Chugai files for Japan approval of risdiplam in SMA

16 October 2020

Japanese drugmaker Chugai Pharmaceutical (TYO: 4519) says it has filed a new drug application with the Ministry of Health, Labor and Welfare (MHLW) for risdiplam, an oral survival motor neuron-2 (SMN2) splicing modifier, for the treatment of spinal muscular atrophy (SMA).

Risdiplam received orphan drug designation from the MHLW on Mar 27, 2019, and the application is subject to a priority review.

Analysts have forecast that risdiplam could bring in up to $2 billion global sales at peak, partly due to its patient-friendly oral formulation, differentiating it from rival options in the SMA space, one of which is a gene therapies.

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