Orphan Status Granted to GW's Cannabis Drug - Drug Discovery and Development

Epidiolex, a cannabis-based drug designed to treat epilepsy made by GW Pharmaceuticals, was granted orphan status by the Food and Drug Administration (FDA).

The designation was given based on the drug’s potential viability in treating a rare genetic disorder called tuberculosis sclerosis complex (TSC), which can induce epilepsy in an estimated 80 to 90 percent of patients diagnosed with it, reported Reuters. An approximate 50,000 people in the U.S. have TSC with nearly 1 million patients diagnosed with this disease worldwide based on statistics published on the TSC Alliance’s homepage.

Currently, GW is in the middle of a Phase 3 study assessing Epidiolex’s impact on this TSC.

TSC marks the third indication GW is pursuing. The firm announced last month Epidiolex successfully lowered the rate of convulsive seizures brought on by external factors like high fever in patients suffering from another rare disorder called Dravet syndrome.

Orphan drug status provides GW with incentives like seven-year marketing exclusivity and expedited reviews as it seeks to prove the therapeutic benefits of drugs derived from cannabis, but the journey to regulatory approval could have some obstacles.

The Associated Press reported earlier this week that medical marijuana activists are wary that FDA approval “will mark the beginning of Big Pharma’s takeover of the marijuana plant, undercutting patients’ ability to treat themselves as they see fit.”

GW’s stock was up 0.74 percent in early morning trading.

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