Blog
RSS FeedPosted by Michael Wonder on 03 Mar 2025
Vertex receives CHMP positive opinion for expanded label for Kaftrio in combination with ivacaftor for people with cystic fibrosis to include rare mutations
28 February 2025 - If approved, approximately 4,000 people with cystic fibrosis in the European Union will be eligible for ...
Posted by Michael Wonder on 10 Dec 2024
Chiesi Global Rare Diseases and Protalix BioTherapeutics announce validation of variation submission by EMA for pegunigalsidase alfa
9 December 2024 - Application to label a less frequent dosing regimen at a dose of 2 mg/kg body weight ...
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 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 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 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 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, ...
Posted by Michael Wonder on 24 Feb 2023
Chiesi Global Rare Diseases and Protalix BioTherapeutics receive positive CHMP opinion for pegunigalsidase alfa for treatment of Fabry disease
24 February 2023 - European Commission decision anticipated in beginning of May 2023. ...
Posted by Michael Wonder on 28 Jan 2023
Ipsen receives CHMP negative opinion for palovarotene as a treatment for fibrodysplasia ossificans progressiva in EU
27 January 2023 - Ipsen to request re-examination of CHMP opinion on palovarotene as a potential treatment for fibrodysplasia ossificans progressiva ...
Posted by Michael Wonder on 18 Sep 2022
Agios receives positive CHMP opinion for Pyrukynd (mitapivat) for the treatment of pyruvate kinase deficiency in adult patients
16 September 2022 - Agios expects a decision on the marketing authorisation application by the European Commission within 67 days of ...
Posted by Michael Wonder on 26 Aug 2022
Patient registries: EMA officials highlight opportunities in orphan drug development
25 August 2022 - Officials from the EMA tout the benefits of patient registries to support regulatory decision making for ...
Posted by Michael Wonder on 23 Jul 2022
Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
22 July 2022 - Positive opinion based on HELIOS-A Phase 3 study. ...
Posted by Michael Wonder on 28 Jun 2022
Nexviadyme (avalglucosidase alfa) approved by European Commission as a potential new standard of care for the treatment of Pompe disease
28 June 2022 - Approved for the treatment of the full spectrum of both late-onset Pompe disease and infantile-onset Pompe disease. ...
Posted by Michael Wonder on 21 May 2022
CHMP recommends approval of Xenpozyme (olipudase alfa), the first and only treatment for ASMD
20 May 2022 - Recommendation based on positive results from two clinical trials in which Xenpozyme provided improvement across multiple non-CNS ...