Blog
RSS FeedPosted by Michael Wonder on 01 Mar 2021
Rare Disease Day 2021: FDA shows sustained support of rare disease product development during the public health emergency
1 March 2021 - Rare Disease Day is a time to reflect on both the progress that has been made, and ...
Posted by Michael Wonder on 26 Jan 2021
Recordati Rare Diseases: Carbaglu (carglumic acid) receives U.S. FDA approval for a new indication to treat acute hyperammonaemia associated with propionic acidemia and methylmalonic acidemia
26 January 2021 - Carbaglu is first and only FDA approved medication for hyperammonaemia associated with these rare conditions. ...
Posted by Michael Wonder on 11 Jan 2021
Evaluating New Zealanders’ values for drug coverage decision-making: trade-offs between treatments for rare and common conditions
8 January 2021 - The objectives of this study were to measure the relative societal importance of values of New Zealanders ...
Posted by Michael Wonder on 02 Dec 2020
IQWiG publishes 1,000th report
2 December 2020 - In the 16th year of its existence, IQWiG has published its 1,000th report: a dossier assessment ...
Posted by Michael Wonder on 02 Dec 2020
Amicus Therapeutics initiates rolling biologic license application to the U.S. FDA for AT-GAA in late-onset Pompe disease
1 December 2020 - On track for completing the BLA submission in 1H2021. ...
Posted 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. ...