PTC Therapeutics Receives Positive CHMP Opinion for Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
PTC Therapeutics (NASDAQ: PTCT) has received a positive opinion from the European Medicines Agency's CHMP for Sephience™ (sepiapterin), a treatment for phenylketonuria (PKU) in children and adults. The approval recommendation includes a comprehensive label covering all age groups and disease severities.
The company plans to launch immediately after European Commission ratification, expected in approximately two months. The authorization will apply to all 27 EU member states plus Iceland, Norway, and Liechtenstein, with initial focus on Germany and key European markets offering immediate named patient access.
Additionally, Sephience is under review by the FDA with a PDUFA date of July 29, 2025, and approval applications are being processed in Japan and Brazil.
PTC Therapeutics (NASDAQ: PTCT) ha ricevuto un parere positivo dal CHMP dell'Agenzia Europea per i Medicinali riguardo Sephience™ (sepiaptrina), un trattamento per la fenilchetonuria (PKU) in bambini e adulti. La raccomandazione di approvazione include un'etichetta completa che copre tutte le fasce d'età e le gravità della malattia.
L'azienda prevede di lanciare il prodotto subito dopo la ratifica della Commissione Europea, attesa entro circa due mesi. L'autorizzazione sarà valida in tutti i 27 stati membri dell'UE più Islanda, Norvegia e Liechtenstein, con un focus iniziale su Germania e mercati europei chiave che offriranno accesso immediato ai pazienti nominativi.
Inoltre, Sephience è in fase di revisione da parte della FDA con data PDUFA fissata per il 29 luglio 2025, mentre le domande di approvazione sono in corso in Giappone e Brasile.
PTC Therapeutics (NASDAQ: PTCT) ha recibido una opinión positiva del CHMP de la Agencia Europea de Medicamentos sobre Sephience™ (sepiapterina), un tratamiento para la fenilcetonuria (PKU) en niños y adultos. La recomendación de aprobación incluye una etiqueta completa que abarca todos los grupos de edad y grados de severidad de la enfermedad.
La compañía planea lanzar el producto inmediatamente después de la ratificación de la Comisión Europea, prevista en aproximadamente dos meses. La autorización aplicará a los 27 estados miembros de la UE más Islandia, Noruega y Liechtenstein, con un enfoque inicial en Alemania y los principales mercados europeos que ofrecerán acceso inmediato a pacientes nominativos.
Además, Sephience está bajo revisión de la FDA con una fecha PDUFA para el 29 de julio de 2025, y las solicitudes de aprobación están en proceso en Japón y Brasil.
PTC Therapeutics (NASDAQ: PTCT)는 어린이와 성인의 페닐케톤뇨증(PKU) 치료제인 Sephience™(세피아프테린)에 대해 유럽의약품청(EMA) CHMP로부터 긍정적인 의견을 받았습니다. 승인 권고안에는 모든 연령대와 질병 중증도를 포괄하는 상세한 라벨이 포함되어 있습니다.
회사는 약 2개월 후 예상되는 유럽위원회 승인 후 즉시 출시할 계획입니다. 이 허가는 27개 EU 회원국과 아이슬란드, 노르웨이, 리히텐슈타인에 적용되며, 초기에는 독일과 주요 유럽 시장에 집중하여 즉각적인 지정환자 접근을 제공할 예정입니다.
또한 Sephience는 FDA 심사 중이며, PDUFA 날짜는 2025년 7월 29일로 예정되어 있으며 일본과 브라질에서도 승인 신청이 진행 중입니다.
PTC Therapeutics (NASDAQ : PTCT) a reçu un avis favorable du CHMP de l'Agence européenne des médicaments concernant Sephience™ (sépiaptrine), un traitement de la phénylcétonurie (PCU) chez l'enfant et l'adulte. La recommandation d'approbation inclut une étiquette complète couvrant tous les groupes d'âge et degrés de gravité de la maladie.
L'entreprise prévoit un lancement immédiat après la ratification par la Commission européenne, attendue dans environ deux mois. L'autorisation s'appliquera aux 27 États membres de l'UE ainsi qu'à l'Islande, la Norvège et le Liechtenstein, avec un focus initial sur l'Allemagne et les principaux marchés européens offrant un accès immédiat aux patients nommés.
De plus, Sephience est en cours d'examen par la FDA avec une date PDUFA fixée au 29 juillet 2025, et les demandes d'approbation sont en cours au Japon et au Brésil.
PTC Therapeutics (NASDAQ: PTCT) hat eine positive Stellungnahme des CHMP der Europäischen Arzneimittelagentur für Sephience™ (Sepiapterin) erhalten, eine Behandlung für Phenylketonurie (PKU) bei Kindern und Erwachsenen. Die Zulassungsempfehlung umfasst ein umfassendes Etikett, das alle Altersgruppen und Krankheitsgrade abdeckt.
Das Unternehmen plant die Markteinführung unmittelbar nach der Ratifizierung durch die Europäische Kommission, die in etwa zwei Monaten erwartet wird. Die Zulassung gilt für alle 27 EU-Mitgliedstaaten sowie Island, Norwegen und Liechtenstein, mit anfänglichem Fokus auf Deutschland und wichtige europäische Märkte, die sofortigen Zugang für benannte Patienten bieten.
Zusätzlich wird Sephience von der FDA geprüft, mit einem PDUFA-Datum am 29. Juli 2025, und Zulassungsanträge werden in Japan und Brasilien bearbeitet.
- Received positive CHMP opinion for Sephience, indicating likely EU approval
- Broad label approval covering all ages and disease severities
- Imminent market access in 30 European countries
- Named patient access immediately available in key European markets
- Full EU Commission approval still pending
- FDA approval not yet secured
Insights
CHMP positive opinion for Sephience gives PTC Therapeutics a clear path to European approval for PKU treatment with an inclusive label.
The positive CHMP opinion for Sephience (sepiapterin) represents a critical regulatory milestone in bringing a new treatment option to the phenylketonuria (PKU) patient community in Europe. The broad label spanning all ages and disease severities is particularly significant, as it will allow treatment across the full spectrum of patients without restrictions that could limit market access.
PKU is a rare genetic disorder where patients cannot properly metabolize the amino acid phenylalanine, which can build up to harmful levels in the body. Traditional management requires extremely restrictive diets that significantly impact quality of life. Sephience appears positioned to address this challenge, with the CEO highlighting its potential to enable "diet liberalization even for those with the most severe disease subtypes" while lowering phenylalanine levels.
From a regulatory perspective, the European Commission typically adopts CHMP recommendations within approximately 60 days, aligning with PTC's stated expectation of about two months. This would make the therapy available across 27 EU member states plus Iceland, Norway, and Liechtenstein. The company appears well-prepared for commercialization, with launch planning prioritizing Germany and other key markets where named patient access will be immediately available following authorization.
This European progress complements ongoing regulatory reviews in other major markets, including a pending FDA decision with a PDUFA date of July 29, 2025, as well as submissions in Japan and Brazil. The coordinated global regulatory strategy demonstrates comprehensive planning for worldwide commercialization of this potential new therapy for an indication with continuing unmet needs.
Positive CHMP opinion for Sephience reduces regulatory risk and positions PTC for potential revenue from European PKU market ahead of US decision.
The positive CHMP opinion for Sephience represents a significant derisking event for PTC Therapeutics' development program addressing phenylketonuria (PKU). The company has secured a recommendation for a comprehensive label covering all ages and disease severities, which maximizes the potential addressable patient population in Europe.
PKU, though rare, requires lifelong management, creating a stable, enduring market for effective therapies. The CEO's reference to "significant unmet medical need" suggests Sephience could occupy an important position in the treatment paradigm. The possibility of diet liberalization addresses a major quality-of-life concern, potentially enhancing the therapy's value proposition to patients, clinicians, and payers.
PTC's launch readiness is evident in their statement that preparations are "well underway with priorities in Germany and other key European markets." The mention of immediate named patient access following approval suggests a strategy to generate early revenue even before full commercial rollout. The German market is typically prioritized in European launches due to its size and relatively favorable rare disease reimbursement environment.
This European regulatory progress occurs in parallel with reviews in other major pharmaceutical markets. The FDA decision expected by July 29, 2025, represents another potential catalyst. The global approach to registration in the US, Europe, Japan, and Brazil demonstrates a comprehensive commercialization strategy targeting the major pharmaceutical markets simultaneously.
The advancement toward European approval reduces one component of regulatory risk in PTC's overall development program for Sephience, though final commercialization remains contingent on European Commission authorization and successful market access negotiations in individual countries.
- Label includes full spectrum of PKU patients -
- European launch preparations underway -
"This positive opinion is great news for the PKU patient community in
Launch planning in
PTC expects the European Commission to ratify the marketing authorization for Sephience in approximately two months. The decision will be applicable to all 27 European Union member states, as well as
The New Drug Application (NDA) for sepiapterin is under review by the
About Sephience™ (sepiapterin)
Sephience, an oral formulation of synthetic sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase (PAH) enzyme. First, Sephience is a precursor compound that is rapidly absorbed and converted intracellularly to tetrahydrobiopterin (BH4), a critical cofactor of PAH. Sephience also has an independent pharmacological chaperone effect, correcting PAH misfolding to enhance the enzyme function. Through this dual mechanism of action, Sephience 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 (Phe). If left untreated or poorly managed, Phe – 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 PKU initially don't have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of Phe in the first few years of life is irreversible. Diagnosis of PKU usually takes place during newborn screening programs. There are an estimated 58,000 people living with PKU 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 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, X, and LinkedIn.
For More Information:
Investors:
Ellen Cavaleri
+1 (615) 618-6228
ecavaleri@ptcbio.com
Media:
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+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 clinical trials and studies, availability of data, regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; expectations with respect to Sephience, including any regulatory decision made by the European Commission; 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 Sephience, including any regulatory submissions and potential approvals, commercialization and 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 Sephience.
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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FAQ
When will Sephience (PTCT) be available in the European market?
What is the scope of Sephience's CHMP-recommended approval for PKU treatment?
Which countries will have access to Sephience following EU approval?
When is the FDA expected to decide on Sephience's approval?
Where else is Sephience seeking regulatory approval besides Europe and the US?
