American pharmaceutical company Biogen has announced that it has received preliminary approval in the United States for the release of its drug Qalsody, which is used to treat genetic forms of amyotrophic lateral sclerosis (ALS), which affects a small number of these patients, AFP and Agerpres reported.

The US Food and Drug Administration (FDA) has approved Qalsody (trade name Tofersen) in the United States for the treatment of ALS-SOD1, a genetic form of amyotrophic lateral sclerosis, a disease known as Charcot disease. “, Biogen said in a statement released Tuesday evening.

A phase 3 study, with final results expected in 2026-2027, should confirm this anticipated decision.

Qalsody is the first approved treatment targeting a genetic cause of amyotrophic lateral sclerosis (ALS), its US maker said, saying that in Europe the European Medicines Agency (EMA) had agreed to review Tofersen’s marketing authorization in December 2022. the procedure is still ongoing.

Genetic forms of Charcot disease affect only 10% to 15% of patients. According to data published by the ALS Research Association, the SOD1 gene is present in only 3-4% of all ALS cases.

This approval is based, in particular, on the results of several consecutive preliminary studies conducted on several dozen patients aged 23 to 78 years. Doctors found less pronounced decline in physical abilities in patients who received Qalsody than in those who received a placebo.

To date, there is no cure for ALS

The green light given by the US health authorities gives “hope that people living with this rare form of ALS can benefit from a reduction in their physical strength, clinical and respiratory functions,” said Timothy Miller, who tested the drug. Kalsodi and who is co-director of the ALS Center at Washington University in St. Louis. Louis

Despite certain successes achieved in recent years, amyotrophic lateral sclerosis still remains an incurable disease.

This leads to progressive muscle paralysis, a process that gradually makes it impossible to walk, feed, speak and breathe, creating a state of “imprisonment” for the patient as his brain and intellectual abilities remain intact.

After making this diagnosis, the patient’s life expectancy does not exceed three to five years. However, some treatments can provide a few extra months of life by slowing the progression of the disease.