Blog
RSS FeedPosted by Michael Wonder on 10 Mar 2025
Wainzua (eplontersen) approved in the EU for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy
10 March 2025 - Second major approval for Wainzua, which is marketed in the US as Wainua. ...
Posted by Michael Wonder on 02 Mar 2025
Krystal Biotech receives positive CHMP opinion for Vyjuvek for the treatment of dystrophic epidermolysis bullosa
28 February 2025 - EC approval decision anticipated in second quarter of 2025. ...
Posted by Michael Wonder on 04 Feb 2025
Fondazione Telethon submits EU marketing authorisation application for etuvetidigene autotemcel gene therapy for the treatment of Wiskott-Aldrich syndrome
3 February 2025 - Fondazione Telethon announced that it has submitted the marketing authorisation application for the gene therapy - etuvetidigene ...
Posted by Michael Wonder on 04 Feb 2025
Comparison of clinical evidence submitted to the FDA and EMA for cell and gene therapies
3 February 2025 - Harmonisation in regulatory submissions across agencies may support timelier access to innovative treatments, including cell and gene ...
Posted by Michael Wonder on 23 Jan 2025
FDA and EMA accept applications for higher dose regimen of nusinersen in spinal muscular atrophy
23 January 2025 - Applications are based on data from the DEVOTE study, which demonstrate the potential for the investigational higher ...
Posted by Michael Wonder on 11 Dec 2024
Krystal Biotech provides update on EMA’s on-going regulatory review of B-VEC for dystrophic epidermolysis bullosa
9 December 2024 - CHMP opinion now expected in Q1, 2025. ...
Posted by Michael Wonder on 21 Oct 2024
Wainzua (eplontersen) recommended for approval in the EU by CHMP for the treatment of adult patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis
21 October 2024 - Recommendation based on NEURO-TTRansform Phase 3 results showing Wainzua demonstrated consistent and sustained benefit improving neuropathy ...
Posted by Michael Wonder on 17 Oct 2024
Alnylam submits regulatory application to the EMA for vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy
16 October 2024 - Type II variation submission based on the positive HELIOS-B Phase 3 trial in which vutrisiran significantly reduced ...
Posted by Michael Wonder on 26 Jul 2024
European Commission approves Pfizer’s Durveqtix (fidanacogene elaparvovec), a one-time gene therapy for adults with haemophilia B
25 July 2024 - A one-time dose of Durveqtix has reduced bleeds post-treatment compared to standard of care with a median ...
Posted by Michael Wonder on 03 Jun 2024
Biogen receives European Commission approval for Qalsody (tofersen), the first therapy to treat a rare, genetic form of ALS
30 May 2024 - Qalsody is Biogen’s third rare disease therapy to be approved in the EU, demonstrating the company’s commitment ...
Posted by Michael Wonder on 01 Jun 2024
New gene therapy treatment for haemophilia B
31 May 2024 - The EMA has recommended granting a conditional marketing authorisation in the European Union for Durveqtix (fidanacogene ...
Posted by Michael Wonder on 31 May 2024
Highlights from the 27-30 May 2024 CHMP meeting
31 May 2024 - The EMA’s CHMP recommended 14 medicines for approval at its May 2024 meeting. ...
Posted by Michael Wonder on 04 Apr 2024
Rocket Pharmaceuticals announces EMA acceptance of RP-L102 marketing authorisation application for the treatment of Fanconi anaemia
2 April 2024 - Positive, previously disclosed results from the global Phase 1/2 trial demonstrated genetic and phenotypic correction combined ...
Posted by Michael Wonder on 24 Feb 2024
New treatment for rare motor neurone disease recommended for approval
23 February 2024 - The EMA has recommended granting a marketing authorisation in the European Union for a new therapy ...
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 ...