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Cytokinetics granted FDA orphan-drug tag for ALS drug


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Cytokinetics granted FDA orphan-drug tag for ALS drug

Cytokinetics Inc.'s reldesemtiv received the U.S. Food and Drug Administration's orphan drug designation to treat patients with amyotrophic lateral sclerosis.

ALS, also known as motor neuron disease or Lou Gehrig's disease, is a progressive nervous system condition that causes the death of nerve cells that control voluntary muscles. The disease is estimated to affect about 20,000 individuals in the U.S., with about 5,000 new cases diagnosed annually, Cytokinetics said in a Dec. 18 press release. Only about 10% of patients survive for more than a decade after diagnosis, with most deaths caused by respiratory failure due to diminished strength in the skeletal muscles responsible for breathing.

The U.S. FDA's orphan-drug designation encourages companies to develop cures for rare diseases by offering tax reductions, fee exemptions and a license to be the sole sellers of approved treatments for seven years.

Previously, the U.S. regulator and the European Medicines Agency have classified reldesemtiv as an orphan medicinal product for the potential treatment of spinal muscular atrophy, which is a genetic disease characterized by progressive, debilitating muscle weakness.

Cytokinetics in May said reldesemtiv failed to improve lung function in patients with ALS during a mid-stage study.

Reldesemtiv is being developed by Cytokinetics in collaboration with Tokyo-based Astellas Pharma Inc.