Cytokinetics Granted European Orphan Designation for Reldesemtiv to Treat Spinal Musclar Atrophy

Cytokinetics (NASDAQ:CYTK) has announced that the European Medicines Agency (EMA) has granted the company orphan medicinal product designation to reldesemtiv to treat spinal musclar atrophy (SMA).

As quoted in the press release:

he U.S. Food and Drug Administration previously granted orphan drug designation to reldesemtiv for the potential treatment of SMA in 2017. In collaboration with Astellas, Cytokinetics is developing reldesemtiv, a fast skeletal muscle troponin activator (FSTA), as a potential treatment for people with SMA and certain other debilitating diseases and conditions associated with skeletal muscle weakness and/or fatigue.

Orphan medicinal product designation is adopted by the European Commission based on an opinion by the EMA’s Committee for Orphan Medicinal Products (COMP). Orphan medicinal product designation is granted by the EMA to medicines intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating disease affecting fewer than 5 in 10,000 persons in the European Union, or for which it is unlikely that the costs associated with the development and commercialization of the medicine would be recovered by expected sales under normal market conditions without the incentives provided by the designation. The designation offers potential incentives, which may include a ten-year period of EU marketing exclusivity from the date of marketing authorization, EU-funded research, protocol assistance and fee reductions.

“We’re pleased that reldesemtiv received orphan designation from the European Commission,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “Despite advances with SMN-directed treatments, residual muscle impairment and weakness are expected to present continuing challenges for patients with SMA. Treatment with reldesemtiv may represent an additive and complementary approach to increase muscle function.”

Click here to read the full press release.