Posted by Michael Wonder on 16 Aug 2021
Spinal muscular atrophy: second and third active ingredients undergo benefit assessment
16 August 2021 - With risdiplam there is a hint of an added benefit for spinal muscular atrophy type 1, with onasemnogene abeparvovec an added benefit for no type is proven. Different exclusion criteria in the studies make comparisons difficult.
After nusinersen sodium (Spinraza), risdiplam (Evrysdi) and onasemnogene abeparvovec (Zolgensma) are now approved in Europe for the treatment of patients with 5q associated spinal muscular atrophy.
In two early benefit assessments, the IQWiG has now investigated whether these two active ingredients offer certain groups of patients an additional benefit, especially against nusinersen sodium.
