25 June 2021 - Temporary Authorization for Use (ATU) granted in France to allow access and reimbursement of vosoritide to begin ...
28 April 2021 - New indication includes people ages 12 years and older who have one copy of the F508del mutation ...
23 November 2020 - On Monday, 30 November, EMA is hosting a workshop to discuss the benefits and impact of ...
5 November 2020 - Maralixibat Expanded Access Program now available for patients with pruritus associated with Alagille syndrome in Australia and ...
10 December 2019 - Ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic ...
31 October 2019 - Application supported by positive results from two global Phase 3 studies in people with cystic fibrosis ages ...
27 August 2019 - 98% of adult anti-aquaporin-4 antibody-positive patients treated with Soliris were relapse free compared to 63% receiving placebo ...
3 July 2019 - Ultomiris is the first approved, long-acting complement inhibitor for PNH, administered every other month, reducing the ...
7 May 2019 - Akcea Therapeutics and Ionis Pharmaceuticals announced today that Waylivra has received conditional marketing authorisation from the ...
6 May 2019 - First enzyme substitution therapy approved in Europe to treat the underlying cause of phenylketonuria in patients aged ...
1 March 2019 - First and only therapy approved for FCS, a serious and rare disease with no approved treatment options. ...
1 March 2019 - Decision on marketing authorisation application expected Q2 '19. ...
22 February 2019 - FDA sets action date of 28 June 2019. ...
29 November 2018 - Ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic ...
1 November 2018 - First medicine in the EU to treat the CFTR protein defect in patients who have one copy ...