PTC Therapeutics Announces FDA Approval of Sephience鈩� (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
PTC Therapeutics (NASDAQ: PTCT) has received FDA approval for SEPHIENCE鈩� (sepiapterin), a treatment for phenylketonuria (PKU) in patients aged 1 month and older. The approval covers broad labeling for hyperphenylalaninemia (HPA) in sepiapterin-responsive PKU patients.
The approval is supported by significant efficacy and safety data from the Phase 3 APHENITY trial and its long-term extension study. SEPHIENCE has already received European Commission marketing authorization, with approval applications under review in Japan and Brazil.
The company aims to position SEPHIENCE as the future standard of care for PKU patients, with their experienced teams ready to launch the therapy in the United States.
PTC Therapeutics (NASDAQ: PTCT) ha ottenuto l'approvazione FDA per SEPHIENCE鈩� (sepiapterina), un trattamento per la fenilchetonuria (PKU) in pazienti a partire da 1 mese di et脿. L'approvazione copre un'ampia indicazione per l'iperfenilalaninemia (HPA) nei pazienti con PKU responsivi alla sepiapterina.
Il via libera si basa su dati significativi di efficacia e sicurezza provenienti dal trial di Fase 3 APHENITY e dal suo studio di estensione a lungo termine. SEPHIENCE ha gi脿 ricevuto l'autorizzazione alla commercializzazione dalla Commissione Europea, mentre le domande di approvazione sono in fase di valutazione in Giappone e Brasile.
L'azienda punta a posizionare SEPHIENCE come il futuro standard di cura per i pazienti con PKU, con team esperti pronti a lanciare la terapia negli Stati Uniti.
PTC Therapeutics (NASDAQ: PTCT) ha recibido la aprobaci贸n de la FDA para SEPHIENCE鈩� (sepiapterina), un tratamiento para la fenilcetonuria (PKU) en pacientes de 1 mes en adelante. La aprobaci贸n incluye una indicaci贸n amplia para hiperfenilalaninemia (HPA) en pacientes con PKU que responden a la sepiapterina.
La aprobaci贸n est谩 respaldada por datos significativos de eficacia y seguridad del ensayo de Fase 3 APHENITY y su estudio de extensi贸n a largo plazo. SEPHIENCE ya cuenta con autorizaci贸n de comercializaci贸n de la Comisi贸n Europea y las solicitudes de aprobaci贸n est谩n en revisi贸n en Jap贸n y Brasil.
La compa帽铆a busca posicionar SEPHIENCE como el futuro est谩ndar de cuidado para pacientes con PKU, con equipos experimentados listos para lanzar la terapia en Estados Unidos.
PTC Therapeutics (NASDAQ: PTCT)臧� 1臧滌洈 鞚挫儊 頇橃瀽鞚� 韼橂嫄旒�韱る嚚歃�(PKU) 旃橂鞝滌澑 SEPHIENCE鈩�(靹疙敿鞎勴攧韰岆Π)鞐� 雽�頃� FDA 鞀轨澑鞚� 氚涭晿鞀惦媹雼�. 鞚措矆 鞀轨澑鞚� 靹疙敿鞎勴攧韰岆Π鞐� 氚橃潙頃橂姅 PKU 頇橃瀽鞚� 瓿犿帢雼愳晫霛茧媽順堨(HPA)鞐� 雽�頃� 甏戨矓鞙勴暅 鞝侅潙歃濎潉 韽暔頃╇媹雼�.
鞚措矆 鞀轨澑鞚� 3靸� APHENITY 鞛勳儊鞁滍棙瓿� 鞛リ赴 鞐办灔 鞐瓣惮鞐愳劀 鞛呾霅� 鞙犾潣氙疙暅 須姤 氚� 鞎堨爠靹� 雿办澊韯办棎 旮半皹頃╇媹雼�. SEPHIENCE電� 鞚措 鞙犽熃鞐绊暕 歆戫枆鞙勳洂須岆攵�韯� 鞁滍寪 項堦皜毳� 氚涭晿鞙茧┌, 鞚茧掣瓿� 敫岆澕歆堨棎靹滊弰 鞀轨澑 鞁犾箔鞚� 鞁偓 欷戩瀰雼堧嫟.
須岇偓電� SEPHIENCE毳� PKU 頇橃瀽鞚� 氙鸽灅 響滌 旃橂鞝滊 鞛愲Μ毵り箑頃� 瓿勴殟鞚措┌, 氙戈淡 雮� 旃橂鞝� 於滌嫓毳� 鞙勴暣 瓴巾棙 毵庫潃 韺�鞚� 欷�牍勴晿瓿� 鞛堨姷雼堧嫟.
PTC Therapeutics (NASDAQ : PTCT) a obtenu l'approbation de la FDA pour SEPHIENCE鈩� (s茅piaprtine), un traitement de la ph茅nylc茅tonurie (PCU) chez les patients 芒g茅s d'un mois et plus. L'autorisation couvre une indication large pour l'hyperph茅nylalanin茅mie (HPA) chez les patients atteints de PCU r茅pondant 脿 la s茅piaprtine.
Cette approbation est soutenue par des donn茅es significatives d'efficacit茅 et de s茅curit茅 issues de l'essai de phase 3 APHENITY et de son 茅tude d'extension 脿 long terme. SEPHIENCE a d茅j脿 re莽u l'autorisation de mise sur le march茅 de la Commission europ茅enne, avec des demandes d'approbation en cours d'examen au Japon et au Br茅sil.
L'entreprise vise 脿 positionner SEPHIENCE comme la future r茅f茅rence en mati猫re de soins pour les patients atteints de PCU, avec des 茅quipes exp茅riment茅es pr锚tes 脿 lancer la th茅rapie aux 脡tats-Unis.
PTC Therapeutics (NASDAQ: PTCT) hat die FDA-Zulassung f眉r SEPHIENCE鈩� (Sepiapterin) erhalten, eine Behandlung f眉r Phenylketonurie (PKU) bei Patienten ab 1 Monat. Die Zulassung umfasst eine breite Kennzeichnung f眉r Hyperphenylalanin盲mie (HPA) bei sepiapterin-ansprechenden PKU-Patienten.
Die Zulassung st眉tzt sich auf bedeutende Wirksamkeits- und Sicherheitsdaten aus der Phase-3-APHENITY-Studie und deren Langzeitverl盲ngerung. SEPHIENCE hat bereits eine Marktzulassung von der Europ盲ischen Kommission erhalten, und Zulassungsantr盲ge in Japan und Brasilien werden derzeit gepr眉ft.
Das Unternehmen plant, SEPHIENCE als zuk眉nftigen Standard der Versorgung f眉r PKU-Patienten zu etablieren, wobei erfahrene Teams bereitstehen, die Therapie in den USA einzuf眉hren.
- FDA approval received for SEPHIENCE with broad labeling for all PKU subtypes
- Treatment approved for wide age range (1 month and older)
- European Commission marketing authorization already secured
- Additional market opportunities with pending approvals in Japan and Brazil
- Strong efficacy and safety data from Phase 3 APHENITY trial
- None.
Insights
FDA approval of Sephience for PKU represents significant market opportunity with broad labeling and strong efficacy data.
The FDA approval of Sephience (sepiapterin) marks a significant therapeutic advancement for phenylketonuria (PKU) treatment. The notably broad labeling - covering all disease subtypes for patients as young as 1 month - substantially expands the addressable patient population compared to more restrictive approvals typically seen with orphan drugs.
The approval's strength comes from its dual clinical foundation: the pivotal Phase 3 APHENITY trial demonstrating efficacy and safety, plus extension study data confirming durability of effect. This robust evidence package likely facilitated the comprehensive indication.
From a regulatory perspective, this approval represents a multi-regional regulatory success story. With European Commission authorization already secured and applications under review in Japan and Brazil, PTC is executing a well-coordinated global regulatory strategy typical of successful rare disease commercialization.
The endorsement from the National PKU Alliance adds credibility, highlighting the treatment's potential impact on patient quality of life. PKU requires lifelong management to prevent neurological damage from elevated phenylalanine levels, and current treatment options are limited with significant compliance challenges. Sephience addresses a genuine unmet need in this space.
PTC's readiness for immediate commercialization through their "experienced customer facing teams" suggests confidence in rapid uptake and revenue generation, critical for specialty pharmaceuticals in the rare disease space.
Sephience approval unlocks significant revenue potential in underserved PKU market with advantageous broad labeling across age groups.
This approval positions PTC Therapeutics to capture substantial market share in the PKU treatment landscape. The exceptionally broad labeling - covering patients from just 1 month of age and all sepiapterin-responsive subtypes - provides PTC with a competitive commercial advantage compared to existing treatments.
The PKU market has historically been underserved despite affecting approximately 1 in 10,000-15,000 newborns in the United States. Current treatment options primarily consist of dietary restrictions, medical foods, and limited pharmacological interventions like Kuvan (sapropterin) and Palynziq (pegvaliase-pqpz).
PTC's statement about becoming "the future standard of care" isn't mere corporate optimism - it's grounded in clinical reality. The drug's mechanism targeting sepiapterin-responsive PKU addresses fundamental disease pathophysiology rather than just symptom management.
The global regulatory strategy shows commercial sophistication. With European approval secured and applications under review in Japan and Brazil, PTC is implementing a coordinated international launch sequence that maximizes revenue potential across major pharmaceutical markets.
The CEO's emphasis on their "experienced customer facing teams" indicates readiness for the specialized marketing required in the rare disease space, where identifying patients and securing reimbursement requires deep expertise. This infrastructure suggests PTC anticipates minimal commercial delays between approval and generating meaningful revenue from Sephience.
- Broad labeling inclusive of all disease subtypes for individuals 1 month of age and older -
- PTC will host a conference call on Monday, July 28th at 5:00 pm ET -
"We are excited to have reached this important milestone for those affected by PKU," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The broad labeling reflects the potential of Sephience to meet the significant unmet need of PKU patients. The Sephience clinical data along with our expertise in launching rare disease therapies position Sephience to become the future standard of care. Our experienced customer facing teams are ready to bring this therapy to children and adults with PKU in
The FDA approval is based on the evidence of significant efficacy and safety from the听
Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study. 听听
"The approval marks an exciting milestone for the PKU community," said Catherine Warren, Executive Director of the National PKU Alliance. "This progress brings renewed hope, and we are eager to see the positive impact this new treatment option will have on advancing care and potentially improving quality of life for individuals of all ages and PKU subtypes that respond to this therapy."
SEPHIENCE was recently granted marketing authorization by the European Commission. Review of approval applications is ongoing in several other countries including
Conference Call and Webcast
PTC will hold a conference call Monday, July 28, 2025 at 5:00 pm ET to discuss this news. To access the call by phone, please click听听to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at . A replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call.
About SEPHIENCE鈩� (sepiapterin)
SEPHIENCE is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Through its mechanism of action, Sephience is able to effectively reduce blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients. Sephience is approved in the European Economic Area and
Indication and Important Safety Information
Indication
SEPHIENCE is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). SEPHIENCE is to be used in conjunction with a phenylalanine (Phe)-restricted diet.
Contraindications
None
Important Safety Information
Treatment with SEPHIENCE should be directed by physicians knowledgeable in the management of PKU. Biochemical response to SEPHIENCE can only be determined by a therapeutic trial with careful monitoring of ongoing dietary and nutritional balance to ensure adequate Phe control.
Warnings and Precautions
- Increased Bleeding: SEPHIENCE may increase the risk of bleeding. Bleeding events, including superficial hematomas, prolonged bleeding, and heavy menstrual bleeding have occurred in patients treated with SEPHIENCE. Inform patients about the risk of bleeding associated with SEPHIENCE and have patients follow up with their healthcare provider should such a bleeding event occur. Consider treatment interruption with SEPHIENCE in patients with active bleeding.
- Hypophenylalaninemia: Some pediatric patients receiving SEPHIENCE experienced hypophenylalaninemia. Monitor blood Phe levels during treatment and modify the dosage of SEPHIENCE and/or dietary protein and Phe intake as needed to ensure adequate blood Phe level control. Frequent blood monitoring is recommended in the pediatric population.
- Interaction with Levodopa: In a 10-year post-marketing safety surveillance program for a non-PKU indication using another drug that is a phenylalanine hydroxylase (PAH) activator, 3 patients with underlying neurological disorders experienced seizures, exacerbation of seizures, over-stimulation, and irritability during co-administration with levodopa. Monitor patients who are receiving levodopa for changes in neurological status during treatment with SEPHIENCE.
Adverse Reactions
Most common adverse reactions with SEPHIENCE (鈮�
Drug Interactions
Avoid concomitant use of drugs known to inhibit folate synthesis dihydrofolate reductase (DHFR) (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, and piritrexim) while taking SEPHIENCE. Concomitant administration of such drugs may reduce sepiapterin metabolism to BH4. If concomitant use is not avoidable, monitor blood Phe levels.
SEPHIENCE and PDE-5 inhibitors (e.g., sildenafil, vardenafil, or tadalafil) induce vasorelaxation and may reduce blood pressure. Monitor for signs and symptoms of hypotension.
For medical information, product complaints, or to report an adverse event, please call 1鈥�866鈥�562鈥�4620 or email at [email protected].
You may also report adverse events directly to FDA at 1鈥�800鈥揊DA鈥�1088 or .
Please see .
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鈥攁n essential amino acid found in all proteins and most foods鈥攃an 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 do not 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.听
PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit听www.ptcbio.com听and follow on Facebook, X, and LinkedIn.
For More Information:鈥�听
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Jeanine Clemente听
+1 (908) 912-9406听
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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 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 timing of commercialization and of potential regulatory decisions; 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
What is the FDA approval status of PTC Therapeutics' SEPHIENCE for PKU treatment?
What age groups can receive SEPHIENCE treatment for PKU?
What clinical trials supported SEPHIENCE's FDA approval?
In which countries is SEPHIENCE approved for PKU treatment?
How does SEPHIENCE benefit PKU patients?
