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Kuvan · 1 trial · 1 indication
Response to Kuvan® (sapropterin dihydrochloride) treatment was defined as a reduction in blood phenylalanine levels of greater than or equal to 30% at Day 8 as compared to baseline.
| Arm | Type | Description |
|---|---|---|
| Kuvan® | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Kuvan® | DRUG | Kuvan® (sapropterin dihydrochloride) will be administered orally at a dose of 20 milligram per kilogram per day (mg/kg/day) once daily for 8 days. If there is 30 percent (%) decrease in blood phenylalanine levels from baseline at the end of Day 8, then treatment will be continued at the same dose for further 6 weeks. |
Inclusion Criteria: * Willing and able to provide written informed consent (for children under 18 years old the parent\[s\]/guardians give informed consent, subjects 14-17 years old give additionally their own written informed consent) * Age of 4 - 18 years, inclusive * Confirmed clinical and bioch...
| 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 used for phenylketonuria, a metabolic disorder. It is a small molecule being developed by Merck & Company, Inc. for this condition. The drug is currently in Phase 3 clinical development and is considered investigational, meaning it has not been approved by regulatory authorities.
Kuvan targets the metabolic pathway involved in phenylketonuria. It is a small molecule designed to address the underlying metabolic defect in this condition. The drug works by modulating the body's ability to process phenylalanine, an amino acid that accumulates in patients with phenylketonuria.
Kuvan is being developed by Merck & Company, Inc., a pharmaceutical company traded on the New York Stock Exchange under the ticker symbol MRK. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with phenylketonuria.
Kuvan is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory agencies. The Phase 3 trial for Kuvan has been completed, and the drug is still undergoing clinical evaluation for the treatment of phenylketonuria.
Kuvan has one completed Phase 3 clinical trial with the identifier NCT01732471. This open-label, uncontrolled study evaluated the response and safety of Kuvan in subjects with phenylketonuria. The trial enrolled 90 participants in Germany, with a minimum age of 4 years, and included both males and females.
Kuvan is the brand name for sapropterin dihydrochloride, a synthetic form of tetrahydrobiopterin. It is used to treat phenylketonuria by helping the body break down phenylalanine. The drug is being developed by Merck & Company, Inc. and is currently in Phase 3 clinical trials.