Biogen’s Qalsody (tofersen) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) as the first therapy for a rare, genetic form of motor neurone disease (MND).
The drug has been authorised by the UK regulator to treat adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene.
An estimated 168,000 people worldwide are affected by ALS, a progressive and fatal neurodegenerative disease that impacts nerve cells in the brain and spinal cord. Patients experience muscle weakness and atrophy, causing them to lose the ability to move, speak, swallow and eventually breath.
Multiple genes have been implicated in ALS, and mutations in the SOD1 gene are responsible for around 2% of all cases.
Given by lumbar puncture at regular intervals by a healthcare professional, Qalsody is an antisense oligonucleotide designed to bind to SOD1 mRNA and reduce SOD1 protein production.
The MHRA’s decision on the drug occurred through the International Recognition Procedure, a fast-track approval process that takes into account prior authorisations from other regulatory partners, and follows the European Commission granting marketing authorisation under exceptional circumstances for tofersen in May last year.
Among the evidence supporting Biogen’s application were results from the late-stage VALOR study, which showed that patients treated with Qalsody experienced significant reductions in plasma neurofilament light chain, a marker of neurodegeneration, compared to those receiving placebo.
Kylie Bromley, general manager, Biogen UK and Ireland, said the approval “represents not only a significant scientific and regulatory achievement, but more importantly, a moment of real hope for the MND community”.
She added that the company is “calling for flexibility from the National Institute for Health and Care Excellence (NICE), Scottish Medicines Consortium (SMC) and NHS England to secure long-term and sustainable access for tofersen through a collaborative reimbursement process”.
The three main UK MND charities – MND Scotland, MND Association and My Name’5 Doddie Foundation – also welcomed the MHRA’s decision and described tofersen as a “genuine breakthrough” for patients with SOD1-ALS.
Qalsody, which Biogen has licensed from Ionis Pharmaceuticals, was granted accelerated approval by the US Food and Drug Administration in April 2023 for the same patient population.
