April 26, 2023 – The FDA has approved the primary treatment that uses a genetic-based approach to slow or stop the progression of a rare type of ALS, a debilitating and fatal disease for which there is no such thing as a cure.
Most individuals with ALS (amyotrophic lateral sclerosis) die inside 3 to five years of symptoms onset, normally from respiratory failure. People with ALS, also often known as Lou Gehrig's disease, experience muscle twitching and lose the flexibility to maneuver their limbs, swallow, speak and breathe.
The newly approved drug, called Qalsody, is manufactured by the Swiss company Biogen. The FDA has accelerated approval based on early study results. The agency stated in a Press release The Commission explained that its decision was based on the drug's proven ability to lower a protein within the blood that could be a sign of degeneration of brain and nerve cells.
Although the drug has been shown to affect the chemical process within the body related to degeneration, there was no significant change in symptoms through the first 28 weeks that patients took the drug, Biogen said in a Press releaseHowever, the corporate found that some patients actually experienced improved functioning after starting treatment.
“I have observed the positive effect of Qalsody in slowing the progression of ALS in people with SOD1 mutations,” said Timothy M. Miller, MD, PhD, researcher and co-director of the ALS Center at Washington University School of Medicine in St. Louis, in an announcement 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 the decline in their strength, clinical function and respiratory function.”
Qalsody is given to patients via a spinal injection. The first treatment consists of three injections every two weeks. Thereafter, the injection is given every 28 days.
The latest treatment is just approved for individuals with a rare type of ALS called SOD1-ALS, which is thought for a genetic mutation. While as much as 32,000 people within the U.S. have ALS, only 2% of ALS patients have the SOD1 gene mutation. According to the FDA, the number of individuals within the U.S. who could use Qalsody is about 500.
In studies, 147 people received either Qalsody or a placebo. The treatment resulted in a big reduction in the extent of proteins within the patients' blood which can be related to the lack of control of voluntary muscles.
Because Qalsody received fast-track approval from the FDA, the corporate still must submit more research data in the longer term, including from a study examining the drug's effects on individuals who carry the SOD1 gene but don’t yet show ALS symptoms.