Potential SMA Treatment Granted Orphan Medicinal Product Designation By The European Medicines Agency (EMA)
31 July 2019
Cytokinetics announced that the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation to reldesemtiv for the potential treatment of SMA. Reldesemtiv is a drug that has been shown, in the laboratory, to increase the force generated by skeletal muscle and delay the onset and extent of muscle fatigue. This could be beneficial to people living with SMA or certain other debilitating conditions associated with skeletal muscle weakness and / or fatigue.
Reldesemtiv has already been granted Orphan Drug Designation in the US (click here for more information), which, like Orphan Medicinal Product Designation, incentivises companies to pursue drugs intended to treat rare conditions with small markets, such as SMA, and can help to expedite drug development.
An Orphan Medicinal Product Designation is granted by the European Commission to medicines intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting fewer than 5 in 10,000 people in the European Union, or for which it is unlikely that the costs associated with the development and commercialisation of the medicine would be recovered by expected sales under normal market conditions. The designation offers potential incentives, which may include a ten-year period of EU marketing exclusivity from the date of marketing authorisation, EU-funded research, protocol assistance and fee reductions.