Pharmaceutical companies and manufacturers, including MilliporeSigma (Burlington, US), Simtra BioPharma Solutions (Parsippany, US), Carbogen AMCIS (Bubendorf, Switzerland), and BioNTech (Mainz, ...

As one FDA initiative works to sustain the momentum of rare disease research, other events threaten to slow progress.

The FDA has approved GSK's Shingrix vaccine (recombinant zoster vaccine or RZV) in a prefilled syringe presentation to prevent shingles.

Sarepta Therapeutics is set to cut 500 jobs as part of its restructuring and pipeline prioritisation plan, which is expected to result in annual cash cost savings of $120m in 2026. With its pipeline ...

Due to concerns surrounding its effectiveness, the US Food and Drug Administration (FDA) ended the Rare Pediatric Disease Designation (RPDD) programme in late 2024.