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Canada has become the first country in the world to approve the drug AMX0035 (to be marketed under the brand name Albriosa), Amylyx Pharmaceuticals’ experimental therapy for amyotrophic lateral sclerosis (ALS). It is an oral therapy dissolved in water that combines two existing drugs, sodium phenylbutyrate and taurursodiol, which prevent nerve cell death.
ALS, whose cause is unknown, is a lethal neurodegenerative disease characterized by the progressive loss of motor neurons in the spinal cord and cerebral cortex. It is the third neurodegenerative pathology in incidence, after dementia and Parkinson’s disease.
It can present at any age, although it is more frequent after the 5th and 6th decades and slightly more frequent in males.
In almost the majority of patients, the disease begins in the muscles that control speech, swallowing, and chewing, or in the muscles of the extremities (only a small group of cases have a generalized or respiratory onset); and approximately 50% of patients die within 3 years of diagnosis.
The Canadian health authority’s decision came three months after the US Food and Drug Administration (FDA) voted against approving the drug, arguing that the clinical study data presented by the company failed to establish that it was effective against the disease. The FDA is expected to rule on the drug again on September 29.
Canada’s approval comes with certain conditions, including publication of data from its global phase III study of approximately 600 patients, results of which are expected in 2024, as well as additional studies.
Nearly 3,000 Canadians are currently living with ALS. In Spain, the Spanish Society of Neurology (SEN) estimates that it has an incidence of 1 to 2 new cases per 100,000 inhabitants per year, which means that, each year, around 700 people will begin to develop the first symptoms of this illness. But its high mortality means that, according to SEN estimates, there are currently about 3,000 affected in our country.
The approval of the drug AMX0035, which will be sold in Canada under the brand name Albrioza, is based on a phase II study in 137 patients and a long-term follow-up phase, which showed that the drug slowed the progression of ALS.
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