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Biogen Receives European Commission Approval for QALSODY (tofersen), the First Therapy to Treat a Rare, Genetic Form of ALS

May 30, 2024 – Clinical Trials, Drug Discovery, Pharmaceutical – ALS, Biogen, clinical trials, motor neuron disease, rare diseases

SOD1-ALS is a devastating, uniformly fatal and ultra-rare genetic form of ALS estimated to affect less than 1,000 people in Europe
QALSODY is Biogen’s third rare disease therapy to be approved in the EU, demonstrating the company’s commitment to addressing diseases with a high unmet need
With QALSODY, Biogen has helped advance neurofilament as a tool to optimise clinical trial design in ALS, offering the potential to expedite further breakthroughs in the field.

30 May 2024 — Massachusetts, US — Biogen announced the European Commission (EC) has granted marketing authorisation under exceptional circumstances and maintained orphan designation for QALSODY (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS). QALSODY is the first treatment approved in the EU to target a genetic cause of ALS, also known as motor neuron disease (MND).

“The European Commission’s approval of QALSODY is a testament to the unwavering dedication of the ALS community – people living with ALS and their loved ones, scientists, clinicians and advocates – who have worked together over the past two decades to bring forward this important new treatment for the SOD1-ALS community,” said Stephanie Fradette, PharmD, head of the Neuromuscular Development Unit at Biogen. “We are working with the medical community and local authorities to bring QALSODY to people living with SOD1-ALS across the region as quickly as possible.”

The marketing authorisation for QALSODY is granted under exceptional circumstances, which is recommended when the benefit/risk assessment of a treatment is determined to be positive but due to the rarity of the disease, it is unlikely that comprehensive data can be obtained under normal conditions of use. The European Medicines Agency (EMA) recommended QALSODY’s designation as an orphan medicinal product be maintained.

“QALSODY’s approval represents a paradigm shift in the treatment of SOD1-ALS, offering hope to patients and loved ones who have long awaited a breakthrough,” said Philip Van Damme MD PhD, professor of Neurology and Director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium. “The European Academy of Neurology has confirmed new treatment guidelines for ALS that recognise QALSODY should be offered as first-line treatment for patients with SOD1-ALS.”

The approval of QALSODY is based on the totality of evidence, including the targeted mechanism of action, biomarker and clinical data. In the randomised, double-blind, placebo-controlled phase 3 VALOR study (n=108), patients were randomised 2:1 to receive treatment with either QALSODY 100mg (n=72) or placebo (n=36) for 24 weeks. The primary efficacy endpoint was the change from baseline to Week 28 in the ALS Functional Ratings Scale-Revised total score. The results numerically favoured tofersen, but were not statistically significant (ITT population: tofersen-placebo adjusted mean difference [95% CI]: 1.4 [-1.3, 4.1]). At Week 28, mean plasma neurofilament light chain (NfL), a marker of axonal injury and neurodegeneration, was reduced by 55% (geometric mean ratio to baseline) in the tofersen-treated participants (ITT), compared to a 12% increase with placebo (difference in geometric mean ratios for tofersen to placebo: 60% (95% CI: 51%, 67%)). Very common adverse reactions (may affect more than 1 in 10 people) reported in QALSODY-treated participants were pain (back pain, pain in arms or legs), feeling tired, muscle and joint pain, fever and an increase in protein and/or white blood cell count occurring in the fluid that surrounds the brain and spinal cord.

“At EUpALS, we are excited that people with SOD1-ALS in Europe will have access to QALSODY, the first treatment targeting a genetic cause of ALS. This is a major milestone for the ALS community, showing that ALS is a treatable disease,” said Evy Reviers, chairwoman of the European Organisation for Professionals and People living with ALS (EUpALS). “As a representative of the European ALS community, I am excited to enter a new evolution in the common fight against ALS. We thank Biogen for the many years of scientific and clinical pioneering efforts that led to this medical success.”

Biogen is committed to working closely with all stakeholders to enable access to this treatment for eligible European patients. Through the Biogen early access programme, about 330 people with SOD1-ALS have received QALSODY across 18 EU countries. QALSODY is also approved for use in the US and Biogen is engaging with regulatory authorities in other regions.

About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at www.biogen.com.