Blog
RSS FeedPosted by Michael Wonder on 28 Nov 2020
Alnylam announces innovative value-based agreement framework for Oxlumo (lumasiran) to accelerate access for patients with primary hyperoxaluria type 1 and deliver ultra-rare orphan disease pricing solutions to U.S. payers
24 November 2020 - Expedited access to Oxlumo aims to support children and adults living with PH1 who face inevitable disease ...
Posted by Michael Wonder on 24 Nov 2020
FDA approves first drug to treat rare metabolic disorder
23 November 2020 - Today, the U.S. FDA approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1, a ...
Posted by Michael Wonder on 23 Nov 2020
Workshop on regulatory support for development of orphan medicines
23 November 2020 - On Monday, 30 November, EMA is hosting a workshop to discuss the benefits and impact of ...
Posted by Michael Wonder on 21 Nov 2020
FDA approves first treatment for Hutchinson-Gilford progeria syndrome and some progeroid laminopathies
20 November 2020 - Today, the U.S. FDA approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford ...
Posted by Michael Wonder on 10 Nov 2020
Mirum Pharmaceuticals broadens Expanded Access Program for maralixibat in Alagille syndrome to Europe and Australia
5 November 2020 - Maralixibat Expanded Access Program now available for patients with pruritus associated with Alagille syndrome in Australia and ...
Posted by Michael Wonder on 29 Oct 2020
reVision Therapeutics announces US FDA grant of rare paediatric disease and orphan drug designation for REV-0100 for the treatment of Stargardt disease
28 October 2020 - reVision Therapeutics today announced that the US FDA has granted the Company's request to designate REV-0100 as ...
Posted by Michael Wonder on 27 Oct 2020
Prevail Therapeutics receives U.S. FDA fast track designation for PR001 for the treatment of neuronopathic Gaucher disease
27 October 2020 - Prevail Therapeutics today announced that the U.S. FDA has granted fast track designation for the Company’s ...
Posted by Michael Wonder on 29 Sep 2020
The estimation of health state utility values in rare diseases: overview of existing techniques
25 September 2020 - There are several techniques for estimating health state utility values, each of which presents pros and cons ...
Posted by Michael Wonder on 18 Sep 2020
NICE U-turn sees Akcea's Waylivra win NHS funding
18 September 2020 - Akcea Therapeutics UK's Waylivra (volanesorsen), the first and only therapy for Familial Chylomicronaemia Syndrome (FCS), will ...
Posted by Michael Wonder on 16 Sep 2020
FDA granted paediatric disease designation for OXi-4503
16 September 2020 - Treatment of acute myeloid leukaemia due to genetic mutations that disproportionately affect paediatric patients. ...
Posted by Michael Wonder on 16 Sep 2020
Orphazyme announces U.S. FDA acceptance and priority review of new drug application for arimoclomol for Niemann-Pick disease Type C
16 September 2020 - If approved, arimoclomol would become the first approved therapy in the U.S. for people with Niemann-Pick disease ...
Posted by Michael Wonder on 08 Sep 2020
South Korea to shorten licensing review period new drugs on rare diseases by 25 days
8 September 2020 - Korean health regulators will shorten licensing review period from 115 days to 90 days for investigational ...
Posted by Michael Wonder on 13 Aug 2020
Impact of rarity on Canadian oncology health technology assessment and funding
11 August 2020 - The pan-Canadian Oncology Drug Review evaluates new cancer drugs for public funding recommendations. ...
Posted by Michael Wonder on 12 Aug 2020
Rare disease drugs strategy, standard coverage in CLHIA’s 2020 budget wish list
12 August 2020 - The Canadian Life and Health Insurance Association is recommending the federal government develop a strategy for ...
Posted by Michael Wonder on 20 Jul 2020
Rare disease drove two women on a mission to change FDA drug review process
19 July 2020 - Melissa Goetz and Lindsey Sutton are campaigning to improve the US drug review process for rare diseases. ...