UPDATE 2-EU medicines regulator backs Biogen's ALS drug
(Adds background on disease and trials in paragraphs 3, 5, 8 and 9)
Feb 23 (Reuters) - Europe's medicines regulator on Friday recommended granting a marketing authorization to Biogen's 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.
Currently, generic drug riluzole is the only treatment authorized for ALS in the EU, the EMA said.
The EMA said there is a large unmet medical need for effective therapies that preserve muscle function and prolong the life of patients with ALS.
The company said the European Commission will review the EMA human medicines committee's recommendation and is expected to decide on marketing authorization in second-quarter 2024.
Qalsody brought in $5.9 million in revenue for Biogen in 2023.
Renowned British physicist Stephen Hawking developed ALS when he was in his early twenties and lived on till the age of 76. However, most patients die from the condition within five years, according to U.S. government data. (Reporting by Puyaan Singh and Christy Santhosh in Bengaluru; Editing by Dhanya Ann Thoppil, Shounak Dasgupta and Saumyadeb Chakrabarty)
