Levi Garraway (Genentech)

Roche re­leas­es more Evrys­di da­ta lead­ing up to FDA de­ci­sion for very young in­fants with SMA

One month away from an FDA de­ci­sion on Evrys­di for in­fants un­der two months old, Roche’s Genen­tech has re­leased new long-term da­ta on its oral drug for spinal mus­cu­lar at­ro­phy (SMA).

The da­ta show that, af­ter three years of treat­ment, 91% of chil­dren who be­gan tak­ing the drug at ages 1-7 months were alive (n=58). In ad­di­tion, of 48 chil­dren eval­u­at­ed, 32 main­tained and four gained the abil­i­ty to sit with­out sup­port.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.