3 June 2019 - The FDA sets new goal date of 6 October 2019 to complete review and issue risk-benefit decision. ...
8 May 2019 - The US FDA created a workaround this week that effectively undercuts the $375,000 price tag of ...
22 April 2019 - The FDA priority review vouchers for neglected tropical diseases are aimed to incentivise pharmaceutical companies to ...
28 March 2019 - Orphan drugs may serve small groups of patients, but they generate big numbers at the Food ...
22 March 2019 - The U.S. FDA today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development. ...
5 March 2019 - Inventiva eligible to receive priority review voucher upon approval of odiparcil for the treatment of MPS VI. ...
28 February 2019 - There are approximately 7,000 rare diseases affecting an estimated 30 million people in the United States. ...
25 February 2019 - First breakthrough therapy for an investigational treatment in Pompe disease. ...
22 February 2019 - FDA sets action date of 28 June 2019. ...
19 February 2019 - Cerecor announced today that the U.S. FDA has designated fast track designation for CERC-801, an ultra-pure, oral ...
14 February 2019 - FDA grants priority review status. ...
17 January 2019 - The FDA has revised its draft guidance for developing rare disease treatments to include the use ...
14 January 2019 - FDA grants a priority review based on Phase 3 ATTR-ACT study findings. ...
10 January 2019 - FDA does not plan to hold advisory committee meeting. ...
5 December 2018 - The 1983 Orphan Drug Act motivated pharmaceutical companies to develop drugs for people who lacked treatments ...