The upcoming Clinical Trials in Rare Diseases conference will cover the most concerning challenges in rare disease research.

Before Novo arrived as its owner, issues at the Bloomington facility caused a two-month delay for Eylea HD’s FDA approval decision in 2023. In November 2024, the FDA also found quality control lapses ...

Padcev-Keytruda will now join Bristol Myers Squibb’s Opdivo in the list of bladder cancer drugs available on the NHS.

Keros received orphan drug designation from the US FDA for its investigational therapy KER-065 to treat Duchenne muscular dystrophy (DMD).

Madrigal Pharmaceuticals’ Rezdiffra has been approved in Europe for the treatment of MASH with moderate to advanced liver ...

Biotechnology company āshibio has entered an exclusive licensing agreement with Mereo BioPharma for the monoclonal antibody, ...

The FDA has rejected vatiquinone, requesting that PTC run an additional study to prove the drug's efficacy prior to ...

Novo is far from the only big pharma company bolstering DTC medicine offerings. Novo’s main rival in the GLP-1RA arena, Eli ...

In June 2022, Enanta filed a lawsuit against Pfizer in a US district court in Massachusetts, claiming that the big pharma ...

Global orphan drug sales are on the rise. To dodge potential pitfalls associated with reaching ultra-rare populations, ...

The European Commission (EC) has granted marketing authorisation to SpringWorks Therapeutics, a subsidiary of Merck KGaA, for ...

Ovarian cancer is one of the most challenging malignancies in oncology. Despite advances in diagnosis and treatment, it is ...