Shire’s investigational Hunter syndrome therapy SHP609 has failed to meet primary and secondary targets in a mid-stage trial. More: Shire Hunter syndrome therapy fails to hit study targets Source: News
read moreSanofi Genzyme and Alnylam Pharmaceuticals have submitted a marketing application to the European Medicines Agency for patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of adults with hereditary transthyretin-mediated amyloidosis. More: Sanofi, Alnylam file hATTR amyloidosis therapy in
read morePatients in England and Wales will now be able to access Takeda’s myeloma treatment Ninlaro on the NHS, after cost regulators cleared its use through the Cancer Drugs Fund (CDF). More: Takeda’s Ninlaro approved for NHS use via CDF Source:
read moreThe National Institute for Health and Care Excellence has rejected Clinuvel UK’s afamelanotide for treating erythropoietic protoporphyria (EPP) in preliminary guidelines deeming it far too expensive for NHS use. More: NICE turns down rare skin disease implant Source: News
read moreBack in September, AstraZeneca’s Tagrisso posted data showing it could outdo the competition in treating first-line, EGFR mutation-bearing lung cancer patients. And now, the FDA is helping the drug speed toward an approval in that population. More: AstraZeneca snags Tagrisso
read moreAt a time when drugmakers—even rare disease drugmakers—routinely draw fire for their pricing decisions, Spark Therapeutics is surely prepping a pitch for its now-FDA-approved Luxturna. Analysts figure the pioneering therapy’s retail price could break the $1 million barrier. More: Spark
read more© 1994 - 2026 B.M. Pharmaceuticals