Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Kuvan · 1 trial · 1 indication
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 \[mcmol/L\]).
| 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. |
| 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. |
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...
| 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 |
Kuvan is a small molecule used for the treatment of phenylketonuria (PKU), a metabolic disorder. It is being developed by BioMarin Pharmaceutical Inc. (BMRN) and is currently in Phase 3 clinical development for this indication.
Kuvan works by targeting the metabolic pathway involved in phenylketonuria. As a small molecule, it is designed to address the underlying metabolic deficiency in patients with PKU, helping to manage the condition.
Kuvan is developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. The company is advancing Kuvan through clinical trials for the treatment of phenylketonuria.
Kuvan is in Phase 3 clinical development for phenylketonuria. It has completed one Phase 3 trial, and it is not yet approved by the FDA, meaning it remains an investigational drug in clinical development.
Kuvan has been studied in a Phase 3 clinical trial with the identifier NCT01376908, titled 'Kuvan® in Phenylketonuria Patients Less Than 4 Years Old'. This trial, which enrolled 56 patients, has been completed and was conducted across multiple countries including Austria, Belgium, Czechia, Germany, Italy, Netherlands, Slovakia, Turkey, and the United Kingdom.
Kuvan is the brand name for sapropterin dihydrochloride, a synthetic form of tetrahydrobiopterin (BH4). It is used in the treatment of phenylketonuria and is being developed by BioMarin Pharmaceutical Inc.