News

National Institute for Health and Care Excellence2d
Awaiting development: Guidance and quality standards
trastuzumab or both together [TSID11781] Technology appraisal guidance UX111 for treating mucopolysaccharidosis type IIIA [ID6540] Technology appraisal guidance Valoctocogene roxaparvovec for treating ...
National Institute for Health and Care Excellence3d
Awaiting development: Guidance and quality standards
UX111 for treating mucopolysaccharidosis type IIIA [ID6540] Technology appraisal guidance Valoctocogene roxaparvovec for treating severe haemophilia A [ID3806] Technology appraisal guidance Venetoclax ...
pharmaphorum4d
BioMarin gene therapy for haemophilia on course for EU approval
The CHMP backed a conditional marketing authorisation for Roctavian (valoctocogene roxaparvovec or valrox) for severe haemophilia A in adults without inhibitors – antibodies that make standard ...
pharmaphorum4d
Roctavian okay sets up another gene therapy test for Europe
The US biotech claimed conditional marketing approval from the European Commission for Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe haemophilia A, who don’t have ...