Blog
RSS FeedPosted by Michael Wonder on 07 May 2019
European Commission approves Palynziq (pegvaliase injection) for treatment of phenylketonuria in patients aged 16 years or older
6 May 2019 - First enzyme substitution therapy approved in Europe to treat the underlying cause of phenylketonuria in patients aged ...
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22 March 2019 - The U.S. FDA today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development.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 12 Mar 2019
Vertex receives approval for Symdeko (tezacaftor/ivacaftor and ivacaftor) in Australia, to treat the underlying cause of cystic fibrosis in people aged 12 and older with certain CFTR gene mutations
12 March 2019 - A new treatment option for patients with two copies of the F508del mutation, the most common mutation ...
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 04 Mar 2019
Akcea and Ionis receive positive EU CHMP opinion for Waylivra (volanesorsen)
1 March 2019 - First and only therapy approved for FCS, a serious and rare disease with no approved treatment options. ...
Posted by Michael Wonder on 04 Mar 2019
BioMarin receives positive CHMP opinion in Europe for Palynziq (pegvaliase injection) for treatment of patients with phenylketonuria aged 16 and older
1 March 2019 - Decision on marketing authorisation application expected Q2 '19. ...
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. ...