Agency approved the drug based on evidence of a reduction in plasma neurofilament light; no evidence the drug slowed the disease<\/p>\n<\/h3>\n
By Physician\u2019s Briefing Staff HealthDay Reporter<\/b><\/p>\n
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WEDNESDAY, April 26, 2023 (HealthDay News) — The U.S. Food and Drug Administration on Tuesday 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<\/em>-ALS gene. <\/p>\n 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.<\/p>\n “Since SOD1<\/em> 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<\/a>. “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<\/em>-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.”<\/p>\n In the meantime, Biogen will be required to continue studying Qalsody in people who carry the mutation but do not yet have symptoms.<\/p>\n Patients will receive Qalsody through a spinal injection from a health care professional, with a dose of 100 mg each time. SOD1<\/em>-ALS patients will receive the first three doses at 14-day intervals, followed by a maintenance dose every 28 days.<\/p>\n Side effects for those using the medication include joint and muscle pain, fatigue, and increased cerebrospinal fluid white blood cells.<\/p>\n Approval of Qalsody was granted to Biogen.<\/p>\n