8 April 2024 - If approved, elamipretide would become the first approved therapy for Barth syndrome. ...
13 February 2024 - Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent ...
8 January 2024 - We found that 491 novel orphan drugs were approved between 1990 and 2022. ...
31 October 2023 - FDA sets a PDUFA target action date of 30 April 2024. ...
30 October 2023 - New rules for the reimbursement of orphan drugs and other unlisted medicines will become effective in Switzerland ...
27 October 2023 - Egetis Therapeutics today announced that its marketing authorisation application to the EMA for Emcitate (tiratricol) for ...
26 October 2023 - Catalyst expects to commercially launch Agamree in Q1, 2024. ...
23 October 2023 - Ipsen plans to submit a new marketing authorisation application for the treatment of Alagille syndrome by the ...
29 September 2023 - Today, the US FDA is taking steps to help further accelerate the development of novel drug and ...
18 August 2023 - Approval represents tenth FDA approved medicine invented by Regeneron. ...
9 May 2023 - The objective of this study was to analyse the US FDA approval, trials, unmet needs, benefit, and ...
9 May 2023 - In Europe, the number of approvals for new drugs targeting rare diseases (orphan drugs) has risen ...
27 March 2023 - CHMP opinion for miglustat, the oral enzyme stabiliser component of AT-GAA, on track for 2Q, 2023. ...
10 March 2023 - Company expects Daybue to be available by the end of April 2023. ...
7 March 2023 - Genethon today announced that the EMA has granted PRIME (Priority Medicines) status to the gene therapy, ...