Blog
RSS FeedPosted by Michael Wonder on 22 May 2026
Otarmeni (lunsotogene parvec) receives EMA filing acceptance for genetic hearing loss
22 May 2026 - Regeneron Pharmaceuticals today announced the EMA has accepted for review under accelerated assessment the marketing authorisation application ...
Posted by Michael Wonder on 01 May 2026
Ray Therapeutics granted Priority Medicines (PRIME) designation from the EMA for RTx-015 in retinitis pigmentosa
29 April 2026 - PRIME designation follows recent FDA regenerative medicine advanced therapy designation, continues regulatory momentum for RTx-015. ...
Posted by Michael Wonder on 17 Jan 2026
Fondazione Telethon receives european marketing authorisation for Waskyra (etuvetidigene autotemcel), a gene therapy for the treatment of Wiskott-Aldrich syndrome
16 January 2026 - The European Commission’s decision follows the positive opinion issued by the EMA’s CHMP in November 2025. ...
Posted by Michael Wonder on 18 Nov 2025
High dose regimen of nusinersen receives positive CHMP opinion for the treatment of spinal muscular atrophy
17 November 2025 - Positive CHMP opinion is based on data from the DEVOTE study which evaluated the high dose regimen ...
Posted by Michael Wonder on 15 Nov 2025
CHMP positive opinion for Waskyra, a gene therapy for the treatment of Wiskott-Aldrich syndrome
14 November 2025 - Fondazione Telethon announces the positive opinion issued by the CHMP of the EMA, recommending marketing authorisation in ...
Posted by Michael Wonder on 26 Jul 2025
Roche provides regulatory update on Elevidys gene therapy for Duchenne muscular dystrophy in the EU
25 July 2025 - Roche will continue its dialogue with the EMA to explore a potential path forward to make Elevidys ...
Posted 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 ...