Blog
RSS FeedPosted by Michael Wonder on 28 Apr 2021
Biogen to expand access to its ALS drug, but move may come too late for some patient
27 April 2021 - After weeks of controversy, Biogen has agreed to provide an experimental drug for combating ALS to ...
Posted by Michael Wonder on 20 Apr 2021
Alnylam submits new drug application with U.S. FDA for vutrisiran for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults
19 April 2021 - Alnylam has submitted a new drug application to the U.S. FDA for the approval of vutrisiran for ...
Posted by Michael Wonder on 11 Mar 2021
Denali Therapeutics announces fast track designation granted by the U.S. FDA to ETV:IDS (DNL310) for the treatment of patients with Hunter syndrome
11 March 2021 - Denali Therapeutics today announced that the U.S. FDA has granted fast track designation to ETV:IDS (DNL310) for ...
Posted 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 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 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 ...