Blog
RSS FeedPosted by Michael Wonder on 28 Mar 2019
Drug makers continue to clamour for FDA approval of orphan drugs
28 March 2019 - Orphan drugs may serve small groups of patients, but they generate big numbers at the Food ...
Posted by Michael Wonder on 23 Mar 2019
FDA takes new steps to advance natural history studies for accelerating novel treatments for rare diseases
22 March 2019 - The U.S. FDA today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development. ...
Posted by Michael Wonder on 06 Mar 2019
FDA grants rare paediatric disease designation to odiparcil for the treatment of MPS VI
5 March 2019 - Inventiva eligible to receive priority review voucher upon approval of odiparcil for the treatment of MPS VI. ...
Posted by Michael Wonder on 05 Mar 2019
FDA is working to bridge gaps and meet needs for rare disease product development
28 February 2019 - There are approximately 7,000 rare diseases affecting an estimated 30 million people in the United States. ...
Posted by Michael Wonder on 25 Feb 2019
U.S. FDA grants breakthrough therapy designation to Amicus’ AT-GAA in late onset Pompe disease
25 February 2019 - First breakthrough therapy for an investigational treatment in Pompe disease. ...
Posted by Michael Wonder on 23 Feb 2019
FDA grants priority review and accepts sBLA of Soliris (eculizumab) as a treatment for patients with neuromyelitis optica spectrum disorder
22 February 2019 - FDA sets action date of 28 June 2019. ...
Posted by Michael Wonder on 21 Feb 2019
Cerecor receives fast track designation from FDA for CERC-801 for the treatment of PGM1 deficiency
19 February 2019 - Cerecor announced today that the U.S. FDA has designated fast track designation for CERC-801, an ultra-pure, oral ...
Posted by Michael Wonder on 15 Feb 2019
Sarepta announces FDA acceptance of golodirsen (SRP-4053) new drug application for patients with Duchenne muscular dystrophy amenable to skipping exon 53
14 February 2019 - FDA grants priority review status. ...
Posted by Michael Wonder on 17 Jan 2019
FDA updates guidance on development of rare disease therapies
17 January 2019 - The FDA has revised its draft guidance for developing rare disease treatments to include the use ...
Posted by Michael Wonder on 14 Jan 2019
US FDA accepts regulatory submissions for review of tafamidis to treat transthyretin amyloid cardiomyopathy
14 January 2019 - FDA grants a priority review based on Phase 3 ATTR-ACT study findings. ...
Posted by Michael Wonder on 11 Jan 2019
US FDA sets PDUFA date for Scenesse
10 January 2019 - FDA does not plan to hold advisory committee meeting. ...
Posted by Michael Wonder on 06 Dec 2018
Federal officials call out FDA over lapses in rare-disease drug approval
5 December 2018 - The 1983 Orphan Drug Act motivated pharmaceutical companies to develop drugs for people who lacked treatments ...
Posted by Michael Wonder on 03 Dec 2018
FDA faulted for lapses in orphan drug program
30 November 2018 - The FDA has failed to ensure that drugs given prized rare-disease status meet the intent of ...
Posted by Michael Wonder on 01 Dec 2018
‘A loss for the rest of us’: An FDA approval is a boon for a drug maker, but could come at a major cost for patients
30 November 2018 - Vickie Moored can walk again. Her words don’t slur, her vision isn’t blurred, and she no ...
Posted by Michael Wonder on 29 Nov 2018
FDA approves first treatment for Lambert-Eaton myasthenic syndrome, a rare auto-immune disorder
28 November 2018 - The U.S. FDA today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome in adults. ...