Roche announced that FDA grants priority review to risdiplam for spinal muscular atrophy

Wie start the week with a great news!

Roche announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. Risdiplam is designed to increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. The FDA is expected to make a decision on approval by May 24, 2020.

“The FIREFISH and SUNFISH trials were designed to represent the real world spectrum of people living with SMA and include many people previously underrepresented in clinical trials,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We look forward to working closely with the FDA to explore broad access to risdiplam for all individuals in the community who might benefit.”

Framsteg i behandlingen av spinal muskelatrofi – nya fenotypen, nya utmaningar, nya implikationer för vårdsystem

Jag delar gärna en underbar ny artikel från kollegorna från Freiburg / Tyskland som gör så mycket framsteg i SMA forskning

  • The SMA landscape has changed considerably since the first reports more than a century ago of patients with spinal muscular atrophy (SMA) by Werdnig and Hofmann in 1891 [1] and 1893 [2]. Decoding the disease’s genetic background, first in linkage analyses [3, 4] and later by identifying mutations in SMN1 as disease-causing [5], paved the way for targeted medical approaches. In this review we provide an overview of both the latest therapeutic options and emerging therapies for SMA