Zevra has produced a late-stage rare disease clinical pipeline and two partnered commercial products.
Olpruva (sodium phenylbutyrate) is the company's lead candidate and was approved for the treatment of certain UCDs in December 2022. The drug is an oral suspension and is a prescription medicine used along with certain therapies, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).
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