Blog
RSS FeedPosted by Michael Wonder on 26 Apr 2024
FDA grants Medivir´s MIV-711 rare paediatric disease designation and orphan drug designation for the treatment of Legg-Calvé-Perthes Disease
25 April 2024 - Medivir announced today that its selective cathepsin K inhibitor, MIV-711, has been granted rare paediatric disease designation ...
Posted by Michael Wonder on 08 Apr 2024
Stealth BioTherapeutics announces FDA acceptance of new drug application for elamipretide for the treatment of Barth syndrome
8 April 2024 - If approved, elamipretide would become the first approved therapy for Barth syndrome. ...
Posted by Michael Wonder on 09 Jan 2024
Orphan drug label expansions: analysis of subsequent rare and common indication approvals
8 January 2024 - We found that 491 novel orphan drugs were approved between 1990 and 2022. ...
Posted by Michael Wonder on 01 Nov 2023
X4 Pharmaceuticals announces FDA acceptance with priority review of US NDA for mavorixafor in WHIM syndrome
31 October 2023 - FDA sets a PDUFA target action date of 30 April 2024. ...
Posted by Michael Wonder on 27 Oct 2023
Catalyst Pharmaceuticals reports FDA approval of Agamree (vamorolone) for Duchenne muscular dystrophy granted to Santhera Pharmaceuticals
26 October 2023 - Catalyst expects to commercially launch Agamree in Q1, 2024. ...
Posted by Michael Wonder on 29 Sep 2023
FDA launches pilot program to help further accelerate development of rare disease therapies
29 September 2023 - Today, the US FDA is taking steps to help further accelerate the development of novel drug and ...
Posted by Michael Wonder on 19 Aug 2023
Veopoz (pozelimab-bbfg) receives FDA approval as the first treatment for children and adults with CHAPLE disease
18 August 2023 - Approval represents tenth FDA approved medicine invented by Regeneron. ...
Posted by Michael Wonder on 11 May 2023
FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis
9 May 2023 - The objective of this study was to analyse the US FDA approval, trials, unmet needs, benefit, and ...
Posted by Michael Wonder on 11 Mar 2023
Acadia Pharmaceuticals announces US FDA approval of Daybue (trofinetide) for the treatment of Rett syndrome in adult and paediatric patients two years of age and older
10 March 2023 - Company expects Daybue to be available by the end of April 2023. ...
Posted by Michael Wonder on 28 Feb 2023
My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility.
28 February 2023 - My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the ...
Posted by Michael Wonder on 23 Feb 2023
FDA continues important work to advance medical products for patients with rare diseases
23 February 2023 - Patients with rare diseases are experts in their health condition. They provide a unique perspective, and ...
Posted by Michael Wonder on 17 Feb 2023
Chiesi Global Rare Diseases announces FDA approval of Lamzede (velmanase alfa-tycv) for alfa mannosidosis
16 February 2023 - First and only enzyme replacement therapy for the treatment of non-central nervous system manifestations of alfa mannosidosis ...
Posted by Michael Wonder on 23 Jan 2023
Despite court ruling, FDA will continue with its approach to approving orphan drug exclusivity
23 January 2023 - In an unexpected move, the FDA will continue to apply exclusive marketing rights for so called ...
Posted by Michael Wonder on 04 Oct 2022
Rare disease endpoint advancement pilot program
3 October 2022 - FDA is establishing a Rare Disease Endpoint Advancement (RDEA) Pilot Program to support novel efficacy endpoint development ...
Posted by Michael Wonder on 12 Sep 2022
Chiesi Global Rare Diseases announces FDA acceptance of BLA filing for velmanase alfa for the proposed treatment of alfa mannosidosis
12 September 2022 - FDA grants priority review designation with PDUFA date in the first half of 2023. ...