PTC Therapeutics Announces FDA Acceptance of Translarna™ NDA Resubmission
PTC Therapeutics announced FDA acceptance of its New Drug Application (NDA) resubmission for Translarna™ to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). The resubmission is supported by Study 041 results showing significant benefits in multiple endpoints after 72 weeks of treatment, including improved six-minute walk distance and other mobility measures. The STRIDE registry data demonstrated a 3.5-year delay in loss of ambulation and 1.8-year delay in reaching critical lung function threshold. As this is a resubmission following a previous complete response letter, no FDA action date has been provided.
PTC Therapeutics ha annunciato l'accettazione da parte della FDA della sua nuova richiesta di autorizzazione per il farmaco (NDA) relativa a Translarna™ per il trattamento della distrofia muscolare di Duchenne con mutazione nonsense (nmDMD). La ri-sottomissione è supportata dai risultati dello Studio 041, che mostrano benefici significativi in multiple variabili dopo 72 settimane di trattamento, inclusi miglioramenti nella distanza percorsa in sei minuti e in altre misure di mobilità. I dati del registro STRIDE hanno dimostrato un ritardo di 3,5 anni nella perdita dell'abilità ambulatoriale e un ritardo di 1,8 anni nel raggiungimento della soglia critica di funzione polmonare. Essendo una ri-sottomissione in risposta a una lettera di risposta completa precedente, non è stata fornita una data di azione da parte della FDA.
PTC Therapeutics anunció la aceptación por parte de la FDA de su reenvío de la Solicitud de Nuevo Medicamento (NDA) para Translarna™, para tratar la distrofia muscular de Duchenne por mutación sin sentido (nmDMD). El reenvío está respaldado por los resultados del Estudio 041, que muestran beneficios significativos en múltiples objetivos después de 72 semanas de tratamiento, incluida la mejora en la distancia recorrida en seis minutos y otras medidas de movilidad. Los datos del registro STRIDE demostraron un retraso de 3.5 años en la pérdida de la capacidad de deambulación y un retraso de 1.8 años en alcanzar el umbral crítico de función pulmonar. Dado que se trata de un reenvío tras una carta de respuesta completa previa, no se ha proporcionado una fecha de acción por parte de la FDA.
PTC Therapeutics는 무의미 돌연변이 두셴 근이영양증(nmDMD) 치료를 위한 Translarna™에 대한 신규 의약품 신청(NDA) 재제출이 FDA에 의해 수락되었다고 발표했습니다. 재제출은 72주 치료 후 여러 평가 지표에서 유의미한 이점을 보여주는 041 연구의 결과로 뒷받침됩니다. 여기에는 6분 걷기 거리 개선 및 기타 이동성 측정이 포함됩니다. STRIDE 등록부 데이터는 보행 능력 상실의 지연이 3.5년, 중요한 폐기능 기준 도달의 지연이 1.8년임을 보여주었습니다. 이는 이전에 완전 응답 편지가 있었던 재제출이므로, FDA의 조치 날짜는 제공되지 않았습니다.
PTC Therapeutics a annoncé l'acceptation par la FDA de sa nouvelle demande de médicament (NDA) relative à Translarna™ pour traiter la dystrophie musculaire de Duchenne avec mutation sans sens (nmDMD). La réévaluation est soutenue par les résultats de l'Étude 041 montrant des bénéfices significatifs sur plusieurs indicateurs après 72 semaines de traitement, y compris une amélioration de la distance parcourue en six minutes et d'autres mesures de mobilité. Les données du registre STRIDE ont démontré un retard de 3,5 ans dans la perte de l'aptitude à marcher et un retard de 1,8 an dans l'atteinte du seuil critique de fonction pulmonaire. Étant donné qu'il s'agit d'une réévaluation suite à une lettre de réponse complète précédente, aucune date d'action de la FDA n'a été fournie.
PTC Therapeutics gab die Annahme seines Neuen Arzneimittelantrags (NDA) durch die FDA für Translarna™ zur Behandlung der Dystrophie musculorum Duchenne mit Nonsense-Mutation (nmDMD) bekannt. Die erneute Einreichung wird durch die Ergebnisse der Studie 041 unterstützt, die signifikante Vorteile in mehreren Endpunkten nach 72 Wochen Behandlung zeigt, einschließlich der Verbesserung der Gehdistanz in sechs Minuten und anderer Mobilitätsmessungen. Die Daten aus dem STRIDE-Register zeigten eine Verzögerung von 3,5 Jahren beim Verlust der Gehfähigkeit und eine Verzögerung von 1,8 Jahren beim Erreichen der kritischen Lungenfunktion. Da es sich um eine erneute Einreichung nach einem vorherigen vollständigen Antwortschreiben handelt, wurde kein Datum für eine Maßnahme der FDA angegeben.
- Study 041 demonstrated statistically significant benefits across multiple mobility endpoints
- STRIDE registry showed 3.5-year delay in loss of ambulation (p<0.0001)
- Data showed 1.8-year delay in reaching critical lung function threshold (p=0.0028)
- Treatment targets approximately 13% of Duchenne muscular dystrophy patients
- No PDUFA date set due to previous complete response letter
- Previous NDA rejection in 2016 indicates potential regulatory challenges
- to specific mutation type, restricting market size
Insights
The FDA's acceptance of Translarna's NDA resubmission represents a critical regulatory milestone for PTC Therapeutics. The application is supported by robust clinical data from Study 041, showing statistically significant improvements across multiple functional endpoints. The study demonstrated benefits in six-minute walk distance, NorthStar Ambulatory Assessment and other key mobility measures after 72 weeks of treatment.
Particularly compelling is the STRIDE registry data showing a 3.5-year delay in loss of ambulation and 1.8-year delay in respiratory decline. These long-term benefits are especially meaningful for nmDMD patients, who represent approximately
This regulatory advancement could significantly impact PTC's market position in the U.S. DMD space. Translarna is already commercialized in Europe and other regions, making U.S. approval a important expansion opportunity. The addressable market is notable, considering nmDMD affects about
However, investors should consider the historical context - the previous NDA was filed under protest in 2016, suggesting potential regulatory hurdles. The lack of a standard PDUFA timeline adds uncertainty to the approval process and potential commercialization timeline.
"The NDA acceptance for review is a significant milestone that brings us one step closer to providing this important treatment to boys and young men living with nonsense mutation Duchenne muscular dystrophy in
"We thank FDA for accepting the Translarna NDA for review," stated Pat Furlong, Founder and CEO, Parent Project Muscular Dystrophy. "Translarna uniquely addresses individuals with Duchenne muscular dystrophy secondary to nonsense mutation and could provide an important treatment option for our community. Families in
The NDA resubmission is based on the findings of significant benefit demonstrated in the ITT population (N=359) of the global placebo-controlled trial Study 041. Following 72-weeks of Translarna treatment, there was significant benefit demonstrated on the key study endpoints of six-minute walk distance (6MWD) (p=0.0248), NorthStar Ambulatory Assessment (p=0.0283), 10-meter walk/run (p=0.0422),
4-stair climb (p=0.0293), and time to
In addition, the NDA includes the findings of significant long-term Translarna treatment benefit as captured in the STRIDE registry. Translarna treatment resulted in a 3.5-year delay in loss of ambulation (p<0.0001) and a 1.8-year delay in reaching a predicted forced vital capacity of less than
"We are excited that the FDA has accepted the Translarna NDA for review," said Debra Miller, Founder and CEO of CureDuchenne. "We believe that the totality of the data demonstrates the meaningful benefits and strong safety profile of Translarna for people with Duchenne muscular dystrophy caused by a nonsense mutation, which is approximately
As this was an NDA resubmission following a complete response letter to the NDA which was filed over protest in 2016, FDA is not obligated to follow PDUFA review timelines. Thus, an action date has not been provided.
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. Study 041 was a global placebo-controlled trial that enrolled a broad population of nmDMD patients. The study included a primary analysis population of a subgroup of enrollees, which did not reach statistical significance on the primary endpoint of 6MWD. However, significant benefit was recorded across 6MWD and other key study endpoints in the overall enrolled (ITT) population.
About the STRIDE Registry
The STRIDE Registry is an ongoing, multicenter, observational study of the safety and effectiveness of Translarna in routine care. It is the first patient data repository to provide real-world experience regarding the long-term use of Translarna in routine clinical practice.
STRIDE is a collaborative partnership between TREAT-NMD and PTC Therapeutics, led by a Steering Committee comprised of leading experts in Duchenne, patient advocates from around the world and PTC representatives.
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 Facebook, Instagram, LinkedIn and Twitter at @PTCBio.
For More Information:
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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 regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; PTC's plans for interactions with the U.S. Food and Drug Administration (FDA); 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 were the key results from Study 041 for PTCT's Translarna?
When will the FDA make a decision on PTCT's Translarna NDA resubmission?
What percentage of Duchenne muscular dystrophy patients could benefit from PTCT's Translarna?
