Tuesday, June 18, 2024

Amyotrophic Lateral Sclerosis Treatment: European Commission Approves Biogen’s QALSODY® (tofersen) for Rare Genetic Form

Similar articles

Amyotrophic lateral sclerosis (ALS), specifically SOD1-ALS, is a devastating, uniformly fatal, and ultra-rare genetic form that affects fewer than 1,000 people in Europe. In a significant development, the European Commission (EC) has granted marketing authorization under exceptional circumstances for Biogen’s QALSODY® (tofersen) for the treatment of adults with ALS associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS). This approval marks a critical milestone as QALSODY is the first treatment in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).

QALSODY is Biogen’s third rare disease therapy to receive approval in the EU, demonstrating the company’s ongoing commitment to addressing diseases with high unmet needs. Biogen has played a crucial role in advancing neurofilament as a tool to optimize clinical trial design in ALS, which may expedite future breakthroughs in the field. According to Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen, the approval of QALSODY is a testament to the dedication of the ALS community, including patients, their families, scientists, clinicians, and advocates, who have collaborated over the past two decades to bring this new treatment to the SOD1-ALS community.

Amyotrophic Lateral Sclerosis: EMA Recommends Orphan Status for QALSODY’s Approval Based on Comprehensive Evidence

The European Medicines Agency (EMA) recommended maintaining QALSODY’s designation as an orphan medicinal product. The marketing authorization under exceptional circumstances is recommended when the benefit/risk assessment of a treatment is positive, but comprehensive data cannot be obtained due to the rarity of the disease. QALSODY’s approval is based on a comprehensive body of evidence, including the targeted mechanism of action, biomarker data, and clinical trial results.

In the Phase 3 VALOR study, QALSODY demonstrated promising results, although the primary efficacy endpoint was not statistically significant. The study showed that QALSODY could reduce mean plasma neurofilament light chain (NfL), a marker of axonal injury and neurodegeneration, by 55% in treated participants compared to a 12% increase in the placebo group. Common adverse reactions included pain, fatigue, muscle and joint pain, fever, and increased protein or white blood cell count in cerebrospinal fluid.

Philip Van Damme, M.D., Ph.D., Professor of Neurology at the University Hospital Leuven in Belgium, noted that QALSODY’s approval represents a paradigm shift in the treatment of SOD1-ALS, offering hope to patients and their families who have long awaited a breakthrough. The European Academy of Neurology has confirmed new treatment guidelines recognizing QALSODY as a first-line treatment for patients with SOD1-ALS.

Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis: EUpALS Chairwoman Hails QALSODY Approval as Milestone in Treatability

Evy Reviers, Chairwoman of the European Organisation for Professionals and People living with ALS (EUpALS), expressed excitement over the approval, stating that this milestone shows ALS is a treatable disease. Biogen’s efforts over many years have contributed to this success, and the company is committed to working with stakeholders to ensure access to QALSODY for eligible European patients. Through Biogen’s early access program, approximately 330 people with SOD1-ALS have already received QALSODY across 18 EU countries.

QALSODY is also approved for use in the United States, and Biogen is engaging with regulatory authorities in other regions to expand its availability. The drug is being studied further in the ongoing open-label extension (OLE) of the Phase 3 VALOR study and in the Phase 3 ATLAS study, which evaluates whether QALSODY can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and elevated plasma NfL levels.

ALS is a rare, progressive, and fatal neurodegenerative disease that leads to the loss of motor neurons responsible for controlling voluntary muscle movement. People with ALS experience muscle weakness and atrophy, eventually losing the ability to move, speak, eat, and breathe. The average life expectancy for ALS patients is three to five years from symptom onset. Mutations in the SOD1 gene are responsible for about 2% of ALS cases globally. These mutations cause the production of a toxic misfolded form of the SOD1 protein, leading to motor neuron degeneration.

You can follow our news on our Telegram and LinkedIn accounts.

Biogen has been committed to ALS research for over a decade, continuously investing in understanding all forms of the disease. The company has applied important learnings to its portfolio of assets for genetic and other forms of ALS, with the goal of bringing potential therapies to patients in need. In addition to QALSODY, Biogen has a robust discovery pipeline, including efforts to address TDP43 pathology, which is seen in 97% of ALS cases and is considered a hallmark of the disease.

In conclusion, the European Commission’s approval of QALSODY represents a significant advancement in the treatment of SOD1-ALS, providing new hope for patients and their families. Biogen’s dedication to ALS research and its commitment to working with stakeholders to ensure access to this treatment underscore the company’s role in advancing care for rare diseases.

Resource: Biogen, May 30, 2024

Subscribe to our newsletter

To be updated with all the latest news, offers and special announcements.

Latest article