European Medicines Agency (EMA) has granted orphan medicinal product designation to reldesemtiv for the potential treatment of spinal muscular atrophy (SMA)

The 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.

”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.”

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Two Studies Show Addition of Reldesemtiv to SMN-Directed Treatment Increases Muscle Force of Spinal Muscular Atrophy

In these studies, the addition of reldesemtiv to either nusinersen or SMN-C1 resulted in a leftward shift of the force-frequency curve, indicating an increase in calcium sensitivity of the muscle at submaximal stimulation frequencies and confirming the efficacy of fast skeletal muscle activation in muscle in conjunction with SMN upregulators. Augmentation of muscle force at submaximal stimulation frequencies may be relevant to activities such as breathing and walking. These data suggest that treatment with reldesemtiv in combination with an SMN upregulating therapy such as nusinersen or SMN-C1, may complement and further improve muscle function in SMA.

Nusinersen is an approved anti-sense oligonucleotide therapy for SMA that increases production of Survival Motor Neuron (SMN) protein. SMN-C1 is a small molecule therapy that increases SMN protein expression, similar to risdiplam, an investigational treatment in development for SMA.

Publicerat den: 25 jun, 2019
Uppdaterad den: 28 jun, 2019