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Ionis announces that FDA accepts New Drug Application and grants Priority Review of tofersen for a rare, genetic form of ALS
SOD1-ALS is a rare genetic form of ALS that affects approximately 330 people in the U.S., it is progressive, leads to the loss of everyday functions and is
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"SOD1-ALS is a rare genetic form of ALS that affects approximately 330 people in the U.S., it is progressive, leads to the loss of everyday functions and is uniformly fatal If approved, tofersen would be the first treatment to target a genetic cause of ALS12-month data included in the filing show that earlier initiation of tofersen slowed decline across measures of clinical and respiratory function, strength, and quality of life; tofersen also led to robust and sustained reductions in neurofilament, a marker of neurodegenerationCARLSBAD, Calif., July 26, 2022 /PRNewswire/ -- Ionis Pharmaceuticals (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational antisense medicine for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS). The NDA was submitted by Biogen, which licensed tofersen from Ionis in 2018. The application has been granted priority review and given a Prescription Drug User Fee Act action date of Jan. 25, 2023. The FDA has noted that it is currently planning to hold an Advisory Committee meeting for this application. The average life expectancy for people with ALS is three to five years from time of symptom onset; patients with some SOD1 mutations have an even shorter life expectancy. There is currently no treatment targeted for SOD1-ALS. \n\n \n \n \n \n \n \n\n \n\"Acceptance of the new drug application for tofersen is a monumental milestone, not just for Ionis but for all people with SOD1-ALS, their families and healthcare professionals battling this devastating disease. To them we extend our deepest gratitude. Their courage has been instrumental to the achievement of this goal,\" said C. Frank Bennett, Ph.D., executive vice president, chief scientific officer and franchise leader for neurological programs at Ionis. \"We also want to thank Biogen for their commitment to advancing tofersen, which, if approved, will be the first treatment that targets a genetic cause of ALS.\" Dr. Bennett added, \"Acceptance of the NDA for tofersen further strengthens Ionis' platform strategy to target all forms of ALS and central nervous system disorders more broadly.\"\nBiogen is seeking approval of tofersen under the FDA's accelerated approval pathway, based on the use of neurofilament as a surrogate biomar...