Biogen’s Qalsody, containing tofersen, has received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) as the initial treatment for a rare genetic type of motor neurone disease (MND). Specifically, this drug has been greenlit by the UK regulator to be used in adults diagnosed with amyotrophic lateral sclerosis (ALS) linked to a mutation in the superoxide dismutase 1 (SOD1) gene. ALS is a severe and fatal neurodegenerative condition affecting nerve cells in the brain and spinal cord, leading to muscle weakness and atrophy, ultimately resulting in the loss of essential functions like movement, speech, swallowing, and breathing.
The global population impacted by ALS is estimated at around 168,000 individuals. Among the various genes associated with ALS, mutations in the SOD1 gene account for approximately 2% of all cases. Qalsody is administered through lumbar puncture at specified intervals by a healthcare professional. It is classified as an antisense oligonucleotide developed to bind to SOD1 mRNA, thereby reducing the production of SOD1 protein within the body.
The MHRA’s approval of Qalsody came via the International Recognition Procedure, a streamlined process that considers prior authorizations from other regulatory bodies. This decision follows the European Commission’s exceptional circumstances approval for tofersen in May of the previous year. Noteworthy supporting data for Biogen’s submission included findings from the VALOR study’s late stage, where patients treated with Qalsody exhibited significant decreases in plasma neurofilament light chain levels, a key marker of neurodegeneration, in comparison to those on a placebo.
Kylie Bromley, Biogen UK and Ireland’s general manager, expressed that the approval signifies a major scientific and regulatory milestone, offering genuine hope to the MND community. She also emphasized the company’s appeal to the National Institute for Health and Care Excellence (NICE), Scottish Medicines Consortium (SMC), and NHS England for flexibility in ensuring sustainable and continued access to tofersen through a collaborative reimbursement process. The approval was also met with enthusiasm from the three primary UK MND charities – MND Scotland, MND Association, and My Name’5 Doddie Foundation – who hailed tofersen as a groundbreaking advancement for individuals affected by SOD1-ALS.
Qalsody, which Biogen licensed from Ionis Pharmaceuticals, gained accelerated approval from the US Food and Drug Administration in April 2023 for the identical patient cohort. This dual regulatory approval signifies a crucial step forward in addressing the unmet medical needs of individuals battling this challenging disease.
Key Takeaways:
– Biogen’s Qalsody, approved by the MHRA, is the first therapy for a rare genetic form of motor neurone disease.
– Qalsody targets SOD1-associated ALS through a mechanism that reduces SOD1 protein production.
– The drug’s approval follows successful results from the VALOR study, demonstrating significant benefits over a placebo.
– Collaboration between Biogen and regulatory bodies is essential to ensure long-term access to this innovative therapy for the MND community.
Tags: regulatory
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