UK – NICE recommends Santhera’s Duchenne muscular dystrophy drug Agamree
The drug has been recommended for use on the NHS in England, Wales and Northern Ireland to treat the rare muscle-wasting disorder in those...
Europe – First treatment for peripheral thyrotoxicosis in patients with Allan-Herndon-Dudley syndrome
EMA has recommended granting a marketing authorisation in the European Union (EU) for Emcitate (tiratricol), an oral treatment for peripheral thyrotoxicosis (inappropriately high levels of circulating...
Europe – Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP)...
Seventeen new medicines recommended for approval
EMA’s human medicines committee (CHMP) recommended seventeen medicines for approval at its December 2024 meeting. This brings the total...
Europe – First treatment recommended for rare progressive lung conditions in children and adolescents
EMA has recommended extending the indication of Ofev (nintedanib) to include the treatment of progressive fibrosing interstitial lung diseases (ILDs) in children and adolescents from the age of...
Europe – ICH Guideline M13A on bioequivalence for immediate-release solid oral dosage forms –...
ICH M13A Guideline is intended to provide recommendations on conducting bioequivalence (BE) studies during both development and post approval phases for orally administered immediate-release (IR) solid oral dosage forms...
Canada – Drug and medical device databases
This list of Health Canada's drug and medical device databases briefly describes the information available in each database. It does not provide an in...
UK – NICE endorses Neuraxpharm’s ublituximab for treating MS
Neuraxpharm Group has announced that the National Institute for Health and Care Excellence (NICE) has recommended ublituximab (BRIUMVI®) for the treatment of relapsing-remitting multiple...
UK – NICE recommends Immunocore’s Kimmtrak as first drug for aggressive eye cancer
Kimmtrak will now be available on the NHS in England to treat HLA-A*02:01-positive adults with unresectable or metastatic uveal melanoma, with more than 100...
USA – Researchers suggest FDA needs authority to require pediatric studies for orphan drugs
The US Food and Drug Administration (FDA) may need greater authority to require pediatric clinical studies for drugs, including orphan drugs, according to a...
USA – FDA finalizes BIMO electronic submission guidance
The US Food and Drug Administration (FDA) has finalized guidance for sponsors on electronically submitting data on drugs and biologics that the agency will...
