July 12, 2017

Biohaven receives FDA's orphan drug designation for Rett syndrome treatment

New Haven-based Biohaven Pharmaceutical Holding Co. Ltd. said its investigational compound has been granted orphan drug designation by the U.S. Food and Drug Administration to treat patients with Rett syndrome, for which there is no approved drug therapy available.

Rett syndrome is a rare and severe genetic neurodevelopmental disorder mostly affecting females. Symptoms emerge from 6 to 18 months of age after apparently normal postnatal development. Patients with Rett syndrome develop conditions that include slowing of psychomotor function and loss of acquired cognitive skills, such as language and purposeful hand movement. Patients may survive into adulthood, yet they are severely physically and cognitively impaired.

Biohaven's investigational compound is called BHV-5000.

Potential benefits of orphan drug designation include tax credits for 50 percent of clinical trial costs, waiver of marketing application user fees, which is estimated to save more than $2 million and seven years of marketing exclusivity if regulatory approval is ultimately received.

"Patients with Rett syndrome develop debilitating symptoms," said Dr. Robert Berman, chief medical officer of Biohaven. "Preclinical studies on BHV-5000 have demonstrated benefits in transgenic mouse models of Rett syndrome, particularly on symptoms of abnormal breathing. We are eager to test BHV-5000 in patients with Rett syndrome to understand if these preclinical effects translate into therapeutic benefits for patients."

Biohaven expects to initiate a Phase 1 trial of BHV-5000 in healthy volunteers with a commercially ready formulation this year and then proceed to a study in patients with Rett syndrome in 2018.

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