On October 25, the High Authority for Health (HAS) in France denied early access to Qalsody, a potential treatment for Charcot's disease, also known as amyotrophic lateral sclerosis (ALS). This decision sparked significant backlash from patients and advocacy groups, particularly the Association for Research on ALS (ARSLA). The drug, developed by Biogen, is currently available only through compassionate access, costing approximately 30,000 euros per month for a spinal injection. Qalsody targets a specific genetic mutation (SOD1) found in about 1% of ALS cases, aiming to slow disease progression.
The HAS clarified its decision on November 8, stating that while the drug meets criteria for treating a serious and rare disease, it did not provide sufficient evidence of clinical efficacy. The results from Biogen's phase III clinical trial did not show a statistically significant difference between Qalsody and placebo after 28 weeks. The HAS has invited Biogen to submit more robust data to support its claims.
Despite the negative opinion, the HAS continues to evaluate Qalsody for standard reimbursement under ordinary law. The authority expressed its awareness of the urgent need for effective treatments in ALS and remains open to further discussions with Biogen. Specialists suggest that Qalsody could be more effective if administered early, potentially before symptoms manifest in patients with the SOD1 mutation.
