The European Medicines Agency has approved a medicine indicated to reduce the consequences of certain forms of Charcot disease or amyotrophic lateral sclerosis (ALS). The European Union’s decision should come before June.
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization for Qalsody® (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene.
ALS-SOD1: Certain genetic forms of Charcot disease
Amyotrophic lateral sclerosis (ALS) or Charcot disease is a rare, progressive and fatal neurodegenerative disease. Highlighted by Stephen Hawking or via the “Ice Bucket Challenge”, this rare disease causes the loss of motor neurons in the brain and spinal cord which control the voluntary muscles responsible for motor skills.
Affected people may thus present muscular weakness and atrophy, then a loss of autonomy due to their progressive inability to move, to speak, to eat and, ultimately, to breathe, with a very poor long-term prognosis. .
Nearly 168,000 people are affected by ALS worldwide. Among these patients, approximately 2% are affected by a particular genetic form: ALS-SOD1 in which mutations in the SOD1 gene lead to the creation of a toxic form of the SOD1 protein causing the degeneration of motor neurons.
A new drug for 2% of patients
Qalsody (tofersen) is an antisense oligonucleotide that binds to SOD1 gene mRNA to reduce SOD1 protein production. By reducing the amount of defective SOD1 protein, this drug should improve ALS symptoms. An antisense oligonucleotide is a fragment of RNA, usually synthesized in the laboratory, that can bind specifically to a natural messenger RNA.
During the 28th week of phase 3 of the VALOR study conducted in around 100 patients, a 60% reduction in plasma neurofilament light chain (NfL) was observed in participants who received Qalsody compared to the group placebo, suggesting a reduction in neuronal damage.
Trends toward improvement in physical abilities of participants who received Qalsody were observed compared to those who received placebo, as measured by a validated scale (ALS Functional Ratings Scale-Revised (ALSFRS-R). The most common adverse reactions reported were: pain, arthralgia, fatigue, increased CSF white blood cells, increased protein, myalgia and fever.
An arrival on the market for the 2nd quarter of 2024
The European Agency asked the laboratory to submit post-authorization data to further characterize the long-term effectiveness and safety of Qalsody, or on the possible delay of symptoms in presymptomatic SOD1-ALS patients.
The CHMP recommendation for Qalsody will now be considered by the European Commission for a marketing authorization decision in the European Union, with the final decision expected in the second quarter of 2024. If the Marketing Authorization is granted, Qalsody will become the first treatment targeting a genetic cause of ALS, motor neurone disease, to be authorized in the European Union.
“CHMP recommendation for marketing authorization of Qalsody brings hope to the ALS community in Europe“, underlines Prof. Philip Van Damme, professor of neurology and director of the Neuromuscular Reference Center at the University Hospital of Louvain in Belgium. “This is an important milestone for the entire ALS community – for the first time, we have a treatment that has resulted in lasting reductions in neurofilaments, a marker of axonal damage and neurodegeneration“.
Currently, there is only one treatment for ALS (riluzole) authorized in the EU. Patients are offered supportive treatment to relieve symptoms of the disease, such as physical, occupational or speech therapy and respiratory assistance. There is a significant unmet medical need for effective therapies that preserve muscle function and prolong the lives of patients with ALS.
