FDA grants orphan drug designation for oral idiopathic hypersomnia treatment

The FDA granted orphan drug exclusivity for JZP258, an oral low-sodium oxybate solution for the treatment of idiopathic hypersomnia in adults, according to Jazz Pharmaceuticals.

"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 [orphan drug exclusivity] for the treatment of idiopathic hypersomnia," Robert Iannone, MD, MSCE, executive vice president, research and development, and chief medical officer of Jazz Pharmaceuticals, said in a company press release.

"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 was approved with the possibility of use with more than one dose, offering medical professionals the leeway to prescribe based on patient need.

According to the FDA’s summary of clinical superiority findings, Xywav possesses a greater safety factor than its predecessor, Xyrem, as the former provides a greatly reduced chronic sodium burden compared with the latter.

The report 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."

Editor's note: On Jan. 7, 2022, this article was updated to clarify that the orphan drug designation applies to treatment of adults with idiopathic hypersomnia. Healio regrets the error.