Gamma-Hydroxybutyrate (orphan medical).

Area of Science:

• Pharmacology
• Neurology
• Regulatory Affairs

Background:

• Gamma-hydroxybutyrate (GHB) is a naturally occurring substance with prior use in narcolepsy treatment.
• GHB was classified as a Schedule I controlled substance, necessitating legislative changes for medical use.
• Orphan Medical is pursuing FDA approval for GHB (Xyrem) for narcolepsy.

Purpose of the Study:

• To evaluate the development and regulatory status of gamma-hydroxybutyrate (Xyrem) for the treatment of narcolepsy.
• To track the progress of Orphan Medical's New Drug Application (NDA) and FDA interactions.
• To document the legislative changes impacting the medical use and scheduling of GHB.

Main Methods:

• Review of regulatory filings, including the New Drug Application (NDA) for Xyrem.
• Monitoring of FDA communications, such as the approvable letter and data requests.
• Tracking of legislative actions concerning the medical use and classification of gamma-hydroxybutyrate.

Main Results:

• An NDA for Xyrem was filed in October 2000, and an approvable letter was received in July 2001.
• Orphan Medical committed to completing required trials, including one in respiratory-compromised patients, by end of 2001.
• Legislative changes reclassified medically formulated GHB from Schedule I to Schedule III, facilitating its development.

Conclusions:

• The development of Xyrem for narcolepsy faced regulatory and clinical trial requirements.
• Successful navigation of these requirements was anticipated for a mid-2002 launch.
• Legal reclassification of GHB supported its continued medical development.