Europe's medicines watchdog gave the green light Friday to a new treatment for a rare form of the neurodegenerative disease ALS, saying it should reduce the symptoms of the deadly illness.
Amyotrophic lateral sclerosis, sometimes called Lou Gehrig's disease after the famous baseball player, devastates nerve cells in the brain and spinal cord. The mean survival time with ALS is two to five years.
Sold under the brand name "Qalsody", the medicine toferson is aimed at adults suffering from ALS as the result of a genetic mutation, the European Medicines Agency said.
"The exact causes of ALS are unknown but are believed to include genetic and environmental factors," the Amsterdam-based EMA said.
"In approximately 2.0 percent of people living with ALS, the condition is caused by a genetic mutation (change) that leads to the production of defective SOD1 enzymes, causing nerve cells to die," it said.
Qalsody reduces the production of the SOD1 protein and is therefore "expected to improve the symptoms of ALS."
To date, there is no effective neuroprotective treatment for all ALS patients.
Despite the progress made in recent years, the disease currently remains incurable.
There is only one other treatment for ALS, called "Riluzole", authorised in the EU.
Patients are also offered supportive treatment to relieve the symptoms of the disease, such as physical, occupational or speech therapy and breathing support.
"There is a large unmet medical need for effective therapies that preserve muscle function and prolong the life of patients with ALS," the EMA said.