PTC Therapeutics Announces Sepiapterin NDA Submission to FDA
PTC Therapeutics (NASDAQ: PTCT) has submitted a New Drug Application (NDA) to the FDA for sepiapterin, targeting the treatment of phenylketonuria (PKU) in both pediatric and adult patients. The submission is based on significant results from the phase 3 APHENITY trial, which showed a 63% mean reduction in Phe levels overall and 69% in classical PKU patients. Notably, 84% of subjects achieved Phe control (<360 µmol/L) and 22% normalized Phe levels. The Phe tolerance sub-study revealed that 60% of subjects could increase protein intake while maintaining Phe control, potentially allowing for diet liberalization. Marketing applications for Europe are under review, with submissions for Japan and Brazil expected later in 2024.
PTC Therapeutics (NASDAQ: PTCT) ha presentato una Nuova Domanda di Farmaco (NDA) alla FDA per sepiapterin, mirato al trattamento della fenilchetonuria (PKU) in pazienti pediatrici e adulti. La domanda si basa sul trial di fase 3 APHENITY, che ha mostrato risultati significativi:
- Riduzione media dei livelli di Phe del 63% in generale
- Riduzione del 69% nel sottogruppo con PKU classica
- L'84% dei soggetti ha raggiunto il controllo dei Phe (<360 µmol/L)
- Il 22% ha mostrato normalizzazione dei livelli di Phe
Lo studio supplementare sulla tolleranza ai Phe ha rivelato che il 60% dei soggetti potrebbe aumentare l'assunzione di proteine oltre i livelli raccomandati mantenendo il controllo dei Phe, consentendo potenzialmente una liberalizzazione della dieta. Le domande di marketing per l'Europa sono in fase di revisione, con domanda per Giappone e Brasile prevista entro il 2024.
PTC Therapeutics (NASDAQ: PTCT) ha presentado una Nueva Solicitud de Medicamento (NDA) a la FDA para sepiapterin, dirigido al tratamiento de fenilcetonuria (PKU) en pacientes pediátricos y adultos. La presentación se basa en el ensayo de fase 3 APHENITY, que mostró resultados significativos:
- Reducción media del 63% en los niveles de Phe en general
- Reducción del 69% en el subgrupo de PKU clásica
- El 84% de los sujetos logró control de Phe (<360 µmol/L)
- El 22% mostró normalización de los niveles de Phe
El subestudio de tolerancia a Phe reveló que el 60% de los sujetos podrían aumentar su consumo de proteínas por encima de los niveles recomendados mientras mantenían el control de Phe, lo que podría permitir una liberalización de la dieta. Las solicitudes de comercialización para Europa están en revisión, con solicitudes para Japón y Brasil previstas para más tarde en 2024.
PTC Therapeutics (NASDAQ: PTCT)는 세피아프테린에 대한 새로운 약물 신청(NDA)를 FDA에 제출했습니다. 이는 소아 및 성인 환자의 페닐케톤뇨(PKU) 치료를 목표로 하고 있습니다. 이 제출은 APHENITY 3상 시험에 기반하고 있으며, 상당한 결과를 보여주었습니다:
- 전체 평균 Phe 수치 63% 감소
- 고전적 PKU 하위 그룹에서 69% 감소
- 84%의 피험자가 Phe 조절(<360 µmol/L) 달성
- 22%가 Phe 수치 정상화 보임
Phe 내성 하위 연구는 60%의 피험자가 권장 수준 이상의 단백질 섭취를 증가시키면서 Phe 조절을 유지할 수 있음을 보여주어, 잠재적으로 식단의 자유화를 허용할 수 있습니다. 유럽을 위한 마케팅 신청은 검토 중이며, 일본과 브라질에 대한 신청은 2024년 후반에 예상됩니다.
PTC Therapeutics (NASDAQ: PTCT) a présenté une Nouvelle Demande de Médicament (NDA) à la FDA pour sepiapterin, visant le traitement de phénylcétonurie (PKU) chez des patients pédiatriques et adultes. La soumission repose sur l'essai de phase 3 APHENITY, qui a montré des résultats significatifs :
- Réduction moyenne des niveaux de Phe de 63% au total
- Réduction de 69% dans le sous-groupe PKU classique
- 84% des sujets ont atteint le contrôle de Phe (<360 µmol/L)
- 22% ont montré une normalisation des niveaux de Phe
Le sous-étude sur la tolérance aux Phe a révélé que 60% des sujets pouvaient augmenter leur apport en protéines au-delà des niveaux recommandés tout en maintenant le contrôle de Phe, ce qui permettrait potentiellement une libéralisation de l'alimentation. Les demandes de commercialisation pour l'Europe sont en cours d'examen, avec des soumissions pour le Japon et le Brésil prévues plus tard en 2024.
PTC Therapeutics (NASDAQ: PTCT) hat einen neuen Arzneimittelantrag (NDA) bei der FDA für Sepiapterin eingereicht, der auf die Behandlung von Phenylketonurie (PKU) bei pädiatrischen und erwachsenen Patienten abzielt. Die Einreichung basiert auf der APHENITY Phase 3 Studie, die bedeutende Ergebnisse zeigte:
- Durchschnittliche Reduktion der Phe-Spiegel um 63% insgesamt
- 69% Reduktion in der klassischen PKU-Untergruppe
- 84% der Probanden erreichten die Kontrolle über Phe (<360 µmol/L)
- 22% zeigten eine Normalisierung der Phe-Spiegel
Die Zusatzstudie zur Phe-Toleranz ergab, dass 60% der Probanden die Proteinzufuhr über die empfohlenen Werte erhöhen konnten, während sie die Kontrolle über Phe aufrechterhielten, was möglicherweise eine Liberalisierung der Diät ermöglichen könnte. Die Markanträge für Europa werden geprüft, während die Anträge für Japan und Brasilien später im Jahr 2024 erwartet werden.
- NDA submission to FDA for sepiapterin to treat PKU in all age groups
- Phase 3 APHENITY trial showed 63% mean reduction in Phe levels
- 84% of subjects achieved Phe control (<360 µmol/L)
- 60% of subjects in Phe tolerance sub-study achieved higher protein intake while maintaining Phe control
- Potential for diet liberalization in PKU patients
- Marketing applications in progress for Europe, Japan, and Brazil
- None.
The submission of the sepiapterin New Drug Application (NDA) to the FDA by PTC Therapeutics is a significant development in the treatment of phenylketonuria (PKU). This impactful news could potentially reshape the therapeutic landscape for PKU patients.
The phase 3 APHENITY trial results are particularly noteworthy:
- A mean reduction in phenylalanine (Phe) levels of
63% in the overall treated population 69% reduction in the classical PKU subgroup84% of subjects achieved Phe control (<360 µmol/L)22% of subjects showed normalization of Phe levels
These results suggest a robust efficacy profile for sepiapterin. The durability of effect demonstrated in the open-label extension study further strengthens the drug's potential.
The Phe tolerance sub-study results are particularly intriguing. With
From a market perspective, the comprehensive data package, including the Phe tolerance results, could position sepiapterin favorably for both physician adoption and payer acceptance. The potential for improved patient outcomes and quality of life could drive strong demand if approved.
However, it's important to note that FDA approval is not guaranteed and the review process will scrutinize the data for safety and efficacy. The timeline for potential approval and market entry remains uncertain at this stage.
The submission of the sepiapterin NDA to the FDA represents a significant milestone for PTC Therapeutics, potentially expanding their product portfolio in the rare disease space. This development could have substantial implications for the company's financial outlook and market position.
Key financial considerations include:
- Market Opportunity: PKU affects approximately 1 in 10,000 to 15,000 newborns in the United States. While this is a relatively small patient population, orphan drugs often command premium pricing, potentially translating to significant revenue if approved.
- Competition: The PKU treatment landscape includes established players like BioMarin's Kuvan and Palynziq. Sepiapterin's efficacy profile and potential for diet liberalization could provide a competitive edge, potentially capturing market share.
- Global Expansion: With marketing authorization applications planned or under review in Europe, Japan and Brazil, PTC is positioning sepiapterin for a global market, which could substantially increase the drug's revenue potential.
- R&D Investment: The successful completion of the phase 3 trial and NDA submission represents a significant R&D investment. If approved, this could validate PTC's R&D strategy and pipeline.
However, investors should be cautious. The FDA review process is rigorous and approval is not guaranteed. Additionally, even if approved, market uptake and reimbursement negotiations will be critical factors in determining the drug's commercial success.
From a financial perspective, while the NDA submission is a positive development, it's important to consider PTC's overall financial health, including its current revenue streams, cash position and burn rate. The potential approval and launch of sepiapterin could significantly impact the company's financial trajectory, but this remains speculative until more concrete information is available.
"This NDA submission is an important step in bringing this therapy to children and adults living with PKU in the
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 Phe levels of
The European marketing authorization application is currently under review and the marketing authorization applications for
About Sepiapterin
Sepiapterin (formerly PTC923) is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products. Sepiapterin is a more bioavailable precursor than exogenously administered synthetic BH4 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 patients 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:
Investors:
Kylie O'Keefe
+1 (908) 300-0691
kokeefe@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; 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; expectations with respect to sepiapterin, including any regulatory submissions and potential approvals, commercialization, the potential achievement of development, 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 sepiapterin.
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.
Acronyms:
FDA:
NDA: New Drug Application
Phe: Phenylalanine
PKU: Phenylketonuria
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SOURCE PTC Therapeutics, Inc.
FAQ
What is the purpose of PTC Therapeutics' sepiapterin NDA submission to the FDA?
What were the key results from the phase 3 APHENITY trial for PTCT's sepiapterin?
How might sepiapterin impact the diet of PKU patients according to PTC Therapeutics' study?
What is the status of PTC Therapeutics' (PTCT) sepiapterin marketing applications outside the US?
