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Therapy for obstructive hypertrophic cardiomyopathy receives orphan drug designation

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MyoKardia Inc. announced that the FDA has granted an orphan drug designation to its novel drug for treatment of symptomatic obstructive hypertrophic cardiomyopathy.

The drug (MYK-461) is the first therapy developed to target the underlying cause of hypertrophic cardiomyopathy; there is no FDA-approved therapeutic product for the disease, according to a press release from the company.

In phase 1 studies of healthy volunteers and patients with hypertrophic cardiomyopathy, the drug has shown the ability to reduce excessive cardiac muscle contractility, a biomarker of hypertrophic cardiomyopathy, which could indicate its ability to modulate cardiac myosin, the company stated in the release.

In addition, the drug has been well tolerated in early studies, according to the release.

The FDA’s Office of Orphan Products Development designates orphan status for drugs to treat underserved populations or rare disorders affecting fewer than 200,000 people in the United States.

As part of the designation, MyoKardia will receive market exclusivity if the drug is approved, waiver of FDA application fees, and tax credits for clinical trials that meet certain qualifications, according to the release.