When the FDA recently accepted Biogen’s new drug application for tofersen, it signaled hope for thousands of people with SOD1 mutations that cause ALS. The FDA has granted priority review to this new drug application and is expected to decide on whether or not to approve tofersen by January 25, 2023.
New long-term analysis published in the Journal of Neurology, Neurosurgery & Psychiatry showed AMX0035 was effective at reducing a variety of harms associated with ALS during the previously published Phase 2 Centaur trial.
Expanded Access, or “compassionate use” as it is often referred, allows patients with a terminal diagnosis early access to new therapeutics that show promise – even if the patient is not involved in the ongoing clinical trial – or if the medication has not yet been approved by the FDA.
Earlier today the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6 to 4 against recommending AMX0035 for approval to treat people with ALS. It’s important to note that the Advisory Committee’s views are not binding on the FDA. Following the vote, the ALS Association called on the FDA to take into account the strong safety profile of AMX0035, as well as the serious unmet medical need of people living with this devastating condition, and approve the drug for clinical use.
It is on us—those who have experienced this disease firsthand, those of us who are living with the disease, those who are serving as caregivers—to determine the value and quality of life with ALS. We need to stand up and object to discriminatory assessments that overlook the most important things that give life value.