WASHINGTON – The Food and Drug Administration published draft guidance on Tuesday that would streamline approvals of some individualized gene therapies, a win for rare disease advocates who have criticized the agency’s approach to these treatments.
“It’s sort of putting a flag in the ground and saying, ‘This is important,’” Paul Scribner, vice president of community programs and education at the PKD Foundation, said of the draft guidance at a rare disease event on Tuesday.
Breakthroughs in gene therapies, or treatments that treat or cure diseases by altering faulty genes inside a person’s cells, have energized researchers and companies who believe many patients with rare mutations could benefit from these personalized medicines.