FDA grants a second approval to Ultragenyx drug for rare disease

The Food and Drug Administration on Thursday approved Ultragenyx Pharmaceutical Inc.’s RARE, 4.77% Crysvita as a treatment for some patients with tumor-induced osteomalacia (TIO), a rare disease that affects between 500 and 1,000 people in the U.S. Crysvita was first approved by the FDA in 2018 for a rare form of rickets. The drug generated net revenue of $103.7 million in 2019. “While the expansion of Crysvita’s label into TIO …is a smaller indication, which may only incrementally increase sales, it further demonstrates RARE’s ability to get programs across the finish line, providing more credibility to the mgmt. team,” SVB Leerink’s Joseph Schwartz wrote in a note to investors on Thursday. A spokesperson for Ultragenyx said the drug costs $160,000 per year for children and $200,000 per year for adults. Ultragenyx’s stock has gained 69.7% year-to-date, while the S&P 500 SPX, -0.35% is down 3.5% since the start of the year.
https://www.marketwatch.com/story/fda-grants-a-second-approval-to-ultragenyx-drug-for-rare-disease-2020-06-19