Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Phenoptin · 1 trial · 2 indications
Baseline blood phenylalanine(Phe) value is the latest measurement taken prior to initiation of Phenoptin treatment. The ideal range for blood Phe levels is approximately 120-360 µmol/L.
Baseline blood phenylalanine(Phe) value is the latest measurement taken prior to initiation of Phenoptin treatment. The objective of this outcome was to compare to Phe levels achieved using previous treatment regimens.
| Arm | Type | Description |
|---|---|---|
| Single Arm on Active Drug | EXPERIMENTAL | 5mg/kg/day orally, dose may be adjusted to between 5-20 mg/kg/day by investigator at week 6 to control blood Phe levels Outcomes were also evaluated by the subject's type of BH4 deficiency either defects in the genes encoding the enzymes involved in biosynthesis or defects in the genes encoding the enzymes involved in recycling. |
| Name | Type | Description |
|---|---|---|
| Phenoptin | DRUG | 5mg/kg/day orally, dose may be adjusted to between 5-20 mg/kg/day by investigator at week 6 to control blood Phe levels |
Inclusion Criteria: * Documented history of blood Phe level \> 180 µmol/L on at least one occasion * Established diagnosis of hyperphenylalaninemia (HPA) due to primary BH4 deficiency with a documented defect in biopterin metabolism with blood or urine tests * Willing and able to provide written in...
Phenoptin is an investigational small molecule being developed for tetrahydrobiopterin deficiencies, a rare disease. It is studied in patients with hyperphenylalaninemia due to BH4 deficiency, a condition also referred to as non-phenylketonuric hyperphenylalaninemia. The drug is not approved and remains in clinical development.
Phenoptin is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker BMRN. BioMarin is conducting clinical research on Phenoptin for the treatment of tetrahydrobiopterin deficiencies.
Phenoptin is in Phase 2 clinical development. It has completed one Phase 2 trial, and no active trials are currently listed. The drug is investigational and has not been approved by regulatory authorities.
Phenoptin has been studied in one completed Phase 2 clinical trial, identified as NCT00355264. This trial evaluated the safety and efficacy of Phenoptin in subjects with hyperphenylalaninemia due to BH4 deficiency. The study enrolled 12 participants and was conducted in the United States and Germany.
Phenoptin is a separate investigational drug from Kuvan, which is also developed by BioMarin. While both are small molecules related to tetrahydrobiopterin pathways, Phenoptin is specifically being studied for tetrahydrobiopterin deficiencies and has a distinct clinical development program.