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Medicines which represented significant progress in their therapeutic areas have been listed in the European Medicines Agency's Human Medicines Highlights 2022 report.
European approval of BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec) one-time gene therapy represents a breakthrough in the treatment of adults with severe haemophilia A.
Roche shares initiative commitments that will extend prophylactic treatment access to patients with haemophilia, as well as investments to provide testing capabilities for HIV and Tuberculosis in developing countries.
Efanesoctocog alfa significantly and meaningfully reduces annualised bleeding rates in haemophilia A patients, according to new Phase III data.
Giroctocogene fitelparvovec had a mean annualised bleeding rate (ABR) of just 1.4 percent over two years, with no bleeding events in the first year post infusion.
The occurrence of non-fatal thrombotic events in Phase III study participants has called into question the safety of concizumab.
New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018.
New research has found that of the drugs in the pre-registration phase, 10 are expected to become blockbusters over the next six years.
The CMA has cleared the anticipated purchase of gene therapy company Spark Therapeutics by pharmaceutical company Roche Holdings.
Hemlibra has been approved to prevent the frequency of bleeding episodes in patients who have developed FVIII inhibitors...