Kalsodi, new treatment approved by European regulator – release

The European regulator announced this Friday, February 22, that it had approved a drug from the American laboratory Biogen called Clsodi, which is used to treat certain genetic forms of Charcot disease that affect a small proportion of patients. This rare disease, also called amyotrophic lateral sclerosis (ALS), causes progressive paralysis of the muscles, creating a state of incarceration in the patient and usually causing death under the age of five. The main effect of the treatment in question is to slow the decline of the patients’ physical abilities.

In many cases, inherited mutations in the gene that produces the protein in question are the cause of the disease. But this mutation can occur even without a family history. “Clasodi is indicated for the treatment of adults with ALS who have mutations in the superoxide dismutase 1 (SOD1) gene.”The European Medicines Agency (EMA) explains in a press release. “In about 2% of people living with ALS, the disease is caused by a genetic mutation that causes the production of defective SOD1 enzymes, which leads to nerve cell death.”

EMA explains.

EMA’s recommendation will be sent to the European Commission for adoption of a decision on marketing authorization in the EU. Currently, only one treatment for ALS is authorized in the EU, called riluzole. In the United States, an advance marketing authorization was issued in April 2023 for the drug Closodi.

But to date there is no effective neuroprotective treatment for all patients with Charcot disease. Despite progress in recent years, the disease remains incurable at this stage. In late January, an American study conducted on mice also revealed a promising new treatment against Charcot’s disease. A glimmer of hope that, however, still needs to be tested and demonstrate its effectiveness in humans.

According to the Association for Research on Amyotrophic Lateral Sclerosis and Other Motor Neuron Diseases (ARSLA), 8,000 people in France suffer from Charcot disease and it causes five deaths every day. By 2040, ARSLA predicts a 20% increase in people with the disease.