Rare diseases

News

The FDA has raised the pressure on Amgen to withdraw vasculitis drug Tavneos from the market with a report linking serious side effects to the drug.

News

The EU has approved UCB's Kygevvi for rare disease TK2d, and Sanofi's acute GvHD therapy Rezurock, in a pair of decisions announced this morning.

News

AstraZeneca's long-acting Strensiq follow-up efzimfotase alfa has hit the mark in children with rare disease HPP, but the data in adults is unclear.

News

Rocket Pharma has won FDA approval for the first gene therapy for LAD-I, a devastating genetic disorder causing severe vulnerability to infections.

News

Denali's Avlayah is the first drug for neurological complications associated Hunter syndrome to be approved in the US.

News

Rhythm Pharma has secured the first approval in the US for a drug to treat hypothalamic obesity.