FDA Approves Drug for Rare Form of ALS

The agency approved the drug based on evidence of a reduction in plasma neurofilament light and no evidence that the drug slowed the disease.

HealthDay News The US Food and Drug Administration approved a new drug for people who have a rare, inherited type of amyotrophic lateral sclerosis (ALS). The medication, Qalsody (tofersen), targets a mutation in the SOD1-ALS gene.

The FDA approved the drug based on evidence of a reduction in plasma neurofilament light. It did not show that the drug slowed the disease. Still, “the findings are reasonably likely to predict a clinical benefit in patients,” the FDA said in a statement.

“Since SOD1 mutations were first identified as a cause of ALS 30 years ago, the familial ALS community has been searching for genetically targeted treatments,” Jean Swidler, chair of Genetic ALS & FTD: End the Legacy, said in a Biogen news release. “Qalsody offers families who have lost generation after generation in the prime of their life to this devastating disease a therapy targeting the underlying cause of SOD1-ALS. Today marks an important moment in ALS research as Qalsody is the first ALS treatment approved based on a biomarker. We are excited to see what future therapies are developed now that it is understood that lowering levels of neurofilament provides important evidence that a treatment is affecting the neurodegenerative process.”

In the meantime, Biogen will be required to continue studying Qalsody in people who carry the mutation but do not yet have symptoms.

Patients will receive Qalsody through a spinal injection from a health care professional, with a dose of 100 mg each time. SOD1-ALS patients will receive the first 3 doses at 14-day intervals, followed by a maintenance dose every 28 days.

Side effects for those using the medication include joint and muscle pain, fatigue, and increased cerebrospinal fluid white blood cells.

Approval of Qalsody was granted to Biogen.

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