Adverum Biotechnologies granted Priority Medicines (PRIME) designation by EMA for ADVM-022 in wet AMD

24 June 2022 - Adverum Biotechnologies today announced that the EMA has granted ADVM-022 Priority Medicines (PRIME) designation for the treatment ...

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First gene therapy to treat severe haemophilia A

24 June 2022 - EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec) ...

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Givlaari is included in the high-cost protection with limitation

17 June 2022 - Givlaari (givosiran) is included in the high-cost protection with limited subsidy to reduce attacks in patients with ...

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Alnylam announces FDA approval of Amvuttra (vutrisiran), an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults

13 June 2022 - First and only FDA approved treatment demonstrating reversal in neuropathy impairment with subcutaneous administration once every three ...

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ICER publishes evidence report on gene therapy for beta thalassaemia

2 June 2022 - Evidence judged to demonstrate that beti-cel provides net health benefit to patients with transfusion-dependent thalassaemia. ...

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FDA accepts CSL Behring's biologics license application for etranacogene dezaparvovec for priority review

24 May 2022 - If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with haemophilia B. ...

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Draft CADTH recommendation for Spinraza (nusinersen sodium) fails adults in need of treatment for spinal muscular atrophy

20 May 2022 - CADTH does not accept new evidence demonstrating Spinraza's effectiveness and safety for treating spinal muscular atrophy – ...

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PTC Therapeutics receives positive CHMP opinion for Upstaza for the treatment of AADC deficiency

20 May 2022 - First ever gene therapy directly administered into the brain. ...

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FDA, EMA officials discuss impediments to cell and gene therapies

17 May 2022 - The US FDA's top biologics regulator said the use of a “playbook” or platform approach ...

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Amicus Therapeutics receives notification of PDUFA date extensions for AT-GAA

10 May 2022 - Amicus Therapeutics today announced that the U.S. FDA has extended the review period by 90 days ...

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ICER to assess gene therapies for haemophilia A and B

5 May 2022 - Report will be subject of CTAF meeting in November 2022; draft scoping document open to public ...

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ICER’s favourable assessment of Bluebird Bio’s gene therapy Zynteglo may have important pricing and reimbursement implications

5 May 2022 - ICER evaluated the clinical and cost-effectiveness - as well as budgetary impact - of one of two ...

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First million dollar medicine listed on the PBS

4 May 2022 - Zolgensma has the great honour of being the first medicine listed on the PBS with a ...

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TLV submits proposals for increased access to new gene therapies

2 May 2022 - How should society handle new gene therapies for serious diseases, where the effect of the drugs is ...

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ICER releases draft evidence report on gene therapy for beta thalassaemia

13 April 2022 - Public comment period now open until 10 May 2022; requests to make oral comment during public ...

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