Blog
RSS FeedPosted 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 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 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 ...
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. ...
Posted by Michael Wonder on 19 Jun 2020
Dicerna receives rare paediatric disease designation from U.S. FDA for nedosiran for the treatment of primary hyperoxaluria
18 June 2020 - Dicerna Pharmaceuticals today announced that the U.S. FDA has granted rare paediatric disease designation for nedosiran, ...
Posted by Michael Wonder on 11 Jun 2020
Rare disease therapy development and access remain top FDA priorities during COVID-19
11 June 2020 - The FDA’s work on behalf of people with rare diseases is more important than ever as these ...
Posted by Michael Wonder on 03 Jun 2020
Clarity Pharmaceuticals announces that the US FDA grants rare paediatric disease designation to Copper Cu 67 sartate for the treatment of neuroblastoma
3 June 2020 - Clarity Pharmaceuticals is pleased to announce that the U.S. FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 26 Mar 2020
Gilead declines 'rare disease' status for experimental coronavirus drug
25 March 2020 - In a surprising turnabout, drugmaker Gilead Sciences asked the FDA on Wednesday to rescind orphan status ...
Posted by Michael Wonder on 26 Mar 2020
Orphan Technologies receives rare paediatric disease designation from FDA for OT-58 to treat cystathionine B-synthase deficiency homocystinuria
26 March 2020 - Orphan Technologies today announced that OT-58 for the treatment of cystathionine beta synthase deficiency homocystinuria has ...
Posted by Michael Wonder on 23 Feb 2020
Rare Disease Day 2020: FDA continues important work on treatments for rare diseases
21 February 2020 - As leaders of the FDA, every day we see the unwavering commitment of our agency in working ...