Roche (RHHBY.US) submits application for marketing authorization of SMA oral therapy "risdiplam tablets" in China.

On September 12, the China National Medical Products Administration Drug Evaluation Center (CDE) announced on its official website that Roche's application for the marketing of risdiplam tablets has been accepted, but the specific indications have not been disclosed. Public information shows that risdiplam is an innovative drug for rare neurological diseases developed by Roche, used to treat Spinal Muscular Atrophy (SMA). The oral solution formulation of the drug has already been approved for marketing in China, while the tablet formulation is currently under application for approval. SMA is one of the autosomal recessive genetic diseases that causes infant and child mortality, and is due to a mutation in the Survival Motor Neuron 1 (SMN1) gene leading to a defect in SMN protein function. Risdiplam is a modifier of SMN2 gene mRNA splicing.