Novo Nordisk A/S has received a positive opinion from the European Medicines Agency (EMA) for its new treatment for haemophilia B, a genetic disorder caused by missing or defective factor IX, a blood clotting protein.
An antibody checkpoint inhibitor has become the first drug to be approved in the US for Merkel cell carcinoma, an aggressive form of skin cancer where fewer than half of patients survive more than one year.
Two years after launching in Europe, the Parkinson’s disease drug Xadago (safinamide) has been approved in the US as an add-on treatment to the combination therapy levodopa/carbidopa. The new approval means it will be available in both regions for treating symptoms of the disease such as tremor and impaired mobility.
Cell Medica Ltd has closed a £60 million Series C financing round to support its cell therapy platforms. The financing was announced not long after the company received a fast track designation from the US Food and Drug Administration for its lead oncology product baltaleucel-T.
President Donald Trump announced on 10 March that he intends to nominate Scott Gottlieb as head of the Food and Drug Administration. Mr Trump has chosen a medical doctor with considerable experience in the politics of medicine, often arguing on television and in print that increasing competition is the way to deal with the medical industry’s complex problems.
Kite Pharma Inc has published further positive data for its lead chimeric antigen receptor (CAR) T cell therapy for cancer, while confirming that its rolling application to the FDA to receive marketing approval should be complete by the end of the first quarter.
The US Food and Drug Administration has given regulatory clearance for a Phase 1 trial of Cellectis SA’s allogeneic, gene-edited cell therapy for the treatment of refractory acute lymphoblastic leukaemia (ALL). The product is being developed by Servier and Pfizer Inc.
A case study of a young patient with sickle cell disease who was successfully treated with an experimental gene therapy has been published in the 2 March 2017 edition of The New England Journal of Medicine. It is believed to be the first treatment to deliver substantial relief from a disease that can sometimes lead to an early death.
TxCell SA expects to start a clinical trial in late 2018 of a regulatory T cell product that has been engineered to contain a chimeric antigen receptor (CAR). The French company’s therapeutic focus is severe inflammatory and autoimmune diseases as well as organ transplant rejection.
Innate Pharma SA continued its strategy for attacking cancer by mobilizing cells in the innate immune system in 2016. Under an agreement with Bristol-Myers Squibb, the France-based company received a milestone payment of $15 million following progress in the development of lirilumab.