Ultragenyx's (RARE) NDA For LC-FAOD Drug Accepted By FDA
Zacks Investment Research | Oct 14, 2019 10:57PM ET
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced that the FDA has accepted for review the New Drug Application (NDA) for pipeline candidate UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD).
The FDA has assigned a standard review designation to the NDA and set a Prescription Drug User Fee Act (PDUFA) target date of Jul 31, 2020.
The NDA filing is supported by a comprehensive package of data, comprising results from a company-sponsored phase II study of UX007 in 29 patients and a long-term safety and efficacy extension study in 75 patients, including 20 patients who were previously naïve to UX007. The cumulative data also includes a retrospective medical record review of 20 original compassionate use patients, 67 patients treated through expanded access and a randomized, controlled, investigator-sponsored study of 32 patients showing an effect on cardiac function.
The agency indicated that it is currently not planning to hold an advisory committee meeting to discuss the application.
UX007, an investigational therapy, directly addresses the deficiencies in LC-FAOD by providing patients with an alternative energy source that can be metabolized to increase intermediate substrates in the Krebs cycle, a key energy-generating process.
We note that LC-FAOD is a set of rare metabolic diseases that prevent the conversion of fat into energy and can cause low blood sugar, muscle rupture, and heart and liver diseases.
A potential approval of the drug will broaden the company’s portfolio. Shares of the company have lost 4.2% year to date compared with the Zacks Investment Research
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