ALS: FDA Considering Approval of Drug Tofersen for Rare Form of Disease

April 11, 2023

For months, advocates have lobbied the FDA to approve the drug before the phase 3 trial ended.

Thousands of people with ALS and their family members have emailed the agency urging approval of AMX0035 through a campaign organized by The ALS Association.

In May, a group of 38 doctors and scientists signed off on a letter to the FDA also urging the agency to approve the drug. The clinicians cited the short life expectancy of people with ALS and that many would die waiting for the results of a phase 3 clinical trial.

Jinsy Andrews, MD, MSc

Jinsy Andrews, MD, MSc

“I had a patient die (in June). He had been hoping to participate in an expanded access program,” said Dr. Jinsy Andrews, a professor of neurology at Columbia University in New York.

“There are people who just want access to new drugs because they’ve exhausted all other options… and they’re not going to survive until the end of another study,” she told Healthline.

“It’s important because we haven’t had many successes in ALS” she added. “As these applications are coming in and challenging how we look at the clinical trial data, it’s pushing our field as a whole to think about what’s our minimum level of evidence to have access to a therapy.” [read more]

Source: Healthline