Drug for rare form of Lou Gehrig’s disease OK’d by FDA

April 25, 2023 -WASHINGTON (AP) — Food and Drug Administration regulators on Tuesday approved a first-of-a-kind drug for a rare form of Lou Gehrig’s disease, though they are requiring further research to confirm it truly helps patients.

The FDA approved Biogen’s injectable drug for patients with a rare genetic mutation that’s estimated to affect less than 500 people in the U.S. It’s the first drug for an inherited form of ALS, or amyotrophic lateral sclerosis, a deadly disease that destroys nerve cells needed for basic functions like walking, talking and swallowing.

Approval came via FDA’s accelerated pathway, which allows drugs to launch based on promising early results, before they’re confirmed to benefit patients. That shortcut has come under increasing scrutiny from government watchdogs and congressional investigators.

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The FDA is requiring Biogen to continue studying the drug in a trial of people who carry the genetic mutation but do not yet have ALS symptoms.

ALS patients hope the decision could lay the groundwork for more expedited approvals to fight the disease, which affects 16,000 to 32,000 people in the U.S. The FDA has long used accelerated approval to speed the availability of drugs for cancer and other deadly conditions.