sodium phenylbutyrate
Commercial- Indications
- carbamoyl-phosphate synthetase 1 deficiency (CPS1D), ornithine transcarbamylase deficiency (OTCD), argininosuccinate synthetase deficiency / citrullinemia type I (ASS deficiency)
Company intelligence / Profile preview
Ucyclyd Pharma developed and commercialized therapies for rare metabolic diseases, notably BUPHENYL® (sodium phenylbutyrate) for urea cycle disorders. The company’s assets—including rights to BUPHENYL—were acquired by Hyperion Therapeutics. Its investigational product Ravicti™ (glycerol phenylbutyrate) was also developed in collaboration with Hyperion for orphan indications including urea cycle disorders and hepatic encephalopathy. Clinical studies demonstrated non-inferiority of Ravicti compared to standard therapy in controlling blood ammonia levels in adults with UCD[4].
2 more assets in the full pipeline.
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