USA – FDA offers guidance on observational studies as RWE
The US Food and Drug Administration (FDA) this week issued new draft guidance on study design and analysis for non-interventional studies being included as...
USA – FDA approves safety labeling changes regarding DPD deficiency for fluorouracil injection products
On March 21, 2024, the Food and Drug Administration approved safety labeling changes for fluorouracil injection products. This effort was a collaboration between FDA’s...
USA – FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy
Today, the U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years...
USA – Industry clamors for clarity on FDA’s advanced manufacturing designation program
Industry is welcoming FDA’s upcoming advanced manufacturing technologies (AMT) program but is clamoring for more clarity on crucial details like what methods will be...
Europe – Environmental risk assessment of medicinal products for human use
It is mandatory for a marketing authorisation application (MAA) for a medicinal product for human use (HMP) to include an environmental risk assessment (ERA)....
Europe – Accelerating stakeholder collaboration to enhance the clinical trials environment in the EU
The Accelerating Clinical Trials in the EU (ACT EU) initiative has established a multi-stakeholder platform (MSP) aimed at improving the environment for clinical trials across the European Union (EU). ACT...
USA – FDA proposes rule for difficult-to-compound drugs, drug categories
The US Food and Drug Administration (FDA) is proposing the creation of two lists of drugs or drug categories that are difficult to compound...
UK – New treatment option available today for womb cancer
540 adults will have access to Dostarlimab (also called Jemperli and made by GSK). Dostarlimab is a monoclonal antibody and helps the immune system...
Europe – EU pharmaceutical policy: MEPs support comprehensive reform
On Tuesday, the Environment, Public Health and Food Safety Committee adopted its position on the new directive (66 votes in favour, two against and...
USA – FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy
Today, the U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic...
