PTC Therapeutics Announces FDA Target Regulatory Action Date for Sepiapterin and Lancet Publication of Phase 3 Trial Results
PTC Therapeutics announced that the FDA has set a target regulatory action date of July 29, 2025, for the review of the New Drug Application (NDA) for sepiapterin. The NDA was submitted for the treatment of phenylketonuria (PKU) in pediatric and adult patients, covering all age groups and disease subtypes. Additionally, the results of the Phase 3 APHENITY trial were published in The Lancet.
The NDA includes data from the APHENITY trial and the ongoing open-label extension study, which shows the durability of sepiapterin's effect and patients' ability to liberalize their diet while maintaining phenylalanine (Phe) control. Recent results indicate that approximately 60% of subjects achieve protein intake above the age-adjusted recommended daily allowance for an unaffected individual while maintaining Phe levels < 360 μmol/L.
PTC Therapeutics ha annunciato che la FDA ha fissato una data target per l'azione regolatoria del 29 luglio 2025 per la revisione della Domanda di Nuovo Farmaco (NDA) per sepiapterin. La NDA è stata presentata per il trattamento della fenilchetonuria (PKU) nei pazienti pediatrici e adulti, coprendo tutti i gruppi di età e sottotipologie della malattia. Inoltre, i risultati del trial di Fase 3 APHENITY sono stati pubblicati su The Lancet.
La NDA include dati provenienti dal trial APHENITY e dallo studio di estensione in aperto in corso, che dimostrano la durata dell'effetto di sepiapterin e la capacità dei pazienti di liberalizzare la loro dieta mantenendo il controllo della fenilalanina (Phe). I risultati recenti indicano che circa il 60% dei soggetti raggiunge un'assunzione di proteine superiore al fabbisogno giornaliero raccomandato, corretto per l'età, per un individuo non colpito, mantenendo i livelli di Phe < 360 μmol/L.
PTC Therapeutics anunció que la FDA ha establecido una fecha objetivo de acción regulatoria del 29 de julio de 2025 para la revisión de la Solicitud de Nuevo Medicamento (NDA) para sepiapterin. La NDA se presentó para el tratamiento de fenilcetonuria (PKU) en pacientes pediátricos y adultos, abarcando todos los grupos de edad y subtipos de la enfermedad. Además, se publicaron los resultados del ensayo de Fase 3 APHENITY en The Lancet.
La NDA incluye datos del ensayo APHENITY y del estudio de extensión en abierto en curso, que muestran la durabilidad del efecto de sepiapterin y la capacidad de los pacientes para liberalizar su dieta mientras mantienen el control de la fenilalanina (Phe). Los resultados recientes indican que aproximadamente el 60% de los sujetos logra un consumo de proteínas superior a la ingesta diaria recomendada ajustada por edad para un individuo no afectado mientras mantienen niveles de Phe < 360 μmol/L.
PTC 테라퓨틱스는 FDA가 세피아프테린의 신규 의약품 신청(NDA) 심사를 위한 목표 규제 조치 일자를 2025년 7월 29일로 설정했다고 발표했습니다. NDA는 페닐케톤뇨증(PKU) 치료를 위해 소아 및 성인 환자를 대상으로 제출되었으며, 모든 연령대와 질병 하위 유형을 포함합니다. 또한, 3상 APHENITY 시험의 결과가 The Lancet에 발표되었습니다.
NDA에는 APHENITY 시험과 현재 진행 중인 공개 연장 연구에서 얻은 데이터가 포함되어 있으며, 세피아프테린의 효과 지속성과 환자들이 페닐알라닌(Phe) 수치를 유지하면서 식단을 자유롭게 조절할 수 있는 능력을 보여줍니다. 최근 결과에 따르면 약 60%의 피험자가 영향을 받지 않은 개인을 위한 연령 조정 권장 일일 섭취량 이상으로 단백질 섭취량을 달성하면서 Phe 수치를 < 360 μmol/L로 유지합니다.
PTC Therapeutics a annoncé que la FDA a fixé une date cible d'action réglementaire du 29 juillet 2025 pour l'examen de la Demande de Nouveau Médicament (NDA) pour le sepiapterin. La NDA a été soumise pour le traitement de la phénylcétonurie (PKU) chez des patients pédiatriques et adultes, couvrant tous les groupes d'âge et sous-types de maladies. De plus, les résultats de l'
La NDA inclut des données de l'essai APHENITY ainsi que de l'étude d'extension ouverte en cours, qui montrent la durabilité de l'effet du sepiapterin et la capacité des patients à libéraliser leur régime alimentaire tout en maintenant le contrôle de la phénylalanine (Phe). Des résultats récents indiquent que environ 60 % des sujets atteignent une consommation de protéines supérieure à l'apport journalier recommandé ajusté selon l'âge pour un individu non touché tout en maintenant des niveaux de Phe < 360 μmol/L.
PTC Therapeutics gab bekannt, dass die FDA einen Zieltermin für die regulatorische Maßnahme auf den 29. Juli 2025 festgelegt hat, um den Antrag auf Zulassung eines neuen Arzneimittels (NDA) für Sepiapterin zu überprüfen. Der NDA wurde zur Behandlung von Phenylketonurie (PKU) bei pädiatrischen und erwachsenen Patienten eingereicht, was alle Altersgruppen und Krankheitsuntertypen abdeckt. Darüber hinaus wurden die Ergebnisse der Phase-3-APHENITY-Studie im The Lancet veröffentlicht.
Der NDA enthält Daten aus der APHENITY-Studie und der laufenden offenen Erweiterungsstudie, die die Haltbarkeit der Wirkung von Sepiapterin zeigt und die Fähigkeit der Patienten darstellt, ihre Ernährung zu liberalisieren und gleichzeitig die Kontrolle über die Phenylalanin (Phe) -Spiegel aufrechtzuerhalten. Jüngste Ergebnisse zeigen, dass ungefähr 60 % der Personen die altersangepasste empfohlene Tagesdosis für eine nicht betroffene Person bei gleichzeitiger Beibehaltung von Phe-Niveaus < 360 μmol/L erreichen.
- FDA set a target regulatory action date for sepiapterin NDA review
- Phase 3 APHENITY trial results published in The Lancet
- NDA covers treatment for all age groups and PKU subtypes
- Open-label extension study shows durability of sepiapterin's effect
- 60% of subjects achieve higher protein intake while maintaining Phe control
- Standard review timeline for FDA, potentially longer approval process
Insights
The FDA's target action date for sepiapterin's NDA review is a significant milestone for PTC Therapeutics and PKU patients. The July 29, 2025 date suggests a standard 10-month review process, indicating no major red flags in the initial application. The NDA's broad indication covering all age groups and disease subtypes is ambitious and, if approved, could position sepiapterin as a comprehensive PKU treatment.
The Lancet publication of the Phase 3 APHENITY trial results adds substantial credibility to the drug's efficacy and safety profile. This high-profile publication can influence both regulatory decisions and potential market adoption. The ongoing open-label extension study data, showing 60% of subjects achieving protein intake above age-adjusted recommendations while maintaining controlled Phe levels, is particularly promising. This suggests sepiapterin could offer PKU patients a more normalized diet, addressing a key quality of life issue.
Investors should note that while these developments are positive, FDA approval is not guaranteed. The market potential for sepiapterin will depend on pricing, reimbursement and competition from existing and emerging PKU treatments. Long-term safety data and real-world effectiveness will be important for sustained market success post-approval.
"The Lancet publication reflects the promising and transformative nature of the data collected to date on sepiapterin and the therapy's potential to fill the persistent and significant unmet medical need for PKU patients," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics.
The sepiapterin NDA includes the results from the Phase 3 APHENITY trial discussed in The Lancet publication, as well as data from the ongoing APHENITY open-label extension study, which provides evidence of the durability of effect of sepiapterin and the ability of patients to liberalize their diet and still maintain control of phenylalanine (Phe) levels. The most recent results from the Phe tolerance assessment demonstrate that approximately
About Sepiapterin
Sepiapterin (formerly PTC923), an oral formulation of synthetic sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase (PAH) enzyme. First, sepiapterin is a precursor compound that is rapidly absorbed and converted intracellularly to tetrahydrobiopterin (BH4), a critical cofactor of PAH. Sepiapterin also has an independent pharmacological chaperone effect, correcting PAH misfolding to enhance the enzyme function. Through this dual mechanism of action, sepiapterin effectively reduces blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients.
About Phenylketonuria
Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain. It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine. If left untreated or poorly managed, phenylalanine – an essential amino acid found in all proteins and most foods – can build up to harmful levels in the body. This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with phenylketonuria initially don't have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of phenylalanine in the first few years of life is irreversible. Diagnosis of phenylketonuria usually takes place during newborn screening programs. There are an estimated 58,000 people with phenylketonuria globally.
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.
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FAQ
When is the FDA target regulatory action date for PTC Therapeutics' sepiapterin NDA (PTCT)?
What condition is sepiapterin designed to treat according to PTC Therapeutics' NDA (PTCT)?
Where were the Phase 3 APHENITY trial results for PTC Therapeutics' sepiapterin published (PTCT)?
What percentage of subjects in PTC Therapeutics' sepiapterin study achieved higher protein intake while maintaining Phe control (PTCT)?
