Sapropterin
- Generic Name
- Sapropterin
- Brand Names
- Javygtor, Kuvan, Sapropterin Dipharma
- Drug Type
- Small Molecule
- Chemical Formula
- C9H15N5O3
- CAS Number
- 62989-33-7
- Unique Ingredient Identifier
- EGX657432I
- Background
Sapropterin (tetrahydrobiopterin or BH4) is a cofactor in the synthesis of nitric oxide. It is also essential in the conversion of phenylalanine to tyrosine by the enzyme phenylalanine-4-hydroxylase; the conversion of tyrosine to L-dopa by the enzyme tyrosine hydroxylase; and conversion of tryptophan to 5-hydroxytryptophan via tryptophan hydroxylase.
- Indication
For the treatment of tetrahydrobiopterin (BH4) deficiency.
- Associated Conditions
- Hyperphenylalaninemia
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.
Synlogic's Novel Probiotic Therapy Shows Promise in Phase 2 PKU Trial, Advancing to Phase 3
• Synlogic's engineered probiotic SYNB1934 demonstrated a significant 34% reduction in phenylalanine levels in phenylketonuria patients during Phase 2 trials, outperforming its predecessor SYNB1618.
• The experimental therapy showed a 42% reduction in plasma phenylalanine among responders, with 60% of patients achieving clinically meaningful improvements in their condition.
• Phase 3 trials for SYNB1934 are scheduled to begin in early 2023, offering potential hope for PKU patients who have limited treatment options with current therapies.
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 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.
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