FDA approves Ultragenyx gene therapy and Regeneron ultra-rare disease drug

The Food and Drug Administration gave the green light to a gene‑therapy product from Ultragenyx, marking a new treatment option in the company’s pipeline.

In a separate decision, Regeneron secured approval for a therapy targeting an ultra‑rare disease, underscoring the agency’s focus on niche indications.

The approvals were highlighted alongside other biotech developments, including a promising cancer vaccine from Merck and Moderna and the nomination of a new FDA commissioner, reflecting a busy period for the industry.