Drugs / Sepiapterin
Sepiapterin
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed phenylketonuria 3 milestones | |||||
| Filed | US (FDA) | — | phenylketonuria | 2024-10-01 | “PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin” ptcbio.com ↗ “PTC submitted an NDA to the FDA for sepiapterin for the treatment of PKU. The file was accepted for review” ptcbio.com ↗ |
| Filed | US (FDA) | — | phenylketonuria | 2024-07-30 | “PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the sepiapterin NDA to the U.S. FDA.” ptcbio.com ↗ |
| Filed | EU (EMA) | — | phenylketonuria | 2024-03-28 | “PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the sepiapterin MAA to the European Medicines Agency.” ptcbio.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | US (FDA) | SEPHIENCE | — | 2025-07-28 | fda.gov ↗ |
| Approved | EU (EMA) | Sephience | — | 2025-06-19 | europa.eu ↗ |
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT03519711 | Jan 2019 → Oct 2020 | tetrahydrobiopterin (BH4)-deficient hyperphenylalaninemia | PTC Therapeutics | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT03712124 | Feb → Sep 2019 | gastroparesis | PTC Therapeutics | Completed | Mixed |
| Phase 33 trials · 1 met primary | ||||||
| Phase 3 | NCT06302348 | Mar 2024 → Feb 2031 expected | phenylketonuria | PTC Therapeutics | Recruiting | No outcome recorded |
| Phase 3 | NCT05166161 | Feb 2022 → Jun 2026 overdue | phenylketonuria | PTC Therapeutics | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05099640 | Sep 2021 → Apr 2023 | phenylketonuria | PTC Therapeutics | Completed | Met primary |
News releases announcing trial results or a regulatory action · 14
| Date | Issuer | Release |
|---|---|---|
| 2026-08-24 | PTC Therapeutics | Results PTC Therapeutics Announces New Sephience™ (sepiapterin) Data Reinforcing Broad Patient Benefits Including Sustained Metabolic Control and Diet Liberalization as well as Consistent Favorable Safety Profile ptcbio.com ↗
In addition, new data to be presented at the PTC symposium show treatment with Sephience led to a large number of responsive participants achieving normalization of blood Phe levels (<120 µmol/L) in a rapid timeframe, including those with classical or non-BH4-responsive PKU. |
| 2025-12-22 | PTC Therapeutics | Regulatory PTC Therapeutics Announces Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) in Japan ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that Sephience™ (sepiapterin) has been approved by the Japanese Ministry of Health, Labor and Welfare (MHLW) for the treatment of children and adults living with phenylketonuria (PKU). |
| 2025-07-28 | PTC Therapeutics | Regulatory PTC Therapeutics Announces FDA Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). |
| 2025-06-23 | PTC Therapeutics | Regulatory Sephience™ (sepiapterin) Granted Marketing Authorization by the European Commission for the Treatment of Children and Adults Living with Phenylketonuria (PKU) ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Sephience™ (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). |
| 2025-04-25 | PTC Therapeutics | Regulatory PTC Therapeutics Receives Positive CHMP Opinion for Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). |
| 2025-03-20 | PTC Therapeutics | Results PTC Therapeutics Presents New Sepiapterin Data from Ongoing Studies ptcbio.com ↗
Over 97% of subjects participating in the Phe tolerance protocol of the APHENITY open-label extension study demonstrate the ability to liberalize their diet while on sepiapterin treatment, with a mean increase in protein intake of 126% |
| 2024-10-14 | PTC Therapeutics | Regulatory PTC Therapeutics Announces FDA Target Regulatory Action Date for Sepiapterin and Lancet Publication of Phase 3 Trial Results ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has set a target regulatory action date of July 29, 2025, for the review of the New Drug Application (NDA) for sepiapterin, consistent with a standard review timeline. |
| 2024-10-01 | PTC Therapeutics | Regulatory PTC Therapeutics Announces FDA Acceptance for Filing of NDA for Sepiapterin for the Treatment of Pediatric and Adult Phenylketonuria Patients ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria (PKU). |
| 2024-07-30 | PTC Therapeutics | Regulatory PTC Therapeutics Announces Sepiapterin NDA Submission to FDA ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the sepiapterin NDA to the U.S. FDA. |
| 2024-05-28 | PTC Therapeutics | Regulatory PTC Therapeutics Announces Validation of Sepiapterin European MAA ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA. |
| 2024-03-28 | PTC Therapeutics | Regulatory PTC Announces Submission of Sepiapterin MAA for Treatment of PKU to EMA ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the sepiapterin MAA to the European Medicines Agency. |
| 2023-08-28 | PTC Therapeutics | Results PTC Therapeutics to Present Recent Data for Sepiapterin at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium ptcbio.com ↗
Phe tolerance results continued to demonstrate that sepiapterin treated patients maintain mean blood Phe control (>360 µmol/L) while significantly increasing protein intake, consistent with previous results. |
| 2023-05-17 | PTC Therapeutics | Results PTC Therapeutics Announces APHENITY Trial Achieved Primary Endpoint with Sepiapterin in PKU Patients ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the primary endpoint was achieved in the APHENITY, Phase 3 registration-directed clinical trial of sepiapterin in adult and pediatric patients with phenylketonuria (PKU). |
| 2021-05-26 | PTC Therapeutics | Regulatory PTC Therapeutics Announces Orphan Drug Designations of PTC923 for the Treatment of Hyperphenylalaninemia ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that both the United States Food and Drug Administration (FDA) and European Commission (EC) have granted Orphan Drug Designation (ODD) for PTC923 for the treatment of patients with hyperphenylalaninemia. |
All press releases naming this drug 15 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 7 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Sepiapterin | “A Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate the Safety, Pharmacokinetics and Preliminary Efficacy of CNSA-001(Sepiapterin)...” NCT03519711 ↗ |
| Known as | CNSA-001 | “This study has been designed to demonstrate the safety, pharmacokinetics (PK) and preliminary efficacy of PTC923 (CNSA-001) in reducing blood phenylalanine concentrations in...” NCT03519711 ↗ |
| Known as | L-sepiapterin | ChEMBL registry synonym — accepted as the source's own label CHEMBL1255653 ↗ |
| Known as | PTC923 | “Oral treatment with CNSA-001 (sepiapterin, currently known as PTC923) increased BH4 levels in humans in a previous study.” PMID 34176722 ↗ Jun 2021 |
| Known as | Sepiapterina | ChEMBL registry synonym — accepted as the source's own label CHEMBL1255653 ↗ |
| Known as | Sepiapterine | ChEMBL registry synonym — accepted as the source's own label CHEMBL1255653 ↗ |
| Modality | Small molecule | — CHEMBL1255653 ↗ |
| Route | Oral | “administered orally” NCT03519711 ↗ |