Jazz Pharmaceuticals plc announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Exclusivity (ODE) for Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution, for the treatment of idiopathic hypersomnia in adults, making it the second ODE for the medication following the exclusivity granted in the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy.
As Jazz was the first sponsor to obtain FDA approval for idiopathic hypersomnia, Xywav will have seven-year market exclusivity for this indication from its FDA approval on August 12, 2021. The FDA’s Orphan Drug Designation program is designed to advance the development of drugs that treat a condition affecting 200,000 or fewer U.S. patients annually.
“Prior to the approval of Xywav no treatments were approved for people living with this debilitating and unique sleep disorder, so we are very proud of how we advanced the medicine from concept to commercial availability and are encouraged that FDA has recognized Xywav’s impact by granting ODE for the treatment of idiopathic hypersomnia,” said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development and chief medical officer of Jazz Pharmaceuticals. “We believe FDA’s decision also recognizes the importance of Jazz’s commitment to developing differentiated new medicines where others have not to benefit people with limited treatment options.”
Xywav for idiopathic hypersomnia was approved with multiple dosing options, offering healthcare providers the opportunity to individualize based on the patient need. The majority of clinical trial participants found optimal doses on a twice a night regimen. Idiopathic hypersomnia is characterized by chronic excessive daytime sleepiness, which is the inability to stay awake and alert during the day resulting in the irrepressible need to sleep or unplanned lapses into sleep or drowsiness. Core symptoms may include confusion, irritability and severe sleep inertia or sleep drunkenness (prolonged difficulty in waking up with frequent reentries into sleep).1,2,3,4
In June 2021, FDA recognized seven years of ODE for Xywav for the treatment of cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy. FDA also published its summary of clinical superiority findings for Xywav for the treatment of cataplexy or EDS associated with narcolepsy, stating that “the active moiety, oxybate, was previously approved as Xyrem® (sodium oxybate) for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy.” According to FDA, “Xywav (calcium, magnesium, potassium, and sodium oxybates) is clinically superior to Xyrem by means of greater safety because Xywav provides a greatly reduced chronic sodium burden compared to Xyrem.” The FDA’s summary also stated that “the differences in the sodium content of the two products at the recommended doses will be clinically meaningful in reducing cardiovascular morbidity in a substantial proportion of patients for whom the drug is indicated.”
- Billiard M, Sonka K. Idiopathic hypersomnia. Sleep Med Rev. 2016;29:23-33. doi:10.1016/j.smrv.2015.08.007.
- Trotti LM. Idiopathic hypersomnia. Sleep Med Clin. 2017;12(3):331-344. doi:10.1016/j.jsmc.2017.03.009.
- American Academy of Sleep Medicine. The International Classification of Sleep Disorders. Third Edition (ICSD-3). 2014.
- Khan Z, Trotti LM. Central disorders of hypersomnolence: focus on the narcolepsies and idiopathic hypersomnia. Chest. 2015 Jul;148(1):262-273. doi: 10.1378/chest.14-1304.
Source: Jazz Pharmaceuticals