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.

  • – ALS drug wins FDA approval despite questionable data
  • – FDA panel backs much-debated ALS drug in rare, 2nd review
  • – ALS patients contend with $158K price tag on new drug

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.

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