Would this high price tag make it difficult for public health systems to finance the therapy?...Released by November 22, 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b?
Program runs from April 25 - June 22, 2022. REGISTRATION NOW OPEN Deadline to register: May 11, 2022 LEARN MORE #Training #TrainingandDevelopment #workforce #29.1
Patients with haemophilia B have mutations (changes) in a gene which the body needs to make the clotting protein Factor IX, resulting in either a partial or complete lack of its activity [1] Hemigenix received FDA BLA approval on 22 November 2022 [4, 5]. As of 22 March 2023, Hemgenix has not yet been approved by the TGA, but it was granted orphan designation by the TGA in August 2022 [6]
The models are expected to enable insights into embryo development, problems that may occur during development, and possible new drug targets. [20, 21] ICH reflection paper on integrating real-world evidence into regulatory decision-making The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) has consulted on a reflection paper for public consultation to harmonise real-world evidence terminology and enable the convergence of general principles for planning and reporting studies using real-world data to support regulatory decision-making [22]. It identifies the following areas for harmonisation: convergence on terminology, format for protocols and reports of study results submitted to regulatory agencies throughout the lifecycle of medicines, and registration of protocols and reports
FACT Update May 2022 by: Mikaela VanMoorleghem, MPA, Education and Training Coordinator Foundation for the Accreditation of Cellular Therapy Omaha, NE USA FACT-ISCT Virtual Quality Boot Camp – July 22, 2022 The 7th Annual FACT - ISCT Quality Boot Camp will be held virtually on Friday, July 22 th , from 8 am to 5 pm CT
Updates and regulatory news Health Canada What’s new in Biologics, radiopharmaceuticals and genetic therapies can be found at: https://www.canada.ca/en/health-canada/services/drugs-health-products/biologics-radio-pharmaceuticals-genetic-therapies/what-new-biologics-radiopharmaceuticals-genetic-therapies-health-canada.html Information for health product manufacturers and distributors in relation to COVID-19: https://www.canada.ca/en/health-canada/services/drugs-health-products/covid19-industry.html Health-related consultations: https://www.canada.ca/en/services/health/consultations.html FDA OTP Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls – June 8, 2023 (Recording is now available) https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/otp-town-hall-cell-therapy-chemistry-manufacturing-and-controls-june-2023-06082023 FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy – June 22, 2023 https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy FDA Approves First Cellular Therapy to Treat Patients with Type 1 Diabetes – June 28, 2023 https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-type-1-diabetes FDA Approves First Gene Therapy for Adults with Severe Hemophilia A – June 29, 2023 https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-adults-severe-hemophilia#:~:text=Today%2C%20the%20U.S.%20Food%20and,by%20an%20FDA%2Dapproved%20test
https://www.ema.europa.eu/en/medicines/human/EPAR/casgevy Update on the Guideline on the acceptability of names for human medicinal products processed through the centralised procedure – 15th December 2023 https://www.ema.europa.eu/en/guideline-acceptability-names-human-medicinal-products-processed-through-centralised-procedure-scientific-guideline EMA Management Board: highlights of December 2023 meeting https://www.ema.europa.eu/en/news/ema-management-board-highlights-december-2023-meeting Artificial intelligence workplan to guide use of AI in medicines regulation – 18th December 2023 https://www.ema.europa.eu/en/news/artificial-intelligence-workplan-guide-use-ai-medicines-regulation#:~:text=The%20workplan%20will%20help%20the,benefit%20public%20and%20animal%20health
While we celebrate these landmark approvals, as a field we must recognize the challenges to patient access presented by the price tags of these drugs. The FDA approval of Zynteglo benefits to be put in context with its previous conditional approval in Europe by the EMA for the same patient group in May 2019 and the challenges for patient access
Proceedings from 2021 ISCT Scientific Signature Series Now Available "Advanced in Cell and Gene Therapies for Lung Diseases and Critical Illnesses" PUBLISHED IN CYTOTHERAPY - VOLUME 24, ISSUE 5 , May 22, 2022 Abstract The ISCT Scientific Signature Series Symposium “Advances in Cell and Gene Therapies for Lung Diseases and Critical Illnesses” was held as an independent symposium in conjunction with the biennial meeting, “Stem Cells, Cell Therapies, and Bioengineering in Lung Biology and Diseases,” which took place July 12–15, 2021, at the University of Vermont
Rajiv Khanna (ANZ Regional Vice-President 2020-22) and a President’s welcome by ISCT President 2020-22 Dr