The European Medicines Agency (EMA) has just approved a new medicine for the treatment of patients with certain genetic forms of Charcot disease.
- The European Medicines Agency (EMA) approves a new medicine, Qalsody, for patients with certain genetic forms of Charcot disease.
- The EMA thus gives the green light for marketing in the European Union.
- This recommendation is then sent to the European Commission, which makes a final decision.
Qalsody is the name of the new medicine approved by the European Medicines Agency (EMA) for patients with certain genetic forms of Charcot disease, also called amyotrophic lateral sclerosis (ALS).
A new drug against Charcot’s disease
This medicine, produced by the American laboratory Biogen, is used “for the treatment of adults with ALS who have a mutation in the superoxide dismutase 1 (SOD1) gene“, explains the EMA in a communicated published this Friday February 23. Qalsody aims to slow the decline in patients’ physical abilities.
Indeed, Charcot disease is a progressive and fatal neuromuscular pathology. It is characterized by the progressive death of motor neurons (which allow walking, speaking, swallowing and breathing) which results in muscular atrophy and progressive paralysis of patients. Once diagnosed, life expectancy is three to five years depending on the National Institute of Health and Medical Research (Inserm).
“In about 2% of people living with ALS, the disease is caused by a genetic mutation that causes the production of faulty SOD1 enzymes, leading to nerve cell death“, continues the EMA.
Qalsody: the European Commission must also approve
The EMA has therefore given the green light for marketing in the European Union. However, this recommendation must still be sent to the European Commission, which will make a final decision. In April 2023, advance marketing authorization was issued for this drug in the United States.
Currently, there is no cure for ALS. In the European Union (EU), the only authorized one, riluzole, helps slow the progression of symptoms. In France, about 7,000 people have Charcot disease, according to theBrain Institute, and there are 1,000 new cases in the country every year. The average age of onset of the disease is 59 years.
