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French Health Authority Rejects Early Access to Biogen's ALS Drug

The French health authority denied early access to Biogen's ALS drug Qalsody due to insufficient clinical trial data, sparking controversy and raising questions about its potential effectiveness.

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French

France

HealthHealth PolicyClinical TrialsDrug ApprovalPatient AdvocacyAls Treatment

HasBiogenArslaInsermInstitut Du Cerveau

Séverine BoilléeEmmanuel Macron

Why did the French HAS deny early access to Biogen's Qalsody drug?: The French National Authority for Health (HAS) denied early access to Biogen's Qalsody drug for a rare genetic form of amyotrophic lateral sclerosis (ALS), sparking outrage among patients.
How did patient advocacy groups and researchers react to the HAS's decision?: The HAS's decision was met with criticism from the ARSLA, who described it as "cruel and unacceptable." The HAS maintains that its decision was based on the available clinical trial data, which did not meet the criteria for early access.
What is the mechanism of action of Qalsody, and what type of ALS patients does it target?: Qalsody, containing tofersen, aims to slow ALS progression by targeting a specific gene mutation (SOD1). The HAS cited insufficient data from Biogen's Phase III trial showing a statistically significant benefit over placebo to justify early access.
What were the main reasons behind the HAS's decision, considering that Qalsody is approved in the US and EU?: While approved by US and EU authorities, the HAS's decision was based on the lack of robust evidence demonstrating Qalsody's effectiveness and the absence of a clinically significant biomarker improvement.
What is the future outlook for Qalsody's availability in France, and are there any perspectives on its potential effectiveness?: Although the initial Phase III trial did not demonstrate significant benefit, some experts believe early treatment with tofersen might be effective. Biogen is pursuing standard reimbursement pathways for Qalsody, and the HAS is awaiting further data.

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