16 October 2020 - If approved, inclisiran will be the first and only small interfering RNA (siRNA) in Europe for patients ...
16 October 2020 - EMA has recommended granting a marketing authorisation in the European Union for Oxlumo (lumasiran) for the ...
2 October 2020 - European Medicines Agency will evaluate eli-cel application under accelerated assessment. ...
23 September 2020 - EMA’s PRIME program designed to optimise development and expedite evaluation of innovative medicines for patients with high ...
15 September 2020 - First marketing authorisation application for a gene therapy treating a mitochondrial disease. ...
10 September 2020 - European regulators have requested more data to review the marketing application for BioMarin's haemophilia A gene ...
19 May 2020 - Zolgensma (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy ...
28 March 2020 - AveXis, a Novartis company, today announced that the CHMP of the EMA has adopted a positive ...
3 March 2020 - Givlaari approved based on ENVISION Phase 3 study results showing a 74% reduction in the rate of ...
2 March 2020 - PRIME designation granted based on clinical data from ongoing Phase 1/2 trial of AAV-RPGR. ...
31 January 2020 - Positive opinion is based on data from the pivotal ENVISION Phase 3 study. ...
31 January 2020 - Fifteen new medicines recommended for approval. ...
13 January 2020 - PTC-AADC MAA submitted; BLA now expected to be submitted in 2Q 2020. ...
23 December 2019 - Potential first gene therapy in Europe directed at any type of haemophilia. ...
16 December 2019 - Milestone gives Fanconi anaemia program all accelerated regulatory tools in U.S. and EU, including FDA regenerative medicine ...