Blog
RSS FeedPosted 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. ...
Posted by Michael Wonder on 03 May 2018
RegenxBio receives FDA fast track designation for RGX-121 gene therapy for the treatment of mucopolysaccharidosis Type II
2 May 2018 - Phase I/II clinical trial expected to enrol children with MPS II. ...
Posted by Michael Wonder on 26 Apr 2018
Synlogic receives fast track designation for SYNB1618, a synthetic biotic medicine for the treatment of phenylketonuria
25 April 2018 - Phase 1/2a clinical study of SYNB1618 evaluating safety and tolerability as well as exploratory endpoints expected ...
Posted by Michael Wonder on 19 Apr 2018
Price tag for newly approved Crysvita is "responsible," says PBM Express Scripts
18 April 2018 - Taking the rebates that it expects to negotiate into consideration, Ultragenyx said that burosumab will cost approximately ...
Posted by Michael Wonder on 20 Mar 2018
FDA, industry ponder changes to clinical trials for rare disease treatments
19 March 2018 - FDA statisticians pondered changing how the agency uses statistics to approve drugs for rare disease at ...
Posted by Michael Wonder on 26 Feb 2018
Taking new steps to meet the challenges of rare diseases — FDA marks the 11th Rare Disease Day
26 February 2018 - Today 30 million people in the United States – or one out of every 10 Americans – ...
Posted by Michael Wonder on 22 Feb 2018
Developing anti-cancer drugs in orphan molecular entities — a paradigm under construction
22 February 2018 - Genomic characterisation of cancers has shown that some oncogenic alterations occur at very low frequency and are ...