Rare diseases

News

Stealth BioTherapeutics' filing for Barth syndrome therapy elamipretide has been turned down by the FDA, but there may be a way forward for the drug.

News

Savara shares are pummelled after the FDA says it cannot accept a filing for lead drug Molbreevi, but it is still hopeful of an approval in 2026.

News

Thousands of people living with rare kidney disease IgAN in the UK will soon be able to access a new oral treatment from CSL Vifor.

News

MSD's oral HIF-2 alpha inhibitor Welireg has a third FDA approval, becoming the first oral treatment for a rare solid tumour PPGL or 'pheo para'.

News

Abeona Therapeutics' Zevaskyn has been approved in the US as a treatment for the rare skin disorder recessive dystrophic epidermolysis bullosa (DEB).

News

Merck KGaA has reached an agreement with SpringWorks on a $47-per-share takeover deal that values the US biotech at $3.9 billion.