Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication KUVAN ® is indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN is to be used in conjunction with a Phe-restricted diet. FDA label
Also known as Sapropterin dihydrochloride, Kuvan, BH4, 6R BH4
Phenoptin · 2 trials · 3 indications
Phe tolerance was defined as the amount of dietary Phe prescribed (milligram per kilogram per day \[mg/kg/day\]) while maintaining blood Phe levels within the selected therapeutic target range (defined as greater than or equal to \[\>=\] 120 to less than \[\<\] 360 micromoles per liter \[mcmol/L\]).
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 |
|---|---|---|
| Kuvan® + Phe-restricted diet | EXPERIMENTAL | Subjects will be treated with Kuvan® tablets once daily along with Phe-restricted diet therapy. |
| Phe-restricted diet alone | OTHER | Subjects will follow a Phe-restricted diet alone. |
| 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 |
|---|---|---|
| Kuvan® | DRUG | Kuvan® (sapropterin dihydrochloride) tablets will be administered orally at the dose of 10 mg/kg/day and will be escalated to 20 mg/kg/day if after 4 weeks a subject's Phe tolerance is not increased by at least 20% versus baseline. |
| Phenylalanine (Phe)-restricted diet | OTHER | Phe intake will be adjusted every 2 weeks, based on the mean Phe levels of the previous 2 weeks using pre-defined Phe adjustment criteria. |
| 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: * Male or female PKU infants and young children less than (\<) 4 years of age at the scheduled Day 1 visit of the 26-week study period (taking into consideration the maximum of 21 days in the screening period) * Confirmed clinical and biochemical PKU, including at least two prev...
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.