Blog
RSS FeedPosted by Michael Wonder on 23 Jul 2022
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. ...
Posted by Michael Wonder on 21 Jul 2022
Ultra-expensive SMA drug Zolgensma to get insurance benefits from August
21 July 2022 - Novartis' Zolgensma (onasemnogene abeparvovec), the world's most expensive drug yet to treat spinal muscular atrophy, will ...
Posted by Michael Wonder on 19 Jul 2022
ICER publishes final evidence report and policy recommendations on beti-cel gene therapy for beta thalassaemia
19 July 2022 - Independent appraisal committee unanimously determined that evidence is adequate to demonstrate that beti-cel provides a net ...
Posted by Michael Wonder on 17 Jul 2022
China CDE grants breakthrough therapy designation to Neurophth's NR082 in Leber's hereditary optic neuropathy
15 July 2022 - Neurophth Therapeutics today announced that the Center for Drug Evaluation of China National Medical Products Administration has ...
Posted by Michael Wonder on 04 Jul 2022
Report on 'Cell, Gene and Tissue Regulatory Framework in Australia: Stakeholder Perspectives' - TGA response
4 July 2022 - n November 2021 the TGA commissioned MTP Connect to conduct a stakeholder review of the regulatory ...
Posted by Michael Wonder on 28 Jun 2022
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 ...
Posted by Michael Wonder on 26 Jun 2022
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) ...
Posted by Michael Wonder on 20 Jun 2022
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 ...
Posted by Michael Wonder on 14 Jun 2022
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 ...
Posted by Michael Wonder on 02 Jun 2022
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. ...
Posted by Michael Wonder on 24 May 2022
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. ...
Posted by Michael Wonder on 23 May 2022
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 – ...
Posted by Michael Wonder on 21 May 2022
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. ...
Posted by Michael Wonder on 18 May 2022
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 ...
Posted by Michael Wonder on 11 May 2022
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 ...