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 ...

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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 ...

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Vertex receives CHMP positive opinion for the first CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel), for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia

15 December 2023 - If approved by the European Commission, patients 12 years of age and older with severe sickle cell ...

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Intellia Therapeutics receives Priority Medicines (PRIME) designation from the EMA for NTLA-2002, an investigational in vivo CRISPR genome editing treatment for hereditary angioedema

13 October 2023 - Intellia Therapeutics today announced that the EMA has granted Priority Medicine (PRIME) designation to NTLA-2002 for ...

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Rocket Pharmaceuticals receives EMA Priority Medicines (PRIME) designation for RP-A501 gene therapy for Danon disease

31 May 2023 - Rocket Pharmaceuticals today announced that the EMA has granted Priority Medicines (PRIME) designation to RP-A501, the ...

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Gene therapies are still hampered by substantial delays between approval and launch

3 May 2023 - Gene therapies hold promise for treating a wide range of diseases. Innovative companies such as bluebird bio ...

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GenSight Biologics withdraws its EMA application for Lumevoq

20 April 2023 - GenSight Biologics today announces that the Committee for Advanced Therapies (CAT) of the CHMP of the EMA ...

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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, ...

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First gene therapy for haemophilia B, CSL's Hemgenix, approved by the European Commission

20 February 2023 - Hemgenix underscores CSL's promise to deliver life-changing innovations that have the potential to help patients lead ...

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Forge Biologics receives PRIME designation from the EMA for novel gene therapy FBX-101 for the treatment of patients with Krabbe disease

17 January 2023 - FBX-101 is granted priority medicines (PRIME) designation by the EMA after review of Phase 1/2 RESKUE clinical ...

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CSL receives positive CHMP opinion for etranacogene dezaparvovec – gene therapy for adults with haemophilia B

16 December 2022 - Regulatory milestone moves CSL one step closer to bringing the promise of gene therapy for haemophilia ...

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Ionis announces European Medicines Agency accepts marketing authorisation application of tofersen to treat rare, genetic form of ALS

5 December 2022 - EMA acceptance follows FDA's acceptance of tofersen new drug application earlier this year. ...

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Alnylam receives approval in Europe for Amvuttra (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy

20 September 2022 - Amvuttra demonstrated halting or reversal in neuropathy impairment with subcutaneous administration once every three months. ...

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First gene therapy for adults with severe haemophilia A, BioMarin's Roctavian (valoctocogene roxaparvovec), approved by European Commission

24 August 2022 - Maintains orphan drug designation in the EU providing 10 years of market exclusivity. ...

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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. ...

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