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RSS FeedPosted 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 13 Feb 2024
European Commission approves first CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel), for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
13 February 2024 - Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent ...
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 30 Oct 2023
Switzerland restricts reimbursement of orphan drugs
30 October 2023 - New rules for the reimbursement of orphan drugs and other unlisted medicines will become effective in Switzerland ...
Posted by Michael Wonder on 30 Oct 2023
Egetis announces EMA validation of marketing authorisation application for Emcitate for the treatment of MCT8 deficiency
27 October 2023 - Egetis Therapeutics today announced that its marketing authorisation application to the EMA for Emcitate (tiratricol) for ...
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 24 Oct 2023
Ipsen updates on EU marketing authorisation application for odevixibat in Alagille syndrome
23 October 2023 - Ipsen plans to submit a new marketing authorisation application for the treatment of Alagille syndrome by the ...
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 10 May 2023
Reforming EU and national orphan drug regulations to improve outcomes for patients with rare diseases
9 May 2023 - In Europe, the number of approvals for new drugs targeting rare diseases (orphan drugs) has risen ...
Posted by Michael Wonder on 28 Mar 2023
Amicus Therapeutics announces European Commission approval for Pombiliti in patients with late-onset Pompe disease
27 March 2023 - CHMP opinion for miglustat, the oral enzyme stabiliser component of AT-GAA, on track for 2Q, 2023. ...
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 08 Mar 2023
Genethon given PRIME status by EMA for gene therapy to treat Crigler-Najjar syndrome, a rare liver disease
7 March 2023 - Genethon today announced that the EMA has granted PRIME (Priority Medicines) status to the gene therapy, ...