The FDA has approved the first treatment that takes a genetics-based approach to slowing or stopping the progression of a rare form of ALS, the debilitating and deadly disease for which there is no cure.
Most people with ALS, or amyotrophic lateral sclerosis, die within 3 to 5 years of when symptoms appear, usually of respiratory failure. Also known as Lou Gehrig’s disease, people with ALS experience muscle twitching and lose the ability to move their limbs, swallow, speak, and breathe.
The newly approved drug, called Qalsody, is made by the Swiss company Biogen. The FDA fast-tracked the approval based on early trial results. The agency said in a news release that its decision was based on the demonstrated ability of the drug to reduce a protein in the blood that is a sign of degeneration of brain and nerve cells.
While the drug was shown to impact the chemical process in the body linked to degeneration, there was no significant change in people’s symptoms during the first 28 weeks that they took the drug, Biogen said in a news release. But the company noted that some patients did see improved functioning after starting treatment.
“I have observed the positive impact Qalsody has on slowing the progression of ALS in people with SOD1 mutations,” Timothy M. Miller, MD, PhD, researcher and co-director of the ALS Center at Washington University School of Medicine in St. Louis, said in a statement released by Biogen. “The FDA’s approval of Qalsody gives me hope that people living with this rare form of ALS could experience a reduction in decline in strength, clinical function, and respiratory function.”
Qalsody is given to people via a spinal injection, with an initial course of three injections every 2 weeks. People then get the injection once every 28 days.
The new treatment is only approved for people with a rare kind of ALS called SOD1-ALS, which is known for a genetic mutation. While ALS affects up to 32,000 people in the U.S., just 2% of people with ALS have the SOD1 gene mutation. The FDA says the number of people in the U.S. who could use Qalsody is about 500.
In trials, 147 people received either Qalsody or a placebo, and the treatment significantly reduced the level of a protein in people’s blood that is associated with the loss of control of voluntary muscles.
Because Qalsody received a fast-track approval from the FDA, it must still provide more research data in the future, including from a trial examining how the drug affects people who carry the SOD1 gene but do not yet show symptoms of ALS.
Sources
FDA: “FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene.”
Biogen: “FDA Grants Accelerated Approval for QALSODY™ (tofersen) for SOD1-ALS, a Major Scientific Advancement as the First Treatment to Target a Genetic Cause of ALS.”
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