The FDA Peripheral and Central Nervous System Drugs Advisory Committee voted 9-0 that a reduction in plasma neurofilament light (NfL) concentration is “reasonably likely” to predict clinical benefit of tofersen for treatment of patients with SOD1-ALS. On a second question, the committee voted 5-3 (with one abstention) that the clinical data surrounding tofersen does not provide “convincing evidence” of effectiveness of tofersen in the treatment of patients with SOD1-ALS. These votes support FDA approving tofersen under the accelerated approval pathway.

In response, we issued the following statement:

“We thank the FDA Advisory Committee for their clear and compelling recommendation that tofersen be approved under the accelerated approval pathway. We urge the FDA to swiftly approve this urgently needed new treatment. Americans living with this rare and aggressive genetic form of ALS cannot wait.”

Several people living with ALS and leading ALS advocates spoke during a public forum in support of the first-of-its-kind drug treatment targeting a genetic cause of ALS.

“Knowing there is a treatment for this form of ALS, and better yet, potentially a way to prevent the disease from ever manifesting itself, would not only save me but also give my children the freedom to live their lives without this weight on their shoulders,” said Larry Falivena, who was diagnosed with SOD1-linked ALS.  “Tofersen is the opportunity to break the cycle of genetic ALS for families who have been devastated by the disease for generations.”

Calaneet Balas, President and CEO of The ALS Association, also testified at the hearing, and stated that the ALS Association only makes recommendations on drug approvals after conducting a thorough independent peer-review process. Based on this analysis, the Association believes tofersen meets all the conditions required for accelerated approval.

“We respectfully request you make a favorable recommendation to the FDA, supporting accelerated approval of tofersen,” Balas told the advisory committee. “People with SOD1-linked ALS and their health care providers should have full access to this drug as soon as possible.”

An FDA decision is expected by April 25.