PTC Therapeutics Announces FDA Acceptance and Priority Review for Vatiquinone NDA for the Treatment of Children and Adults with Friedreich's Ataxia
PTC Therapeutics (NASDAQ: PTCT) announced FDA acceptance and Priority Review for its New Drug Application (NDA) for vatiquinone, targeting the treatment of children and adults with Friedreich's ataxia (FA). The FDA has set a PDUFA target action date of August 19, 2025.
The application is supported by data from the placebo-controlled MOVE-FA study and two long-term studies involving pediatric and adult FA patients. These studies demonstrated significant evidence of slowing disease progression and confirmed vatiquinone's safety and tolerability across all age groups.
If approved, vatiquinone would become the first therapy available for pediatric FA patients while offering an alternative treatment option for adults. This marks PTC's fourth FDA application acceptance in 2024.
PTC Therapeutics (NASDAQ: PTCT) ha annunciato l'accettazione da parte della FDA e la Revisione Prioritaria per la sua Domanda di Nuovo Farmaco (NDA) per vatiquinone, mirata al trattamento di bambini e adulti affetti da atassia di Friedreich (FA). La FDA ha fissato una data obiettivo per l'azione PDUFA del 19 agosto 2025.
La domanda è supportata da dati dello studio MOVE-FA controllato con placebo e due studi a lungo termine che coinvolgono pazienti pediatrici e adulti con FA. Questi studi hanno dimostrato evidenze significative di rallentamento della progressione della malattia e hanno confermato la sicurezza e la tollerabilità di vatiquinone in tutti i gruppi di età.
Se approvato, vatiquinone diventerebbe la prima terapia disponibile per i pazienti pediatrici con FA, offrendo nel contempo un'opzione alternativa di trattamento per gli adulti. Questo segna la quarta accettazione della domanda da parte della FDA da parte di PTC nel 2024.
PTC Therapeutics (NASDAQ: PTCT) anunció la aceptación por parte de la FDA y la Revisión Prioritaria para su Solicitud de Nuevo Medicamento (NDA) para vatiquinona, dirigida al tratamiento de niños y adultos con ataxia de Friedreich (FA). La FDA ha establecido una fecha objetivo de acción PDUFA del 19 de agosto de 2025.
La solicitud está respaldada por datos del estudio MOVE-FA controlado con placebo y dos estudios a largo plazo que involucran a pacientes pediátricos y adultos con FA. Estos estudios demostraron evidencia significativa de ralentización de la progresión de la enfermedad y confirmaron la seguridad y tolerabilidad de vatiquinona en todos los grupos de edad.
Si se aprueba, vatiquinona se convertiría en la primera terapia disponible para pacientes pediátricos con FA, ofreciendo al mismo tiempo una opción alternativa de tratamiento para adultos. Este es el cuarto caso de aceptación de solicitud por parte de la FDA para PTC en 2024.
PTC Therapeutics (NASDAQ: PTCT)는 프리드리히 운동 실조증(FA) 치료를 목표로 하는 바티퀴논의 신약 신청(NDA)에 대한 FDA의 수용 및 우선 심사를 발표했습니다. FDA는 2025년 8월 19일을 PDUFA 목표 행동 날짜로 설정했습니다.
이 신청서는 위약 대조 MOVE-FA 연구와 소아 및 성인 FA 환자를 포함한 두 개의 장기 연구 데이터를 뒷받침하고 있습니다. 이러한 연구는 질병 진행을 늦추는 중요한 증거를 보여주었으며, 모든 연령대에서 바티퀴논의 안전성과 내약성을 확인했습니다.
승인될 경우, 바티퀴논은 소아 FA 환자를 위한 첫 번째 치료제가 되며, 성인에게는 대체 치료 옵션을 제공하게 됩니다. 이는 2024년 PTC가 FDA에 제출한 네 번째 신청 수용 사례입니다.
PTC Therapeutics (NASDAQ: PTCT) a annoncé l'acceptation par la FDA et l'examen prioritaire de sa demande de nouveau médicament (NDA) pour le vatiquinone, ciblant le traitement des enfants et des adultes atteints d'ataxie de Friedreich (FA). La FDA a fixé une date cible d'action PDUFA au 19 août 2025.
La demande est soutenue par des données de l'étude MOVE-FA contrôlée par placebo et de deux études à long terme impliquant des patients FA pédiatriques et adultes. Ces études ont montré des preuves significatives de ralentissement de la progression de la maladie et ont confirmé la sécurité et la tolérabilité du vatiquinone dans tous les groupes d'âge.
Si elle est approuvée, le vatiquinone deviendrait la première thérapie disponible pour les patients pédiatriques atteints de FA, tout en offrant une option de traitement alternative pour les adultes. Cela marque la quatrième acceptation de la demande par la FDA pour PTC en 2024.
PTC Therapeutics (NASDAQ: PTCT) gab die Annahme und die vorrangige Prüfung durch die FDA für seinen Antrag auf ein neues Medikament (NDA) für Vatiquinon bekannt, das auf die Behandlung von Kindern und Erwachsenen mit Friedreich-Ataxie (FA) abzielt. Die FDA hat einen Zieltermin für die PDUFA-Aktion auf den 19. August 2025 festgelegt.
Der Antrag wird durch Daten aus der placebokontrollierten MOVE-FA-Studie und zwei Langzeitstudien unterstützt, die pädiatrische und erwachsene FA-Patienten einbeziehen. Diese Studien zeigten signifikante Beweise für eine Verlangsamung des Krankheitsverlaufs und bestätigten die Sicherheit und Verträglichkeit von Vatiquinon in allen Altersgruppen.
Wenn es genehmigt wird, würde Vatiquinon die erste Therapie für pädiatrische FA-Patienten werden und gleichzeitig eine alternative Behandlungsoption für Erwachsene bieten. Dies markiert die vierte Annahme eines FDA-Antrags von PTC im Jahr 2024.
- FDA granted Priority Review status, potentially expediting the approval process
- Vatiquinone would be the first approved therapy for pediatric FA patients
- Clinical trials showed significant evidence of slowing disease progression
- All four of PTC's 2024 FDA applications have been accepted for review
- Final FDA approval still pending and not guaranteed
- Six-month wait until PDUFA date of August 19, 2025
Insights
The FDA's acceptance and Priority Review designation for vatiquinone represents a significant catalyst for PTC Therapeutics, particularly in the orphan drug space. Friedreich's ataxia, affecting approximately 1 in 50,000 people in the United States, represents a substantial market opportunity despite its rare disease status. The Priority Review status is especially noteworthy as it typically reduces the review period from 10 to 6 months, potentially accelerating market entry.
The strategic importance of this development is threefold: First, vatiquinone would be the pioneer therapy for pediatric FA patients, potentially establishing PTC as the standard of care in this previously underserved demographic. Second, the drug's demonstrated safety profile across age groups could facilitate broad adoption and sustained market presence. Third, the successful acceptance of all four FDA applications in 2024 demonstrates exceptional regulatory execution, significantly de-risking PTC's development pipeline.
From a commercial perspective, orphan drug pricing typically ranges from
The August 19 PDUFA date positions PTC for a potential Q4 2025 launch, assuming approval. This timing allows for adequate preparation of market access strategies and sales infrastructure. The broad age range coverage (both pediatric and adult patients) could enable market penetration of
- PDUFA target action date of Aug. 19, 2025 -
"We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich's ataxia," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "If approved, vatiquinone would be the first therapy for pediatric patients with FA, and provide a potential safe, well-tolerated and effective treatment alternative for adults. The granting of priority review by FDA reflects the significant unmet need for younger patients with FA. We look forward to working collaboratively with FDA during the review process."
The vatiquinone NDA is based on data from the placebo-controlled MOVE-FA study as well as results from two long-term studies including pediatric and adult FA patients. Data from these three studies demonstrate significant, durable and clinically meaningful evidence of slowing disease progression on key aspects of disease. In addition, these studies demonstrate that vatiquinone is safe and well tolerated in all age groups studied.
The vatiquinone NDA was the fourth approval application submitted to the FDA by PTC in 2024. All four applications were accepted for review.
About Vatiquinone
Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich's ataxia. Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately decreasing cellular inflammation and oxidative stress and promoting neuronal survival.1,2,3 Vatiquinone has been evaluated in a number of clinical studies, many focused on pediatric patients, and has demonstrated an impact on mortality risk, fatigue, and a number of neurological and neuromuscular disease symptoms.
About MOVE-FA
MOVE-FA was a global registration-directed trial of vatiquinone that enrolled 146 pediatric, adolescent and adult FA patients, the majority of whom were under 18 years of age. While the primary endpoint of change from baseline in the overall mFARS score did not reach statistical significance (p=0.14), a statistically significant effect (p=0.021) was recorded on the mFARS upright stability subscale, which was a pre-specified endpoint, and the portion of the mFARS now understood to be the most sensitive and relevant for the enrolled primary analysis population. In addition, the effect on upright stability was concordant with favorable treatment effect on the 1-minute walk distance test and the functional component of the Modified Fatigue Rating Scale. The study included a 72-week placebo-controlled phase and a long-term open-label extension. Following completion of MOVE-FA, subjects were eligible to enroll in a long-term, open-label extension study which is ongoing.
About Friedreich's Ataxia
Friedreich's ataxia (FA) is a rare, physically debilitating, life-shortening, neuromuscular disorder that mainly affects the central nervous system and the heart.4 It is the most common hereditary ataxia (abnormal, uncoordinated movements) and is usually caused by a single genetic defect in the frataxin (FXN) gene that leads to reduced production of frataxin, a mitochondrial protein that is important for cellular metabolism and energy production.4,5 Decreased frataxin levels are associated with mitochondrial iron accumulation and increased oxidative stress, which can lead to cell death through ferroptosis.6,7,8
Symptoms include progressive loss of coordination and muscle strength leading to poor balance and coordination, difficulty speaking, swallowing, and breathing, curvature of the spine, serious heart conditions, diabetes, and hearing and vision impairment.9,10 The severity of symptoms and speed of progression varies between people and some symptoms may not be evident in all. Friedreich's ataxia is usually diagnosed in childhood or adolescence.5,11 Approximately 25,000 people have Friedreich's ataxia globally.
About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company that discovers, develops, and commercializes 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. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, Instagram, LinkedIn, and X.
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 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; expectations with respect to vatiquinone, including any regulatory submissions and potential approvals, commercialization, the potential achievement of regulatory and sales milestones and contingent payments that PTC may be obligated to make; 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 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.
References:
- Hinman A, et al. PLoS one. 2018;13:e0201369.
- PTC Therapeutics. EPI-743 Pre-Clinical Data Deck.
- Shrader WD, et al. Bioorg Med Chem Lett. 2011;21:3693–3698.
- Lynch DR, Farmer JM, Balcer LJ, et al. Arch Neurol 2002;59(5):743–747.
- Campuzano V, Montermini L, Lutz Y, et al. Hum Mol Genet 1997;11(6):1771–1780.
- Campuzano V, et al. Hum Mol Genet. 1997;6:1771–1780.
- Cook A, Giunti P. Br Med Bull. 2017;124:19–30.
- Pandolfo M, Hausmann L. J Neurochem. 2013;126:142–146.
- Bürk K. Cerebellum Ataxias 2017;4:4.
- Cook A, Giunti P. Br Med Bull 2017;124(1):19–30.
- Delatycki MB, Williamson R, Forrest SM. J Med Genet 2000;37(1):1–8.
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