By Colin Kellaher


Roche Holding AG and PTC Therapeutics Inc. on Tuesday said the European Commission approved Evrysdi for the treatment of 5q spinal muscular atrophy, or SMA, in patients two months of age and older.

The companies said Evrysdi is approved for patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies, adding that the drug is the first and only at-home treatment approved for the disease.

SMA, a severe, progressive neuromuscular disease, is a leading genetic cause of death in infants, and 5q SMA is the most common form of the disease, Roche said.

Swiss pharmaceutical company Roche leads the clinical development of Evrysdi as part of a collaboration with PTC, a South Plainfield, N.J., pharmaceutical company, and the SMA Foundation.


Write to Colin Kellaher at


(END) Dow Jones Newswires

March 30, 2021 06:41 ET (10:41 GMT)

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