FDA Approval of New Drug for SOD1-ALS Marks Huge Step Forward in MND Treatment
26 April 2023
26 April 2023
Tofersen, an antisense oligonucleotide therapy which stops mutant SOD1 protein from being made, is the first approved treatment to target a genetic cause of MND. SOD1 mutations are found in 2-3% of the total MND population. In September 2022, we reported the results of a phase 3 trial of tofersen, which showed it could slow and reduce progression of the disease in people living with MND who had a SOD1 mutation (SOD1-ALS).
On 25th April 2023, the FDA announced the approval of tofersen (now known as Qalsody in the US) to treat people with SOD1-ALS. Tofersen was approved based on the reduction of NfL, a protein that is released into the bloodstream upon injury or degeneration of nerve cells. This is a major step forward, as it is the first time a regulatory decision for an MND treatment has been made based on a biological readout (“biomarker”).
To confirm the clinical benefit of tofersen, a phase 3 randomized, double-blind, placebo-controlled trial is ongoing in individuals who are carriers of the SOD1 genetic mutation, but do not yet have symptoms of the disease. Individual’s NfL levels will be routinely monitored, and if/when NFL starts to increase, treatment with tofersen will be offered, even if other signs of the disease are not apparent. The study will assess the proportion of individuals treated with tofersen who go on to develop symptoms of ALS during the trial compared to placebo.
Jessica Lee, Director of Research at My Name’5 Doddie Foundation said, “FDA’s approval of tofersen for SOD1-ALS is a major step forward in the treatment of MND. Tofersen is the first antisense oligonucleotide treatment to be approved for MND providing hope that similar treatments that target other genetic causes of MND, which are already in the pipeline, will also be effective.
This month saw the launch of My Name’5 Doddie Foundation’s new research strategy which is focused on expediting the development of new treatments for MND. This news brings hope that effective treatments for MND are now within reach. We will continue to build on the excitement and momentum within the field to accelerate the development of effective treatments for all people living with MND.”
Before tofersen is available through the NHS, it must be approved by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and recommended by the National Institute for Health and Care Excellence (NICE).
Biogen, the company that has developed tofersen, provided the update below to My Name’5 Doddie Foundation on 28 April, 2023:
We will keep the MND community updated on any further updates regarding tofersen and its progress through the review pathway in the UK.