EU medicines regulator backs Biogen’s ALS drug

European Medicines Agency

EU medicines regulator backs Biogen’s ALS drug

Feb 23 (Reuters) – Europe’s medicines regulator on Friday recommended granting a marketing authorization to Biogen’s (BIIB.O) drug for the deadly and progressive neurodegenerative disease known as amyotrophic lateral sclerosis (ALS).

The drug, Qalsody, received accelerated approved in the United States in April last year, based on preliminary data that showed it reduced levels of a neurofilament protein that scientists believe is tied with progression of the disease.
 
The same 108-patient trial also formed the basis for the EU recommendation. However, Biogen still needs to confirm the drug’s benefit in a post-approval trial, both US and EU regulators have said.
 
ALS is a rare neurological disease that can affect nerve cells in the brain and spinal cord responsible for muscle movements, leading to progressive paralysis and death.
 
The drug targets a rare form of ALS that is caused by a genetic mutation that leads to the accumulation of toxic levels of the SOD1 protein. The European Medicines Agency (EMA) estimates that this genetic mutation affects about 2% of people with ALS.