2023-10-25 08:00:41
On September 29, 2022, American public health authorities authorized the marketing of a new drug once morest amyotrophic lateral sclerosis or Charcot disease. One year later, the ANSM has just given the green light for pre-early compassionate access so that French patients can benefit from this treatment. Explanations.
Charcot disease, an incurable neurological disease
The amyotrophic lateral sclerosisbetter known as Charcot disease, is a neurological pathology, which affects the motor neurons of the cerebral cortex and spinal cord. It develops insidiously and causes paralysis of the limbs, pharynx and respiratory tract. The diagnosis is generally only made following several months of diagnostic research. The disease will gradually progress towards different disabilities, not only motor, but also respiratory and nutritional.
Currently, there is no drug capable of curing Charcot’s disease. The care is multidisciplinary, with continuous and close monitoring. Only one neuroprotective drug has been available since the 1990s. Unfortunately, its effectiveness remains limited. Patients and their loved ones place their hopes in the development of new treatments.
A new medicine available on exceptional prescription
Among the latest therapeutic discoveries once morest Charcot disease is the drug AMX0035. This medicine is a combination of sodium phenylbutyrate and ursodoxicoltaurine. In 2020, a clinical trial showed good tolerability of this drug in patients and a positive functional effect at 24 weeks. A reduction in the risk of death of more than 40% was observed, with a median survival period extended by more than 6 months for patients receiving the treatment, compared to patients receiving placebo.
Other clinical trials are currently underway to evaluate the efficacy and safety of AMX0035 over a longer period. Already, the American and then Canadian public health authorities had given the green light for the marketing of this drug. The ANSM, in conjunction with the ARSLA, has also just validated the possibility for French patients to receive this treatment. Before the end of the clinical trial currently underway in Europe, patients will receive the drug under an exceptional prescription. The first eligible patients will be patients with amyotrophic lateral sclerosis whose first symptoms appeared for at least one year or more than 3 years. These criteria might be re-evaluated at the beginning of 2024.
Effectiveness to be confirmed by ongoing clinical trials
To receive this treatment, patients must have a nominative request for authorization made by a neurologist practicing in one of the 22 national reference centers for the treatment of amyotrophic lateral sclerosis. If ongoing clinical trials confirm the effectiveness and safety of this new drug, it might obtain marketing authorization and be accessible to more patients.
The medicine AMX0035 comes in the form of individual sachets, each containing 3 g of sodium phenylbutyrate and 1 g of ursodoxicoltaurine. The patient should dissolve the contents of the sachet in 250 ml of water at room temperature. The medicine is administered orally or through the feeding tube within 1 hour of preparation. The dosage is first one sachet per day for 3 weeks, then 2 sachets per day. The main side effects are diarrhea, abdominal pain, nausea and ENT tract infections. Access to this drug represents a step forward for patients suffering from Charcot’s disease, although it should be noted that the results of ongoing clinical trials will make it possible to confirm or not its effectiveness.
Estelle B., Doctor of Pharmacy
Sources
– AUTHORIZATION OF AMX0035 IN THE UNITED STATES. FILSLAN. October 7, 2022. portal-sla.fr. Accessed October 16, 2023.
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