization.
PTC Therapeutics ha annunciato che il CHMP dell'Agenzia Europea per i Medicinali ha mantenuto il suo parere negativo sul rinnovo dell'autorizzazione all'immissione in commercio condizionata per Translarna™ (ataluren) per il trattamento della distrofia muscolare di Duchenne (nmDMD) dopo una riesaminazione. Il parere sarà esaminato dalla Commissione Europea, con una decisione attesa in circa 67 giorni.
Il CEO di PTC, Matthew B. Klein, M.D., ha espresso delusione, affermando che il CHMP ha basato la sua decisione sull'analisi primaria della sottopopolazione dello Studio 041 invece di considerare la totalità delle prove, inclusi i dati di tre studi controllati con placebo e il registro STRIDE. Ha sottolineato che la decisione va contro i desideri dei medici, dei pazienti e delle famiglie in Europa.
Translarna rimarrà autorizzato in Europa in attesa della revisione della CE. PTC continuerà a rendere disponibile Translarna ai pazienti e garantirà che la CE abbia tutte le prove possibili per supportare l'autorizzazione continua.
PTC Therapeutics anunció que el CHMP de la Agencia Europea de Medicamentos ha mantenido su opinión negativa sobre la renovación de la autorización de comercialización condicional para Translarna™ (ataluren) para el tratamiento de la distrofia muscular de Duchenne por mutación sin sentido (nmDMD) tras una re-evaluación. La opinión será revisada por la Comisión Europea, con una decisión esperada en aproximadamente 67 días.
El CEO de PTC, Matthew B. Klein, M.D., expresó decepción, afirmando que el CHMP basó su decisión en el análisis primario de una subpoblación del Estudio 041 en lugar de considerar la totalidad de la evidencia, incluidos los datos de tres ensayos controlados con placebo y el registro STRIDE. Enfatizó que la decisión va en contra de los deseos de médicos, pacientes y familias en Europa.
Translarna seguirá autorizado en Europa mientras se espera la revisión de la CE. PTC continuará poniendo a disposición Translarna para los pacientes y asegurará que la CE disponga de toda la evidencia posible para respaldar la autorización continua.
PTC 테라퓨틱스는 유럽의약청의 CHMP가 부정적인 의견을 유지했다고 발표했습니다. 이는 Translarna™ (ataluren)의 조건부 마케팅 승인 갱신, 즉 의사소통 돌연변이형 듀셴 근육 이영양증(nmDMD) 치료에 관한 것입니다. 이 의견은 유럽연합 집행위원회에 의해 검토될 예정이며, 결정은 약 67일 후에 예상됩니다.
PTC의 CEO인 매튜 B. 클라인(M.D.)은 실망감을 표명하며, CHMP가 증거의 전체성을 고려하는 대신 연구 041의 주요 분석 하위 집단에 기반하여 결정을 내렸다고 지적했습니다. 그는 이 결정이 유럽의 의사, 환자와 가족의 희망과 반대된다고 강조했습니다.
Translarna는 EC 검토 중 여전히 유럽에서 허가를 유지합니다. PTC는 환자들에게 Translarna를 계속 제공하고 EC가 지속적인 승인을 지원할 수 있도록 모든 가능한 증거를 확보할 것입니다.
PTC Therapeutics a annoncé que le CHMP de l'Agence Européenne des Médicaments a maintenu son avis négatif concernant le renouvellement de l'autorisation de mise sur le marché conditionnelle de Translarna™ (ataluren) pour le traitement de la dystrophie musculaire de Duchenne par mutation sans sens (nmDMD) après un nouvel examen. L'avis sera examiné par la Commission Européenne, avec une décision attendue dans environ 67 jours.
Le PDG de PTC, Matthew B. Klein, M.D., a exprimé sa déception, déclarant que le CHMP a fondé sa décision sur l'analyse principale de la sous-population de l'Étude 041 plutôt que de tenir compte de la totalité des preuves, y compris des données de trois essais contrôlés par placebo et du registre STRIDE. Il a souligné que cette décision va à l'encontre des souhaits des médecins, des patients et des familles en Europe.
Translarna restera autorisé en Europe en attendant l'examen de la CE. PTC continuera de rendre Translarna disponible pour les patients et veillera à ce que la CE dispose de toutes les preuves possibles pour soutenir l'autorisation continue.
PTC Therapeutics hat bekannt gegeben, dass das CHMP der Europäischen Arzneimittelagentur seine negative Stellungnahme aufrechterhalten hat bezüglich der Erneuerung der bedingten Marktzulassung für Translarna™ (ataluren) zur Behandlung von Duchenne-Muskeldystrophie (nmDMD) nach einer erneuten Prüfung. Die Stellungnahme wird von der Europäischen Kommission überprüft, eine Entscheidung wird in etwa 67 Tagen erwartet.
Der CEO von PTC, Matthew B. Klein, M.D., äußerte Enttäuschung und erklärte, dass das CHMP seine Entscheidung auf die primäre Analyse der Untergruppe der Studie 041 gestützt hat, anstatt die gesamte Evidenz zu berücksichtigen, einschließlich Daten aus drei placebokontrollierten Studien und dem STRIDE-Register. Er betonte, dass die Entscheidung gegen die Wünsche von Ärzten, Patienten und Familien in Europa verstößt.
Translarna wird solange in Europa autorisiert bleiben, bis die Prüfung durch die CE erfolgt ist. PTC wird weiterhin Translarna für Patienten verfügbar machen und sicherstellen, dass die CE über alle möglichen Beweise für die Unterstützung der fortgesetzten Genehmigung verfügt.
- Translarna remains authorized in Europe pending EC review
- PTC will continue to make Translarna available to patients
- Evidence from three placebo-controlled trials and STRIDE registry supports Translarna's efficacy and safety
- CHMP maintained negative opinion on Translarna's marketing authorization renewal
- Potential risk of losing European market authorization for Translarna
- CHMP decision based on data instead of considering all available evidence
Insights
The CHMP's maintained negative opinion on Translarna™ (ataluren) is a significant setback for PTC Therapeutics. This decision challenges the renewal of conditional marketing authorization for treating nonsense mutation Duchenne Muscular Dystrophy (nmDMD) in Europe. The European Commission's upcoming review adds uncertainty to Translarna's future in this market.
Key points to consider:
- The CHMP focused on Study 041's primary analysis subpopulation, disregarding other evidence presented by PTC.
- PTC argues that data from three placebo-controlled trials and the STRIDE registry support Translarna's efficacy and safety.
- The drug remains authorized pending EC review, expected in about 67 days.
- This decision contradicts the preferences of European physicians, patients and families.
For investors, this creates significant uncertainty around Translarna's future revenue potential in Europe, a important market for PTC. The outcome could impact PTC's financial performance and market position in rare disease treatments.
This negative opinion from CHMP poses a substantial risk to PTC Therapeutics' revenue stream and market position. Translarna is a key product for PTC and losing European market authorization would significantly impact the company's financial outlook. Key financial considerations include:
- Potential loss of European market revenue if authorization is not renewed
- Increased uncertainty may lead to stock price volatility
- Possible need for increased R&D spending to address regulatory concerns
- Impact on investor confidence and future capital raising abilities
While the drug remains authorized pending EC review, the maintained negative opinion suggests a challenging path forward. Investors should closely monitor the EC's decision in the coming months, as it will be important in determining PTC's near-term financial trajectory and long-term growth prospects in the European rare disease market.
- Opinion to be reviewed by European Commission -
"CHMP again based its decision on the results of the primary analysis subpopulation of Study 041 instead of the totality of evidence for Translarna. That evidence includes data across three placebo-controlled trials and our STRIDE registry that demonstrates consistent short and long-term efficacy as well as safety supportive of continued authorization. In addition, the CHMP opinion is clearly against the expressed wishes of physicians, patients and families throughout
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
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 innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on X, Facebook, Instagram and LinkedIn.
For More Information:
Investors:
Investor Relations
+1 (908) 912-9848
ir@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; PTC's plans for interactions with the European Medicines Agency (EMA); the European Commission's potential adoption of the CHMP's negative opinion for Translarna (ataluren); 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
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.
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SOURCE PTC Therapeutics, Inc.
FAQ
What was the CHMP's decision on Translarna for nmDMD treatment?
When is the European Commission expected to decide on the CHMP opinion for PTCT's Translarna?
Will Translarna remain available for patients in Europe despite the CHMP's negative opinion?
What evidence does PTC Therapeutics (PTCT) cite to support Translarna's continued authorization?
