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RSS FeedPosted by Michael Wonder on 19 Jun 2019
Abeona Therapeutics receives FDA fast track designation for ABO-202 AAV9 gene therapy in CLN1 disease
18 June 2019 - Abeona Therapeutics today announced that the U.S. Food and FDA has granted fast track designation to its ...
Posted by Michael Wonder on 11 Jun 2019
Jacobus prices its rare disease drug at half of what Catalyst charges, but will doctors prescribe it?
10 June 2019 - After weeks of anticipation, Jacobus Pharmaceutical, a small, family-run drug maker, has priced its rare disease ...
Posted by Michael Wonder on 06 Jun 2019
Enzyvant announces FDA acceptance of biologics license application and priority review status for RVT-802, a novel investigational tissue-based regenerative therapy for paediatric congenital athymia
5 June 2019 - RVT-802, a one-time therapy, leverages Enzyvant’s T cell generation platform designed to treat profound immunodeficiencies. ...
Posted by Michael Wonder on 04 Jun 2019
US FDA review for Scenesse extended by three months
3 June 2019 - The FDA sets new goal date of 6 October 2019 to complete review and issue risk-benefit decision. ...
Posted by Michael Wonder on 08 May 2019
FDA undercuts $375,000 drug in surprise move
8 May 2019 - The US FDA created a workaround this week that effectively undercuts the $375,000 price tag of ...
Posted by Michael Wonder on 23 Apr 2019
Priority review vouchers have helped to improve access to drugs targeting neglected diseases
22 April 2019 - The FDA priority review vouchers for neglected tropical diseases are aimed to incentivise pharmaceutical companies to ...
Posted 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22 March 2019 - The U.S. FDA today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development.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 ...