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Ultragenyx wins early approval for rare disease drug

A drug from Ultragenyx Pharmaceutical on Tuesday became the first treatment approved in the U.S. for patients who have long-chain fatty acid oxidation disorders, a group of rare, life-threatening diseases.The Food and Drug Administration cleared the drug, which will be sold as Dojolvi, a month earlier than the July 31 target date that regulators had originally set.The California-based biotech said it expects to make the treatment available to patients in the next 30 days, and that it will work to secure patients’ access to the drug.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.

Like other rare disease medications, Dojolvi comes at a high price, with Ultragenyx setting a price of $4,875 per vial. That translates to an average net price of $138,000 per patient per year, according to investment bank Piper Sandler, which estimates the drug could earn $270 million in sales by fiscal year 2025.

Ultragenyx, which specializes in rare diseases, has now won four U.S. approvals in the last three years, including two in June. Its range of marketed drugs and an emerging research pipeline set the company up well, Piper Sandler analyst Christopher Raymond wrote in a note to investors.

In addition to biologic and small molecule drugs, Ultragenyx is also developing two gene therapies for rare diseases.

The company’s shares were up about 7% Wednesday, trading at roughly $84 apiece. The company’s share price has almost doubled since the start of the year.