Morning Briefing
Summaries of health policy coverage from major news organizations
FDA Authorizes Contentious ALS Drug That May Slow Disease Decline
The Food and Drug Administration on Thursday overcame doubts from agency scientists and approved a fiercely debated drug for ALS, a move that heartened patients and advocates who pushed for the medication but raised concerns among some experts about whether treatments for dire conditions receive sufficient scrutiny. “It’s a huge deal,” said Sunny Brous, 35, who was diagnosed with ALS seven years ago after she had trouble closing her left glove while playing softball. She plans to begin taking the drug as soon as she can. (McGinley, 9/29)
Since 1995, only two drugs have been approved to treat amyotrophic lateral sclerosis ― also known as Lou Gehrig’s disease ― and neither works very well. “This approval provides another important treatment option for ALS, a rare, life-threatening disease that currently has no cure,” said Dr. Billy Dunn, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. (Saltzman and Cross, 9/29)
The latest approval followed a remarkably turbulent path, including two negative reviews by the FDA’s internal scientists, who called the company’s results “borderline” and “not persuasive.” A panel of outside advisers backed that negative opinion in March, narrowly voting against the drug. But the FDA has faced intense pressure from ALS patients, advocates and members of Congress. In recent weeks the agency received more than 1,300 written comments from the ALS community supporting the treatment. (Perrone, 9/30)
The FDA's decision was based on a single phase 2 clinical trial of 137 ALS patients that found people who took Amylyx’s drug, which will be sold under the name Relyvrio, lived about 10 months longer than those who didn’t. The drug also appeared to delay hospitalizations. (Lovelace Jr., 9/29)
Amylyx did not immediately disclose how much it will charge for Relyvrio. “Amylyx’s goal is that every person who is eligible for Relyvrio will have access as quickly and efficiently as possible,” the company’s co-CEOs said in a statement, “as we know people with ALS and their families have no time to wait.” (Garde, 9/29)
