Blog
RSS FeedPosted by Michael Wonder on 06 Oct 2018
FDA approves Revcovi, a new enzyme replacement therapy developed by Leadiant Biosciences, for the treatment of ADA-SCID in paediatric and adult patients
5 October 2018 - Leadiant Biosciences today announced that the FDA has granted approval to Revcovi (elapegademase-lvlr) injection in the ...
Posted by Michael Wonder on 19 Sep 2018
Can patient advocates help make Akcea the next Sarepta and overcome the FDA’s rejection?
18 September 2018 - Patients with a rare disease are asking the FDA a question the agency rarely hears: Is ...
Posted by Michael Wonder on 15 Sep 2018
Single FDA centre fo rare diseases could be 'negative' to rare disease cause
14 September 2018 - FDA's Center for Drug Evaluation and Research Director Janet Woodcock advised against a single FDA Centre of ...
Posted by Michael Wonder on 21 Aug 2018
Audentes announces regenerative medicine advanced therapy designation granted by the FDA to AT132 for the treatment of X-linked myotubular myopathy
21 August 2018 - Designation granted based on positive interim data from ASPIRO clinical trial. ...
Posted by Michael Wonder on 21 Aug 2018
FDA accepts priority review of ALXN1210 as a treatment for patients with paroxysmal nocturnal haemoglobinuria in the US
20 August 2018 - EU filing accepted and under review. ...
Posted by Michael Wonder on 16 Aug 2018
FDA approves Kalydeco (ivacaftor) as first and only medicine to treat the underlying cause of CF in children ages 12 to <24 months with certain mutations in the CFTR gene
15 August 2018 - Data from Phase 3 ARRIVAL study support treatment with Kalydeco in children ages 12 to <24 months. ...
Posted by Michael Wonder on 13 Aug 2018
Amicus sets $315,000 price for new Fabry disease treatment
13 August 2018 - Amicus Therapeutics on Monday set an average price of $315,000 per year for its newly approved Fabry ...
Posted by Michael Wonder on 11 Aug 2018
FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease
10 August 2018 - The U.S. FDA today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused ...
Posted by Michael Wonder on 10 Aug 2018
FDA approves new treatment for a rare genetic disorder, Fabry disease
10 August 2018 - The U.S. FDA today approved Galafold (migalastat), the first oral medication for the treatment of adults with ...
Posted by Michael Wonder on 31 Jul 2018
Soleno Therapeutics receives fast track designation from FDA for DCCR for treatment of Prader-Willi syndrome
30 July 2018 - Phase III clinical trial ongoing at multiple sites in the U.S. ...
Posted by Michael Wonder on 26 Jun 2018
Historic new drug application for the use of Scenesse in rare metabolic disorder EPP
25 June 2018 - Priority review requested to FDA. ...
Posted by Michael Wonder on 20 Jun 2018
Alexion submits application for priority review and approval of ALXN1210 as a treatment for patients with paroxysmal nocturnal haemoglobinuria in the U.S.
19 June 2018 - Submission in the European Union on track for mid-year and in Japan for the second half of ...
Posted by Michael Wonder on 07 Jun 2018
FDA grants rare paediatric disease designation to Cellectar Biosciences’ CLR 131 for the treatment of rhabdomyosarcoma
6 June 2018 - Cellectar Biosciences announces today that the U.S. FDA has granted rare paediatric disease designation to CLR 131, ...
Posted by Michael Wonder on 25 May 2018
U.S. FDA grants fast track designation for Krystal Biotech’s KB103 for the treatment of dystrophic epidermolysis bullosa
24 May 2018 - Krystal Biotech today announces that the U.S. FDA has granted fast track designation to KB103 for ...
Posted by Michael Wonder on 25 May 2018
BioMarin receives standard approval for Palynziq (pegvaliase-pqpz) injection for treatment of adults with phenylketonuria, a rare enetic Disease
24 May 2018 - First enzyme therapy to treat phenylketonuria. ...