HQ Team
February 24, 2024: Biogen Inc., a US biotechnology company, has got marketing authorisation from the European medicines regulator for its drug to treat neurodegenerative disease, amyotrophic lateral sclerosis.
The European Medicines Agency’s Committee for Medicinal Products for Human Use has “adopted a positive opinion” recommending a marketing authorisation under exceptional circumstances for Qalsody, according to a company statement.
The regulator’s Committee for Medicinal Products for Human Use (CHMP) assesses applications submitted by medicine developers and recommends whether or not a medicine should be granted marketing authorisation.
The committee is composed of one member and an alternate from each EU Member State, as well as from Iceland and Norway. It also has up to five EU experts in relevant fields such as statistics and quality of medicines, who are nominated by the European Commission.
The Biogen’s drug for adult patients with amyotrophic lateral sclerosis will be the first treatment approved in the EU to target a genetic cause of the disease, also known as motor neuron disease.
‘Totality of evidence’
The regulator’s backing for the drug is based on the “totality of evidence, including the targeted mechanism of action, biomarker and clinical data.”
In the 28-week final stage trials, reductions of 60% in plasma neurofilament light chain were observed in participants who received Qalsody compared to the placebo group, suggesting reduced neuronal injury, according to the statement.
Amyotrophic lateral sclerosis is a fatal motor neuron disease. It is characterized by progressive degeneration of nerve cells in the spinal cord and brain.
The disease affects voluntary control of arms and legs and leads to trouble breathing. As the person’s brain loses connection with the muscles it takes away the ability to walk, talk, eat and finally breathe. ALS does not affect intelligence, thinking, seeing, or hearing, according to Johns Hopkins University.
Every 90 minutes someone is diagnosed with ALS, according to the ALS Association.
Biogen’s drug has reported serious neurological events such as myelitis and radiculitis, papilledema and elevated intracranial pressure, and aseptic meningitis.
A marketing authorization under exceptional circumstances is recommended when the benefit/risk assessment is determined to be positive but due to the rarity of the disease, it is unlikely that large data can be obtained under normal conditions of use.
The regulator’s recommendation for Qalsody will be reviewed in the second quarter of 2024.
