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Ultragenyx wins early approval for rare disease drug

July 1, 2020

Patients with long-chain fatty acid oxidation disorders suffer from severe drops in glucose because their bodies can’t properly convert these molecules into energy. Dojolvi is designed to correct the imbalance, providing fatty acids for energy and replacing metabolites.

The genetic condition affects between 2,000 and 3,500 children and adults in the U.S. and as many as 14,000 patients in the developed world, according to estimates compiled by Ultragenyx. While infants are often screened for the disease, there hasn’t been an approved medication until now. Without treatment, patients with the condition face a range of complications and shortened lives.

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