According to Ionis’ announcement, the U.S. FDA accepted an NDA (New Drug Application) for their drug tofersen, which is a new antisense medicine that could be used as treatment of a rare form of ALS called superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS). Biogen had licensed the drug from Ionic back in 2018, and the application was submitted by them.
Priority Review has been granted and Prescription Drug User Fee Act action date is also given, which is 25th January, 2023. According to the FDA an Advisory Committee meeting plan is underway.
After symptoms begin to show, the average life expectancy for ALS patients is a mere three to five years. It is even less for some patients that have SOD1 mutations. As of now, no treatment exists for SOD1-ALS.
Ionis is a 23-year-old biotechnology company based in Carlsbad, California, that is an industry leader in RNA-targeted therapy. The company has three marketed medicines as of now. The company uses its own innovative antisense technology.
Torfersen is an antisense medicine that is being assessed as the possible treatment of SOD1-ALS. Apart from the continued OLE (open label extension) of VALOR, the medicine is being assessed in Phase 3 ATLAS study which will evaluate if it can slow down clinical onset when used in presymptomatic patients.
ALS, or Amyotrophic lateral sclerosis is an uncommon and fatal neurodegenerative disease that harms the motor neurons in the brain and spinal cord, resulting in the loss of muscle movement in the long-term. Worst-case scenarios include inability to speak, eat, move and ultimately breathe. SODA mutations account for roughly 2% of the approximated 168,000 people globally, who suffer from SOD1-ALS.
Executive Vice President and Chief Scientific Officer at Ionis, Frank Bennett (Ph.D) said, “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.”
SOD1-ALS affects roughly 330 people in the United States and is unvaryingly fatal. Tofersen could be the first medication targeting the genetic cause of ALS upon approval. 1-year data shows that tofersen decelerated the decline of respiratory function as well as quality of life and strength.
FDA’s accelerated approval pathway is being used by Biogen to attain approval for tofersen. New Drug Application for tofersen included multiple study results, including Phase 1, Phase 1/2, Phase 3, OLE (open label extension), and 1-year combined results from OLE and VALOR study.
The OLE and VALOR combined data was an indication that teforsen led to constant reductions in neurofilaments, which is an indicator of neurodegeneration, while also reducing the pace of decline on multiple endpoints. The most common negative effects in patients that received the medicine in the study were back pain, headache, and procedural pain. Most of these were moderate at worst. Approximately 17.3% of the patients stopped the treatment owing to an adverse event.
