FDA Accepts Xyrem sNDA for Review
The FDA has accepted for filing Orphan Medical's supplemental new drug application (sNDA) for Xyrem oral solution.
Xyrem (sodium oxybate) is currently marketed as the first and only approved treatment for cataplexy, a sudden loss of muscle tone, a debilitating symptom of narcolepsy,the company said. The sNDA is expected to expand the Xyrem label to include the reduction of excessive daytime sleepiness (EDS) and the improvement in fragmented nighttime sleep in narcolepsy patients.
With approval of the sNDA, Xyrem would be the first medication approved to treat the primary symptoms of narcolepsy, the company said. Prior to approval of Xyrem, antidepressants were used off-label to treat cataplexy. EDS has been treated with stimulant medications, and disrupted nighttime sleep by sedative-hypnotic medications.
The sNDA includes two Phase IIIb trials with EDS as the primary efficacy measure, as well as positive data relating to the treatment of other components of narcolepsy. The sNDA was the company's first submission in the electronic common technical document format now required by the FDA. The FDA is expected to grant orphan-drug status to the new indication when approved under the sNDA.
Orphan Medical also has received a milestone fee of $1 million from UCB Pharma tied to submission of the sNDA in the U.S.