Here is a selection of this week's news from around the world
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UK researchers make new Alzheimer’s disease discovery Researchers at the Universities of St Andrews and Edinburgh say they have discovered the functions of the area of the brain in which Alzheimer’s begins, potentially opening the door to new approaches to treatment the disease. To read more follow this PharmaTimes link |
UniQure, Pfizer updates hint at gene therapy potential in hemophilia B ORLANDO — Gene therapies for the blood-clotting disorder hemophilia B are trailing about a year behind their hemophilia A counterparts, with pivotal study results from UniQure expected by the end of next year and Pfizer deep into its own Phase 3 trial. To read more follow this BioPharmaDive link |
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In stunning twist, FDA approves Sarepta's Duchenne drug it rejected The Food and Drug Administration approved Sarepta's Duchenne muscular dystrophy therapy Vyondys 53, four months after rejecting the drug over safety concerns. The decision comes as a major surprise, as Sarepta had given no prior hints that it had resubmitted an application to the agency. To read more follow this BioPharmaDive link |
Lilly launches first of its kind trial for RET fusion-positive NSCLC Eli Lilly has kicked off the first ever randomised Phase III study in treatment-naïve RET fusion-positive non-small cell lung cancer. The LIBRETTO-431 clinical trial is assessing selpercatinib, also known as LOXO-292, for treatment-naïve RET fusion-positive non-small cell lung cancer (NSCLC) patients. To read more follow this PharmaTimes link |
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SMC green light for rare blood cancer treatment People in Scotland with the rare blood cancer polycythaemia vera (PV) will soon gain access to a new treatment option on the NHS after cost regulators approved funding for Novartis’ Jakavi (ruxolitinib). To read more follow this PharmaTimes link |
Takeda sees cell, gene therapy in its future. Is it too late? Thanks to a $62 billion acquisition of Shire, Takeda is one of the world's largest developers of rare disease drugs. Despite that, the 238-year-old Japanese pharmaceutical company lacks any mid- or late-stage cell or gene therapies,....... To read more follow this BioPharmaDive
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