2023-05-02 15:51:01
Scientific research on Charcot’s disease is progressing! The proof with this announcement from the Biogen lab which will market Qalsody, a new treatment once morest a rare genetic form (mutation of the SOD1 gene).
« Today marks a turning point in amyotrophic lateral sclerosis research (ALS)”. It is with these words of hope that Christopher A. Viehbacher, CEO of the pharmaceutical company Biogenannounced on April 28, 2023 the news: the Food and Drugs Administration (FDA) (the equivalent of our National Agency for the Safety of Medicines) has just authorized the marketing of a treatment once morest ALS, also called de Charcot, in adults with a mutation in the SOD1 (superoxide dismutase 1) gene, which affects approximately 330 people in the United States and less than a thousand in Europe.
Mistake in injection
Her name ? THE ” Mistake (tofersen). The drug, prescribed as a 100 mg/15 ml injection, has shown good results in slowing down this progressive and fatal neurodegenerative disease by acting on the plasma level of neurofilament light chain (NfL), a biomarker of neurodegeneration. This marketing authorization was obtained following several conclusive clinical trials, conducted in 2022 for 28 weeks on patients aged 23 to 78 with muscle weakness associated with ALS and a confirmed SOD1 gene mutation in laboratory.
Improved motor and lung function
Researchers at the University of Sheffield, England, then found that this treatment had significantly reduced levels of the defective protein (SOD1), improving the motor and lung function of patients. Nevertheless, some warnings and precautions for use relate to serious neurological events, including cases of myelitis and/or radiculopathy, papilledema, intracranial hypertension and aseptic meningitis and may lead to stopping or interrupting treatment. temporarily. Some adverse effects are also seen in 10% or more of patients: pain, severe fatigue, arthralgia, elevated leukocyte count in cerebrospinal fluid (CSF), and myalgia.
« FDA clearance for Qalsody gives me hope that people with this rare form may see declines in strength, physical function and lung function slow “, rejoiced Timothy M. Miller, involved in the research. Her study was published in September 2022 in the National library of medicine.
2% have the SOD1 genetic mutation
15% of people with Charcot’s disease now have a genetic form of the disease (a family form) and 2% have the SOD1 genetic mutation, precisely targeted by the tofersen. It has been thirty years since SOD1 was identified as the cause of ALS. Since then, scientific research has been carried out relentlessly to find treatments targeting genetic factors. ” Confirmation of clinical benefit in confirmatory trials might lead to permanent authorization “, precise Biogenwhich looks like ” pioneer in research on this disease. The study ” of phase 3 ATLAS » currently in progress, which aims to assess the tofersen in presymptomatic carriers of the SOD1 mutation will be considered as “ confirmatory trial “, she continues. In Europe, the European Medicines Agency (EMA) has agreed to examine the application for marketing authorization (MA) of tofersenin December 2022. The procedure is ongoing.
What regarding other forms of the disease?
It now remains to find treatments for the other forms, whether genetic or not, of the disease, which still represent the majority of cases. ” We are convinced that this important scientific breakthrough will give an additional impetus to the development of innovative drugs once morest ALS. concludes Christopher A. Viehbacher. Great hopes rest in this respect on another drug under development, also proposed by Biogenthe « BIIB105 “, which would act on another genetic mutation of the disease, ataxin-2 (ATXN2).
“All reproduction and representation rights reserved.© Handicap.fr. This article was written by Clotilde Costil, Handicap.fr journalist”
1683055419
#treatment #market #United #States
