FDA Approves New ALS Drug, Sparks Hope for the Future of Treatment

• The FDA recently announced fast-track approval for a new ALS drug.
• The drug, tofersen, was developed specifically for patients with SOD1-ALS, a rare form of the disease that occurs because of mutation in the SOD1 gene.
• Experts are hopeful that this milestone will have a substantial impact on future ALS treatment.

The Food and Drug Administration (FDA) announced fast-track approval for a new amyotrophic lateral sclerosis (ALS) drug.

The new drug works to treat a specific genetic form of ALS. The medication, tofersen, which goes under the brand name Qalsody, was developed for people who have SOD1-ALS, a rare form of the neurological disease that results from a mutation in the SOD1 gene.

Approximately 2% of people with ALS have this rare mutation that causes their disease. Researchers say that while this is medication for a very specific subset of ALS cases, its development offers a window into the expanding research and understanding of the disease.

“I was super pleased to hear that tofersen would be available to treat people living with SOD1 mutations and gives me hope that tofersen will be able to substantially slow down this genetic form of ALS,” principal investigator of the drug’s clinical trials Timothy M. Miller, MD, PhD, the David Clayson Professor of Neurology at Washington University, told Health.

The biotechnology company behind the development of the drug, Biogen Inc., revealed that Qalsody will be available for shipment to U.S.-based providers in early May, according to a press release. It stands as the first treatment “to target a genetic cause of ALS” approved by the FDA.

Accelerated FDA approval

Dr. Miller, who is also the co-director of the Washington University School of Medicine’s ALS Center, explained the potential impact for those with SOD1 mutations “is the substantial showing of disease progression and improvements in quality of life.”

Researchers in the field have been trying to find solutions to ALS, a disease that as a whole poses a major puzzle to researchers, for decades. An understanding of clear risk factors for the disease remains elusive, and in many ways, there is no real pattern to determine who is most likely to develop ALS. When it comes to looking into this specific mutation of SOD1, Dr. Miller and his team found a problem that has a clear cause.

Results from the phase 3 clinical trial of tofersen were published last year in The New England Journal of Medicine. They revealed that the drug “reduced molecular signs of disease and curbed neurodegeneration in the first six months of use.”

The recent approval falls under the FDA’s accelerated approval pathway, which means drugs developed for serious conditions that “face an unmet medical need” are approved relatively quickly.

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The Impact of Tofersen

When asked to put this drug approval in context, Martina H. Wiedau, MD, professor of neurology and the director of the ALS Clinic and Research Center at UCLA Health, told Health the FDA approval of tofersen “can motivate doctors to conduct genetic testing for SDO1 mutations early and provide this treatment.”

Dr. Wiedau, who is unaffiliated with Dr. Miller’s research, pointed out that this study did not select participants who were in early disease stages—some had lived with ALS for a long time. She said “one would assume” that the neurodegenerative process from SOD1-ALS “can be slowed with earlier treatment.”

“I am very hopeful that in the long run we will see better clinical benefits from tofersen than presented in the VALOR trial publication,” Dr. Wiedau noted. She expressed her excitement for the approval, adding, “I am sure it will be followed by genetic treatments for other gene mutations in ALS. I am very hopeful that we will make a real difference in some individuals with ALS.”

When asked at what stage of diagnosis would it be appropriate to prescribe this drug, Dr. Miller said it would ideally be “as early as possible” in order to “maintain as much function as possible.”

Improving ALS Patients’ Quality of Life

Symptoms of ALS can vary greatly. Some cases include common degenerative symptoms like difficulty walking and carrying out day-to-day tasks, falling down, leg, feet, and ankle weakness, slurred speech, muscle cramps, and twitching of the arms, among others.

Dr. Miller explained that tofersen can promote the maintenance of one’s overall strength, which in turn, can go to great lengths to make one’s daily life easier.

“The drug should be able to slow down disease progression significantly when given early. Therefore, patients may have a real chance of living longer lives with ALS, allowing middle-aged patients to see their children graduate from school and also be active longer with good function,” Dr. Wiedau added.

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The Future of ALS Care

The Centers for Disease Control and Prevention (CDC) estimates that more than 31,000 people are currently living with ALS in the U.S.

While this new medication only addresses a specific population within those totals, Dr. Miller and Dr. Wiedau assert that it signals a future where those living with ALS might have more, better options for care.

“There is momentum” for the development of new ALS treatments, Dr. Wiedau noted.

“Three drugs that can slow down the disease to a small degree and the first genetic treatment are exciting news for the ALS community,” she said. “The difficult part is transferring the expected successes for patients with familial ALS to patients with sporadic disease.”

Dr. Miller added that this drug “can have substantial effects in this genetic subset of ALS” which points to the fact that this disease is a “treatable disorder.” It offers a ray of light to researchers and those living with ALS who don’t have this rare genetic mutation that treatments that will have a “substantial impact on other forms of ALS” are hopefully on the way.

“The lowering of neurofilament by tofersen shows that this biomarker can be lowered with an effective drug,” he said. “Measurement of this biomarker will likely be included more frequently in ALS drug development programs.”