Ionis’ tofersen may be given accelerated approval by the USFDA for SOD1-ALS, according to the advisory committee’s unanimous vote.

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Tofersen, an investigational antisense drug for the treatment of people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis, was discussed at the Peripheral and Central Nervous System Drugs Advisory Committee meeting by the US Food and Drug Administration (FDA), according to a statement released by Ionis (ALS). A rare hereditary variant of ALS called SOD1-ALS causes loss of daily functioning and eventually death.

The committee voted unanimously in favour of considering an expedited approval (9 yes votes to 0 no) in response to the question, “Is the available evidence sufficient to conclude that a reduction in plasma neurofilament light chain (NfL) concentration in tofersen-treated patients is reasonably likely to predict clinical benefit of tofersen for treatment of patients with SOD1-ALS?” The committee’s decision on the second question, “Does the clinical data from the placebo-controlled study and available long-term extension study results, with additional supporting results from the effects on relevant biomarkers (i.e., changes in plasma NfL concentration and/or reductions in SOD1), provide substantial evidence of the effectiveness of tofersen in the treatment of patients with SOD1-ALS?” was 3 (yes), 5 (no), and 1 (abstain). Also, the committee talked about both of these issues and agreed that the benefit-risk profile for tofersen in patients with SOD1-ALS was favourable after reviewing all available data.

“The conclusion of the advisory committee meeting today gives us hope. Tofersen, if approved, would be the first drug to address a recognised cause of familial ALS, a fatal neurodegenerative condition that impairs motor function and usually kills patients within two to five years of diagnosis “Ionis’ executive vice president and chief scientific officer, C. Frank Bennett, Ph.D., said. “Tofersen is the top investigational drug in our cutting-edge pipeline of neurological treatments that aim to treat the underlying conditions that contribute to incurable illnesses. We are diligently developing these services in order to perhaps assist patients who are in need.”

The FDA is given non-binding recommendations from advisory committees. The FDA received a New Drug Application for tofersen for the management of SOD1-ALS for consideration under accelerated approval. With an action deadline of April 25, 2023 under the Prescription Drug User Fee Act (PDUFA), the FDA is still examining tofersen. An investigational antisense drug called Tofersen is being studied as a potential treatment for SOD1-ALS. Because of mutations in the SOD1 gene, those who have this type of disease produce a toxic form of SOD1 protein in their bodies. Motor neurons deteriorate as a result of this toxic protein, causing increasing muscular weakening. Tofersen is made to attach to the SOD1 mRNA and stop SOD1 protein synthesis.

Tofersen is being researched in the phase 3 ATLAS study, which aims to determine whether tofersen can delay clinical onset when started in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity, in addition to the ongoing open label extension of the phase 3 VALOR study. A collaborative development and licence arrangement between Biogen and Ionis allowed for the licencing of tofersen. A uncommon, deadly neurological condition called amyotrophic lateral sclerosis (ALS) causes the loss of motor neurons in the brain and spinal cord, which are in charge of regulating voluntary muscle movement. Individuals who have ALS have muscle atrophy and weakness, which makes it harder for them to maintain their independence as they gradually lose their ability to move, speak, eat, and eventually breathe. Between three and five years pass after the onset of symptoms before an ALS patient dies on average.

The cause of ALS has been linked to numerous genes. Even in people without a family history of the disease, genetic testing can assist identify whether a person’s ALS is linked to a genetic mutation. There are no genetically targeted therapies available for ALS at the moment. About 330 persons in the US are affected by SOD1-ALS, which is identified in about 2% of all instances of ALS. Although there are drugs approved to treat generalised ALS, there are no medications that specifically target a genetic mutation linked to ALS. Though they may not have a known family history of the disease, it is estimated that 5% to 10% of those who have ALS have a genetic type of the illness. Ionis has been a pioneer in creating new markets and altering medical standards of care for more than 30 years as a leader in RNA-targeted therapy. Ionis now has three products on the market and a strong late-stage pipeline with franchises in the neurological and cardiovascular markets.

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