News CMS extends deadline for MFN pricing pilot scheme CMS has extended the deadline for its Medicaid drug pricing pilot, GENEROUS, to "provide interested drug manufacturers more opportunity to join."
News New FDA knockback hits uniQure and Huntington patients hard uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, delaying the highly anticipated programme.
News CHMP backs Ipsen drug for childhood brain cancer Ipsen's Ojemda is on course to become the first targeted medicine in the EU for some children with a form of brain cancer known as low-grade glioma.
News NHS "isn't delivering equitable care for rare diseases" A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.
News Moderna closes on EU approval for combined flu/COVID jab Moderna's combined vaccine for both flu and COVID-19 has been recommended for approval in the EU, after being knocked back in the US.
News Boehringer gets FDA's second 'national priority' approval Boehringer Ingelheim's lung cancer drug Hernexeos has become the second medicine approved after an FDA national priority review.
News Trial miss doesn't dent Biogen's faith in Alzheimer's drug Biogen will advance its tau-targeting Alzheimer's disease drug diranersen into phase 3, even though it missed the main endpoint in a midstage trial.
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