Roche, PTC Therapeutics Gets European OK of Evrysdi in Spinal Muscular Atrophy
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
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
Write to Colin Kellaher at email@example.com
(END) Dow Jones Newswires
March 30, 2021 06:41 ET (10:41 GMT)
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