Genentech’s Evrysdi (risdiplam) granted FDA priority review for treatment of presymptomatic babies under 2 months of age with spinal muscular atrophy

24 January 2022 - Interim data submitted to the FDA show majority of pre-symptomatic babies treated with Evrysdi for at least ...

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bluebird provides update on FDA review timelines for betibeglogene autotemcel for beta thalasszemia and elivaldogene autotemcel for cerebral adrenoleukodystrophy

18 January 2022 - FDA PDUFA goal dates for both therapies extended by three months. ...

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4D Molecular Therapeutics announces FDA fast track designation granted to 4D-125 for the treatment of X-linked retinitis pigmentosa

10 January 2022 - 4D Molecular Therapeutics announced that the U.S. FDA has granted fast track designation for 4D-125 for treatment ...

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Public Summary Documents – September 2021 PBAC meeting

24 December 2021 - The Public Summary Documents (positive recommendations and subsequent decisions not to recommend) from the September 2021 PBAC ...

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bluebird bio announces FDA priority review of biologics license application for eli-cel gene therapy for cerebral adrenoleukodystrophy in patients without a matched sibling donor

17 December 2021 - If approved, eli-cel will be the first and only gene therapy for the treatment of cerebral adrenoleukodystrophy, ...

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CSL Behring receives accelerated CHMP assessment for etranacogene dezaparvovec for European patients living with haemophilia B

15 December 2021 - CSL Behring today announced that the CHMP, the chief scientific body of the EMA accepted its ...

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EU regulation passed: G-BA works on European HTA assessment

14 December 2021 -  Today the EU Parliament passed the EU-HTA regulation for a joint health technology assessment, which includes, ...

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ICER to assess gene therapy for beta thalassaemia

24 November 2021 - Report will be subject of New England CEPAC meeting in June 2022; draft scoping document open ...

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bluebird bio Announces FDA priority review of biologics license application for beti-cel gene therapy for patients with β-thalassemia who require regular red blood cell transfusions

22 November 2021 - FDA set PDUFA date of 20 May 2022. ...

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Drugs against rare diseases: Libmeldy gene therapy shows additional benefits - Zolgensma cell therapy, on the other hand, does not

4 November 2021 - The Federal Joint Committee (G-BA) today assessed the additional benefit of two active ingredients against very rare, ...

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AvroBio receives rare paediatric disease designation from the U.S. FDA for AVR-RD-05, a gene therapy for mucopolysaccharidosis type II or Hunter syndrome

3 November 2021 - AVROBIO today announced that the U.S. FDA has granted rare paediatric disease designation to AVR-RD-05, its ...

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FDA, NIH and 15 private organisations join forces to increase effective gene therapies for rare diseases

27 October 2021 - The U.S. FDA, the National Institutes of Health, 10 pharmaceutical companies and five non-profit organisations have partnered ...

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Sio Gene Therapies announces granting of FDA fast track designation for investigational AXO-AAV-GM1 (AAV9-GLB1) gene therapy in patients with GM1 gangliosidosis

21 October 2021 - Sio Gene Therapies today announced that the U.S. FDA has granted fast track designation to AXO-AAV-GM1, its ...

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NICE recommends gene silencing therapy for porphyria patients on NHS

21 October 2021 - NICE have recommended the use of givosiran, a gene silencing therapy, on the NHS in England, as ...

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Novartis applauds Quebec, the first province to list Zolgensma for the treatment of paediatric patients with spinal muscular atrophy

15 October 2021 - Public reimbursement under the Régie de l'assurance maladie du Québec, effective October 20th will apply for children ...

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