Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Sepiapterin · 1 trial · 1 indication
Analysis for participants ≥30 months to \<6 years of age.
Analysis for participants ≥6 years to 16 years of age.
| Arm | Type | Description |
|---|---|---|
| Sepiapterin | EXPERIMENTAL | Participants will receive age- and weight-adjusted doses of sepiapterin orally once daily for up to 6 years. |
| Name | Type | Description |
|---|---|---|
| Sepiapterin | DRUG | Sepiapterin powder for oral use will be mixed in water or apple juice prior to administration. |
Key Inclusion Criteria: For all participants: * Women of childbearing potential must have a negative pregnancy test at Screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for at least 90 days after the last dose of ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | BMRN | 5 | PHASE3 | Pegvaliase |
| PTC Therapeutics, Inc. | PTCT | 2 | PHASE3 | PTC923 |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR444836 |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE1 | AG-181 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
| Q32 Bio Inc | QTTB | 1 | - | HMI-102 |
Sepiapterin is an investigational small molecule being developed for the treatment of phenylketonuria (PKU), a metabolic disorder. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.
Sepiapterin is being developed by PTC Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol PTCT. The company is conducting a Phase 3 clinical trial to evaluate the drug in patients with phenylketonuria.
Sepiapterin is currently in Phase 3 clinical development. It is an investigational drug and has not been approved for any use. The ongoing Phase 3 study is actively recruiting participants with phenylketonuria.
Sepiapterin is being evaluated in a Phase 3 clinical trial registered as NCT06302348, titled "A Study of Sepiapterin in Participants With Phenylketonuria (PKU)." The trial is currently recruiting and aims to enroll 56 participants across the United States, Australia, France, Ireland, Poland, and the United Kingdom.
Sepiapterin is the primary name used for this investigational drug in clinical development. No alternative names have been reported for this compound in the available clinical trial information.