WASHINGTON, DC, USA I July 29, 2013 I Vanda Pharmaceuticals Inc. (VANDA) (VNDA) today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing and granted a priority review classification to Vanda’s New Drug Application (NDA) for tasimelteon, a circadian regulator for the treatment of Non-24-Hour Disorder (Non-24) in the totally blind.
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[1]. Currently, there is no approved treatment for Non-24 and tasimelteon has the potential to address this unmet medical need.
“We are extremely pleased that the FDA has granted tasimelteon priority review for the treatment of Non-24 in the totally blind,” said Mihael H. Polymeropoulos M.D., Vanda’s President and Chief Executive Officer. “The agency’s acceptance of the NDA and decision to place tasimelteon in a category of expedited review are important milestones for Vanda as we take another step toward our goal of providing patients with a treatment for Non-24.”
The FDA determined the action target date under Prescription Drug User Fee Act (PDUFA-V), to be January 31, 2014. The FDA has also tentatively scheduled an advisory committee meeting to discuss the tasimelteon application on November 14, 2013.
About Non-24-Hour Disorder
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 U.S. 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.
About Tasimelteon
Tasimelteon 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 U.S. and the European Union. Tasimelteon has not been approved by the FDA or any other regulatory authority.
About Tasimelteon NDA
Summary of Efficacy
The efficacy of tasimelteon as a circadian regulator of the master body clock in the treatment of Non-24 was studied in two randomized, double-masked, placebo-controlled, multicenter, parallel-group trials, SET (Safety and Efficacy of Tasimelteon) and RESET (Randomized withdrawal Study of the Efficacy and safety of Tasimelteon). In the SET study, tasimelteon achieved the primary endpoints of entrainment (synchronizing) of the melatonin rhythm and clinical response as measured by entrainment plus an improvement on the Non-24 Clinical Response Scale. Tasimelteon also demonstrated statistically significant improvement in measures of nighttime sleep, daytime nap duration, timing of sleep, and overall global functioning scale. In patients treated with tasimelteon, daytime naps decreased by 46 minutes per day in the worst 25% of days in a cycle and nighttime sleep increased by 57 minutes per day during the worst 25% of nights in a cycle. The RESET study demonstrated that patients who continued treatment with 20 mg of tasimelteon maintained entrainment of melatonin and cortisol circadian rhythms at statistically significantly greater percentages than patients receiving placebo. Patients treated with tasimelteon also maintained their clinical benefits while patients who received placebo showed significant deterioration in measures of nighttime sleep, daytime naps and timing of sleep.
Summary of Safety
The Integrated Summary of Safety (ISS) for the tasimelteon development program included data from over 1,300 subjects treated with tasimelteon including 111 subjects treated for at least six months and 44 subjects treated for at least one year. Common adverse events (>=2% in tasimelteon and greater than placebo) in placebo controlled studies (tasimelteon n=429, placebo n=203) included, (tasimelteon %, placebo %), Back pain (2.1%, 2.0%), Dreams (vivid or unusual), (2.6%, 0.5%), Diarrhea (2.3%, 1.0%), Dry Mouth (2.3%, 0.5%), Headache (9.6%, 7.4%), Nasopharyngitis (6.5%, 6.4%), Somnolence (3.0%, 1.5%), Upper Respiratory Tract Infection (2.6%, 1.5%). In placebo controlled studies (tasimelteon n=429, placebo n=203), all serious adverse events were deemed unrelated to study drug and the rates between tasimelteon and placebo treated patients were similar (1.6%, 1.5%).
About Vanda Pharmaceuticals Inc.
Vanda Pharmaceuticals Inc. is a biopharmaceutical company focused on the development and commercialization of products for the treatment of central nervous system disorders. For more on Vanda, please visit http://www.vandapharma.com.
SOURCE: Vanda Pharmaceuticals
