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

The prefilled syringe formulation is approved in the US for the vaccination of adults aged 50 and older,and individuals aged 18 and above at greater risk. Credit: Spino Kim/Shutterstock.com. The US ...

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.

Thermo Fisher Scientific has signed an agreement for the acquisition of Sanofi’s steriles manufacturing site in Ridgefield, New Jersey, US.

FDA reviewers have identified high rates of ocular toxicity as GSK looks to place its blood cancer drug back on the market.

In a recent webinar, industry experts explain how real-world data and patient-centric support can shape regulatory success ...

Otsuka Pharmaceutical has signed an agreement to acquire all assets related to Cantargia's early-clinical stage CAN10 programme.

LEO has plans for Spevigo beyond generalised pustular psoriasis as it looks to boost its dermatological pipeline.

Vertex Pharmaceuticals has announced a reimbursement agreement with NHS England for its cystic fibrosis (CF) medicine, Alyftrek.

Ultragenyx Pharmaceuticals has announced the extension of approval by Health Canada for Evkeeza (evinacumab) for HoFH.