PTC Therapeutics Announces FDA Acceptance for Filing of NDA for Sepiapterin for the Treatment of Pediatric and Adult Phenylketonuria Patients
PTC Therapeutics (NASDAQ: PTCT) announced that the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for treating pediatric and adult patients with phenylketonuria (PKU). The NDA is based on the phase 3 APHENITY trial results, which showed a mean reduction in phenylalanine (Phe) levels of 63% in the overall treated population and 69% in classical PKU subjects. 84% of subjects achieved Phe control (<360 µmol/L) as per treatment guidelines, and 22% showed Phe level normalization.
The NDA also includes data from the APHENITY open-label extension study and the Phe tolerance sub-study. The latter demonstrated that about 60% of subjects achieved protein intake above the age-adjusted recommended daily allowance while maintaining Phe control, suggesting sepiapterin could enable diet liberalization for PKU patients.
PTC Therapeutics (NASDAQ: PTCT) ha annunciato che la FDA ha accettato di esaminare la Richiesta di Autorizzazione di Nuovo Farmaco (NDA) per sepiapterin nel trattamento di pazienti pediatrici e adulti affetti da fenilchetonuria (PKU). L'NDA si basa sui risultati del trial di fase 3 APHENITY, che ha mostrato una riduzione media dei livelli di fenilalanina (Phe) del 63% nella popolazione complessiva trattata e del 69% nei soggetti con PKU classica. L'84% dei soggetti ha raggiunto il controllo dei livelli di Phe (<360 µmol/L) secondo le linee guida del trattamento, e il 22% ha mostrato una normalizzazione dei livelli di Phe.
L'NDA include anche dati dallo studio di estensione in aperto APHENITY e dallo studio secondario sulla tolleranza alla Phe. Quest'ultimo ha dimostrato che circa il 60% dei soggetti ha raggiunto un apporto proteico superiore alla dose giornaliera raccomandata in base all'età, mantenendo il controllo della Phe, suggerendo che la sepiapterin potrebbe consentire una liberalizzazione della dieta per i pazienti con PKU.
PTC Therapeutics (NASDAQ: PTCT) anunció que la FDA ha aceptado para su evaluación la Solicitud de Nuevo Medicamento (NDA) de sepiapterina para tratar a pacientes pediátricos y adultos con fenilcetonuria (PKU). La NDA se basa en los resultados del ensayo de fase 3 APHENITY, que mostró una reducción media en los niveles de fenilalanina (Phe) del 63% en la población total tratada y del 69% en sujetos con PKU clásica. El 84% de los sujetos alcanzó el control de Phe (<360 µmol/L) según las directrices de tratamiento, y el 22% mostró normalización de los niveles de Phe.
La NDA también incluye datos del estudio de extensión de etiqueta abierta APHENITY y del sub-estudio de tolerancia a la Phe. Este último demostró que aproximadamente el 60% de los sujetos alcanzó una ingesta de proteínas por encima de la ingesta diaria recomendada ajustada por edad mientras mantenía el control de Phe, sugiriendo que la sepiapterina podría permitir una liberalización de la dieta para pacientes con PKU.
PTC 테라퓨틱스(NASDAQ: PTCT)는 FDA가 세피아프테린의 신약 승인을 위한 신청서(NDA)를 접수하였음을 발표하였습니다. 이 약물은 소아 및 성인 페닐케톤뇨증(PKU) 환자를 치료하는 데 사용됩니다. NDA는 3상 APHENITY 시험 결과를 기반으로 하며, 전체 치료 집단에서 페닐알라닌(Phe) 수치의 평균 63% 감소와 PKU 고전 환자에서 69% 감소를 보였습니다. 84%의 환자가 Phe 조절(<360 µmol/L)에 성공했으며, 22%는 Phe 수치의 정상화를 보여주었습니다.
NDA는 또한 APHENITY 공개 라벨 확장 연구와 Phe 내성 하위 연구의 데이터를 포함합니다. 후자는 대상자의 약 60%가 연령 조정 권장 일일 섭취량 이상의 단백질 섭취를 유지하면서 Phe 조절을 이루었습니다, 이는 세피아프테린이 PKU 환자에게 식이 시판을 가능하게 할 수 있음을 시사합니다.
PTC Therapeutics (NASDAQ: PTCT) a annoncé que la FDA a accepté de traiter la Demande d'Autorisation de Nouveau Médicament (NDA) de sepiapterine pour le traitement des patients pédiatriques et adultes atteints de phénylcétonurie (PKU). La NDA est basée sur les résultats de l'essai de phase 3 APHENITY, qui a montré une réduction moyenne des niveaux de phénylalanine (Phe) de 63% dans la population traitée dans son ensemble et de 69% chez les sujets présentant une PKU classique. 84% des sujets ont atteint le contrôle des niveaux de Phe (<360 µmol/L) conformément aux directives de traitement, et 22% ont montré une normalisation des niveaux de Phe.
La NDA comprend également des données provenant de l'étude d'extension en ouvert APHENITY et de l'étude secondaire sur la tolérance à la Phe. Cette dernière a démontré que environ 60% des sujets ont atteint une consommation de protéines supérieure à l'apport quotidien recommandé en fonction de l'âge tout en maintenant le contrôle de la Phe, ce qui suggère que la sepiapterine pourrait permettre une libéralisation du régime alimentaire pour les patients atteints de PKU.
PTC Therapeutics (NASDAQ: PTCT) gab bekannt, dass die FDA den Antrag auf Zulassung eines neuen Arzneimittels (NDA) für Sepiapterin zur Behandlung von pädiatrischen und erwachsenen Patienten mit Phenylketonurie (PKU) angenommen hat. Der NDA basiert auf den Ergebnissen der Phase-3-APHENITY-Studie, die eine durchschnittliche Reduktion der Phenylalanin (Phe)-Spiegel um 63% in der gesamten behandelten Population und um 69% bei klassischer PKU zeigte. 84% der Probanden erreichten die Phe-Kontrolle (<360 µmol/L) gemäß den Behandlungsrichtlinien, und 22% zeigten eine Normalisierung der Phe-Spiegel.
Der NDA beinhaltet auch Daten aus der offenen Erweiterungsstudie APHENITY und der Phe-Toleranz-Substudie. Letztere zeigte, dass rund 60% der Probanden die proteineinnahme über der altersgerecht empfohlenen Tagesdosis erreichten, während sie die Phe-Kontrolle aufrechterhielten, was darauf hindeutet, dass Sepiapterin eine Liberalisierung der Diät für PKU-Patienten ermöglichen könnte.
- FDA acceptance of NDA filing for sepiapterin in PKU treatment
- Phase 3 APHENITY trial showed 63% mean reduction in Phe levels
- 84% of subjects achieved Phe control as per treatment guidelines
- 22% of subjects showed normalization of Phe levels
- 60% of subjects achieved higher protein intake while maintaining Phe control
- None.
Insights
The FDA's acceptance of PTC Therapeutics' NDA for sepiapterin marks a significant milestone in PKU treatment. This development could potentially address a critical unmet need in the PKU community. The phase 3 APHENITY trial results are particularly impressive, showing a
Key points to consider:
- 84% of subjects achieved Phe control (<360 µmol/L)
- 22% showed normalization of Phe levels
- The Phe tolerance sub-study indicates
60% of subjects could increase protein intake while maintaining Phe control
These results suggest sepiapterin could offer significant benefits, including potential diet liberalization for PKU patients. This could greatly improve quality of life and reduce the burden of strict dietary management. The durability of effect shown in the open-label extension study further supports its long-term potential.
The FDA's acceptance of the sepiapterin NDA is a positive development for PTC Therapeutics (NASDAQ: PTCT). With a market cap of
- Potential market expansion in the PKU treatment space
- Possible revenue growth if sepiapterin receives FDA approval
- Increased competitive advantage in the rare disease therapeutics market
The unmet need in PKU treatment, highlighted by the National PKU Alliance's statement, suggests a potentially receptive market for sepiapterin. If approved, this drug could become a preferred treatment option, potentially driving sales and market share for PTC Therapeutics. Investors should monitor the upcoming PDUFA date and any further clinical or regulatory developments closely.
"The FDA filing acceptance for sepiapterin is a critical milestone toward bringing this potential important therapy to children and adults living with PKU in
"NPKUA is deeply committed to improving the lives of individuals with PKU," said Catherine Warren, Executive Director, National PKU Alliance. "Our families have been anxiously waiting for new treatments that provide an opportunity to lessen their dependance on costly and burdensome diets. We are excited by today's news, as it brings us one step closer to providing another option for people of all ages with PKU to manage their condition."
The sepiapterin NDA is based on the highly statistically significant and clinically meaningful results from the phase 3 APHENITY trial. The results demonstrate a mean reduction in phenylalanine (Phe) levels of
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 chaperone effect, protecting against 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 the 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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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; 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.
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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 sepiapterin.
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SOURCE PTC Therapeutics, Inc.
FAQ
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