The SLA it is a progressive neurodegenerative disease and despite considerable research efforts made over several decades, no new therapies have been approved for more than 25 years. But now a new drug that provides a significant benefit in function and survival has already been approved in the United States and is in the process of being approved in Europe.
Amyotrophic lateral sclerosis is the most common neurodegenerative disorder of middle age. In Europe, most patients spend about a third of their life seeking a diagnosis. The median survival is 2-5 years from the onset of symptoms. ALS is caused by death of motor neurons in the brain and spinal cord, resulting in deterioration of muscle function, inability to move, speak, swallow and eat, respiratory paralysis and, ultimately, death. In the later stages of the disease, a person may need around-the-clock care, a wheelchair to allow for mobility, and mechanical support for communication, eating, and breathing.
AMX0035: the drug for ALS being approved in Europe
A new drug is currently being reviewed for an indication in the treatment of ALS by regulatory bodies in Europe, so it will soon be available to Italian patients as well. “Is called AMX0035 e in Europe it is a therapy not yet approvedcurrently under regulatory review,” he explains Adriano Chio, Director of Neurology 1 at the Molinette Hospital and Professor of Neurology at the University of Turin. “In countries where it is approved for use, AMX0035 is the first ALS treatment to demonstrate a clinically meaningful benefit in function in a clinical trial, as well as showing a long-term overall survival benefit.”
AMX0035 is a fixed dose oral combination therapy of two small molecules: the sodium phenylbutyrate and theursodoxicoltaurina, specifically combined to ensure effective dosing, absorption, exposure and the rigorous quality standards required for a pharmaceutical product. Preclinical studies and early clinical studies demonstrate that the AMX0035 fixed dose combination and its individual components have the ability to target multiple cellular pathways involved in ALS. The approval of the drug in Europe should take place within this year.
