Hyperion ($HPTX) has won its first FDA approval. The South San Francisco-based company grabbed the agency's nod for its drug Ravicti, a treatment for managing genetic conditions known as urea cyclic disorders.
The approval comes after U.S. regulators delayed their decision last year on Hyperion's application to market the med for UCDs. Triggering enzyme deficiencies, the disease leads to toxic buildups of ammonia in the blood that can lead to brain damage and coma. The agency green-lighted the drug for patients aged two years or older with the disorders, which afflict one in 10,000 births, helping the company garner orphan status for Ravicti.
Hyperion is likely to compete against an existing therapy on the market for combatting UCDs, Buphenyl. The biotech proved that its new med, which is taken orally as a liquid three times a day with meals, was just as effective as the older remedy. Now the company faces inevitable hurdles from payers, which can be stingy on reimbursing for meds unless they show improved benefits for patients.
"Ravicti provides another treatment for chronic management of urea cycle disorders, a group of life-threatening conditions," said Dr. Donna Griebel, M.D., director of the Division of Gastrointestinal and Inborn Errors Products in the FDA's Center for Drug Evaluation and Research, in her agency's release. "The approval of this new therapeutic option demonstrates FDA's commitment to providing treatments for patients suffering from rare diseases."
The company's shares jumped 3.2% to $16.13 per share as of 12:38 p.m. ET today. Its stock price has shot up 58% since its lackluster IPO in July, when the shares hit the public market below the company's hoped-for range.
- here's the FDA's release
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