12:00 AM
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Jun 11, 2001
 |  BioCentury  |  Regulation

Xyrem's safety data

Orphan Medical Inc. failed to convince an FDA advisory committee last week that it has demonstrated the safety of its Xyrem gamma hydroxy butyrate (GHB) for the treatment of cataplexy, sudden loss of muscle tone in response to strong emotional reactions in narcolepsy patients. But the company says FDA is probably convinced.

COO William Houghton told BioCentury that the company started work on Xyrem in 1996 as a result of a suggestion from FDA's Office of Orphan Drugs, and "all development was planned absolutely in conjunction with the FDA." Despite this collaboration, significant differences between FDA's and ORPH's understanding of the path to approval have emerged over the last few months.

In March, the agency postponed a planned review of the Xyrem NDA by the Peripheral and Central Nervous System Advisory Committee, citing the company's inability to provide detailed information to support its safety database. Martin Scharf, a physician who has been treating narcolepsy patients with Xyrem for 16 years under a treatment IND, was unable to provide FDA investigators with documentation on many of his patients who were never enrolled in an ORPH-sponsored study.

Houghton said the...

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