We are pleased that the FDA has granted accelerated approval to tofersen for treatment of SOD1-ALS. As the first FDA-approved treatment to target a genetic cause of ALS, this is a landmark moment for the ALS community.
We are grateful to the people living with SOD1-ALS who participated in the clinical trials for tofersen and their caregivers; to the donors who make it possible for our Lois Insolia ALS Clinic at the Les Turner ALS Center at Northwestern Medicine to offer access to clinical trials such as these; and to Biogen for its commitment to the drug’s development. We also thank the FDA for their careful consideration of the evidence.
Study results suggest that reduction of neurofilament levels predicts slower progression of the disease, and that an early start and extended use of tofersen, now known as Qalsody™, may help stabilize muscle strength, respiratory function and quality of life.
Those words are critical: an early start and extended use.
As such, while today’s decision is cause for celebration, it is also a call for urgency. People living with SOD1-ALS must be able to access this treatment without delay. SOD1-ALS has a median survival of 2.7 years, which falls to 1.2 years for people with the faster-progressing A4V mutation.
People living with SOD1-ALS have no time to waste.
Related:
