Sepiapterin
- Generic Name
- Sepiapterin
- Drug Type
- Small Molecule
- Chemical Formula
- C9H11N5O3
- CAS Number
- 17094-01-8
- Unique Ingredient Identifier
- CJQ26KO7HP
- Background
Sepiapterin is under investigation in clinical trial NCT03519711 (A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia).
PTC Therapeutics' Sephience Receives Positive CHMP Opinion for Phenylketonuria Treatment Across All Age Groups
• The European Medicines Agency's CHMP has issued a positive opinion for Sephience (sepiapterin) to treat phenylketonuria in patients of all ages and disease severities, addressing a significant unmet medical need.
• Sephience works through a dual mechanism of action that increases phenylalanine hydroxylase enzyme activity, effectively reducing blood phenylalanine levels while potentially allowing for diet liberalization.
• PTC Therapeutics expects European Commission approval within two months, with launch preparations prioritizing Germany and other key markets, while FDA review continues with a target action date of July 29, 2025.
Phenylketonuria Pipeline Shows Robust Growth with 25+ Therapies in Development
• DelveInsight's latest report reveals a robust phenylketonuria (PKU) pipeline with over 20 companies developing 25+ therapeutic candidates, signaling significant advancement in treatment options for this rare metabolic disorder.
• PTC Therapeutics' sepiapterin has reached registration stage with the FDA accepting its New Drug Application, while Otsuka Pharmaceutical's JNT-517 advances in Phase III trials as a first-in-class oral therapy for PKU patients regardless of age or genotype.
• Novel approaches including enzyme replacements, gene therapies, and innovative oral formulations are transforming PKU management beyond traditional dietary restrictions, with companies like BioMarin, Sanofi, and Relief Therapeutics actively developing next-generation treatments.
IDEAYA Biosciences Reports Strong Pipeline Progress and $1.1B Cash Position for 2024
• IDEAYA Biosciences advances multiple oncology programs, with over 230 patients enrolled in potential registration-enabling trial for darovasertib in metastatic uveal melanoma, targeting key readouts by end of 2025.
• Company strengthens its position with approximately $1.1 billion cash reserve as of December 2024, expected to fund operations into at least 2028.
• Pipeline expansion includes progress on DLL3 TOP1i ADC IDE849 showing 73% response rate in early SCLC data, and advancement of three new candidates targeting solid tumors.
EMA Recommends Approval for New Cancer Treatments and Vaccines
• The European Medicines Agency (EMA) has recommended eight new products for EU-wide approval, expanding treatment options for various conditions.
• Several cancer treatments have received positive recommendations, potentially offering new hope for patients with different types of malignancies.
• A new antiparasitic combination has been endorsed for use in non-EU markets, addressing a critical need in regions affected by parasitic infections.
• The EMA is also reviewing new safety information regarding Leqembi, an Alzheimer's disease treatment, ensuring ongoing monitoring of its benefit-risk profile.
PTC Therapeutics Announces Key Clinical and Regulatory Milestones for 2025
• PTC Therapeutics anticipates a CHMP opinion on sepiapterin's MAA in Q2 2025, marking a crucial step toward potential European approval for PKU treatment.
• The FDA is expected to decide on sepiapterin's NDA by July 29, 2025, potentially bringing a new therapeutic option to phenylketonuria patients in the US.
• Phase 2 results from the PIVOT-HD study of PTC518 in Huntington's Disease are anticipated in Q2 2025, offering hope for a novel treatment approach.
• Vatiquinone's NDA is expected to be accepted in Q1 2025, with potential regulatory approval in the second half of the year, expanding PTC's pipeline.
FDA Accepts PTC Therapeutics' NDA for Sepiapterin to Treat PKU
PTC Therapeutics, Inc. has announced the FDA's acceptance of the New Drug Application (NDA) for sepiapterin, aimed at treating pediatric and adult patients with phenylketonuria (PKU). This marks a significant step towards providing a new treatment option for PKU patients, based on promising clinical trial results.
FDA Grants Priority Review to Vatiquinone for Friedreich's Ataxia Treatment
• The FDA has accepted PTC Therapeutics' NDA for vatiquinone, a potential treatment for Friedreich's ataxia (FA), granting it priority review.
• Vatiquinone aims to address the unmet need for FA patients, particularly children, by targeting energetic and oxidative stress pathways.
• The NDA is supported by data from the MOVE-FA study and long-term studies, showing slowed disease progression and a favorable safety profile.
• The FDA's target action date for a decision on vatiquinone is set for August 19, 2025, marking a significant step toward potential approval.
PTC Therapeutics Halts Utreloxastat Development After Phase 2 ALS Trial Failure
• PTC Therapeutics' Phase 2 CardinALS trial of utreloxastat in amyotrophic lateral sclerosis (ALS) failed to meet its primary endpoint of slowing disease progression.
• The trial did not achieve statistical significance in reducing plasma neurofilament light chain (NfL), a biomarker of neuronal damage, or in secondary efficacy endpoints.
• Despite the setback, analysts maintain a positive outlook on PTC, focusing on the potential of sepiapterin for phenylketonuria (PKU) and Huntington's disease program.
• PTC Therapeutics sold its Rare Pediatric Disease Priority Review Voucher for $150 million following the FDA approval of Kebilidi for AADC deficiency.
FDA Approves PTC Therapeutics' Kebilidi, First Direct-to-Brain Gene Therapy
• The FDA has approved PTC Therapeutics' Kebilidi (eladocagene exuparvovec-tneq) for aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder.
• Kebilidi is the first gene therapy approved by the FDA that is administered directly into the brain, specifically targeting the putamen region.
• Clinical trial data showed that 67% of patients treated with Kebilidi achieved new gross motor milestones within 48 weeks, a significant improvement compared to untreated patients.
• The approval, granted under the accelerated pathway, requires PTC Therapeutics to conduct a confirmatory trial to verify Kebilidi's clinical benefits.
PTC Therapeutics Announces Regulatory Submissions and Revenue Guidance Update
• PTC Therapeutics has submitted three approval applications to the FDA, including sepiapterin for PKU, Translarna for nmDMD, and a BLA for AADC deficiency gene therapy.
• The company anticipates submitting an NDA for vatiquinone to treat Friedreich ataxia in December 2024 and expects topline data for utreloxastat in ALS during Q4 2024.
• PTC Therapeutics updates its full-year revenue guidance to $750-800 million, driven by strong revenue performance in Q3 2024, with total revenues reaching $196.8 million.
• Third-quarter revenues for the DMD franchise were $124 million, including $72 million from Translarna and $52 million from Emflaza, demonstrating continued geographic expansion and patient adherence.
FDA Accepts NDA for PTC Therapeutics' Sepiapterin in Phenylketonuria Treatment
• The FDA has accepted the New Drug Application (NDA) for sepiapterin, a potential treatment for pediatric and adult patients with phenylketonuria (PKU).
• Sepiapterin's NDA is supported by Phase 3 APHENITY trial data, demonstrating a 63% mean reduction in phenylalanine levels in the overall treated population.
• The FDA has set a target regulatory action date of July 29, 2025, for the review of sepiapterin, aligning with a standard review timeline.
• Sepiapterin, an oral formulation, could allow PKU patients to liberalize their diets while maintaining control of phenylalanine levels, addressing a significant unmet need.
PTC Therapeutics' PTC518 Receives FDA Fast Track Designation for Huntington's Disease
• The FDA has granted Fast Track designation to PTC Therapeutics' PTC518 for Huntington's disease, a condition with significant unmet medical needs.
• PTC518, an oral small molecule, aims to reduce the production of mutated Huntingtin protein, which is a key factor in Huntington's disease progression.
• Clinical data from the PIVOT-HD study showed a 43% reduction in mutant Huntingtin protein levels with PTC518, along with favorable clinical effects.
• Fast Track designation will expedite the development and review process, potentially leading to earlier access for patients with Huntington's disease.
FDA Accepts PTC Therapeutics' NDA for Sepiapterin to Treat PKU
PTC Therapeutics, Inc. has announced that the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin, aimed at treating pediatric and adult patients with phenylketonuria (PKU). This marks a significant step towards providing a new treatment option for PKU patients, supported by promising clinical trial data.
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
© Copyright 2025. All Rights Reserved by MedPath