The US Food and Drug Administration's (FDA) Peripheral and Central Nervous System Drugs Advisory Committee (Advisory Committee) voted overwhelmingly to recommend the approval of Vanda Pharmaceuticals' New Drug Application (NDA) for tasimelteon, proposed tradename Hetlioz, for the treatment of Non-24-Hour Disorder (Non-24) in the totally blind.

The advisory panel found that: Non-24 is an appropriate indication for an FDA-approved therapy; the clinical endpoints are appropriate to support the indication; there is substantial evidence of tasimelteon efficacy in Non-24; and and the safety of tasimelteon in Non-24 has been adequately addressed.

"We are extremely pleased that the FDA's advisory committee has recommended that the FDA approve Hetlioz for the treatment of Non-24 in the totally blind," said Mihael H Polymeropoulos, Vanda's president and chief executive officer. "We are now one step closer toward our goal of providing a treatment option that addresses the physiologic cause of this serious, debilitating orphan condition that impacts a majority of totally blind individuals."

Vanda's tasimelteon NDA is currently under Priority Review by the FDA for the treatment of Non-24 in the totally blind, with an action target date under the Prescription Drug User Fee Act (PDUFA-V) of January 31, 2014.  

The FDA grants Priority Review status for a "drug that treats a serious condition and, if approved, would provide a significant improvement in safety or effectiveness" over current therapies. Currently, there is no approved treatment for Non-24 and Hetlioz has the potential to address this unmet medical need.

Non-24 is a serious, rare, and chronic circadian rhythm disorder characterized by the inability to entrain (synchronize) the master body clock with the 24-hour day-night cycle.  Non-24 affects a majority of totally blind individuals, or between 65,000 and 95,000 people in the US Non-24 occurs almost entirely in individuals who lack the light sensitivity necessary to entrain the master body clock in the brain with the 24-hour day-night cycle. Most people have a master body clock that naturally runs longer than 24-hours and light is the primary environmental cue that resets it to 24 hours each day. Individuals with Non-24 have a master body clock that is not reset, and continually delays, resulting in prolonged periods of misalignment between their circadian rhythms and the 24-hour day-night cycle, including the timing of melatonin and cortisol secretion. As a result of this misalignment, Non-24 is associated with significant disruption of the sleep-wake cycle and impairments in social and occupational functioning, and marked subjective distress. Currently there is no approved treatment for Non-24. For more information on Non-24, please visit www.Non-24.com.

Tasimelteon, proposed tradename Hetlioz, is a circadian regulator in development for the treatment of Non-24.  Tasimelteon is a dual melatonin receptor agonist (DMRA) with selective agonist activity at the MT1 and MT2 receptors. Tasimelteon aims to reset the master body clock in the suprachiasmatic nucleus (SCN), resulting in the entrainment of the body's melatonin and cortisol rhythms to align to the 24-hour day-night cycle. The patent claiming Tasimelteon as a new chemical entity extends through December 2022, assuming a 5-year extension to be granted under the Hatch-Waxman Act. Tasimelteon has been granted orphan drug designation for the treatment of Non-24 from both the US and the European Union. Tasimelteon has not been approved by the FDA or any other regulatory authority.

Varmaceuticals Inc. is a biopharmaceutical company focused on the development and commercialization of products for the treatment of central nervous system disorders.