Cell & Gene Therapies
Meeting the regulatory gold standard for drug candidates in ultra-rare diseases can be impractical, a regulatory expert says, but greater collaboration and shared insights from regulatory reviews could help find a viable path forward.
Decentralized manufacturing methods for cell and gene therapies will be critical for improving patient access to treatments, but sponsors must prepare to demonstrate “comparability” with centralized manufacturing.
Hemgenix has now secured reimbursement in several European markets, with more talks ongoing. While the path to reimbursement has not always been easy, innovative access deals have helped to smooth the way.
US and EU regulators have invited two companies to work with them on a pilot scheme for assessing drugs for rare genetic diseases, a move that the European Medicines Agency’s Emer Cooke said would help improve “global alignment.”
Long-term follow-up requirements have taken a conservative approach, but could be ripe for re-examination and global harmonization, Kite Pharma executive director says, while former FDA gene/cell therapy office head Wilson Bryan wants the classwide REMS eliminated.
The move is positive for manufacturers and follows the agency’s decision to drop another contentious proposal that would have ‘stacked’ drug discounts when calculating Medicaid best prices.
An FDA question to a patient concerned about the negative impacts of the CAR-T boxed label warning during a recent listening session indicates the agency may be thinking about improving its dissemination of the information.
Japan plans to offer wider support to foreign firms and ventures with innovative candidates to start clinical trials in the country, as part of key measures from current prime minister Fumio Kishida.
The program for collaborative gene therapy reviews with the European Medicines Agency is starting with simple applications, but after launch could be expanded to earlier discussions of CMC and pharm/tox data requirements, the FDA’s Nicole Verdun said.
Regulators in Europe and the US have demonstrated a commitment to providing guidance that will make decentralized and point-of-care manufacturing a reality, the CSO of GermFree, a company that provides mobile and modular cleanrooms for manufacturing advanced therapies, tells the Pink Sheet.
CBER Director Peter Marks said recent leaps in gene therapy science have not been matched by more affordable manufacturing technology and that the field needs to set a cost-effectiveness target.
Finding the right way to measure outcomes is just one of the difficulties involved in conducting clinical trials of potential new therapies for loss of hearing, which can have a number of different causes.
After 10 years of promised investment following its Nobel Prize for iPS cell research, Japan is cautiously narrowing regulations around the conditional approval of cell therapies and cutting reimbursement prices. How has the sector panned out over the last decade?
It is “absolutely fundamental” that manufacturers of cell and gene therapies interact with inspectors from regulatory agencies to understand their expectations around good manufacturing practice, a compliance consultant at CDMO eXmoor pharma says.
The CMS supplemental reimbursement for the gene therapies is higher than usual for new technologies, but is similar to the formula used for antibiotics.
Nine new entries targeting a wide range of unmet medical needs. Several EU marketing approvals and two withdrawals. And an accelerated assessment tool that was seldom used. These were among the activities noted as part of the European Medicines Agency’s priority medicines scheme during the first half of 2024.
Casgevy, the world’s first CRISPR gene editing therapy, is the second drug to be accepted onto a managed access scheme via England’s Innovative Medicines Fund, offering a new treatment for patients with transfusion-dependent beta thalassemia.
Shanghai issues a new set of policies and financial incentives designed to support and speed up regulatory and commercial activities in the biopharma sector, in a comprehensive stimulus package for companies based in the major Chinese city.
High patient expectations also drove Telethon’s decision to bring forward its marketing authorization application plans for its ultra rare disease gene therapy, etuvetidigene autotemcel.
Despite it’s initial promise, a novel autologous cell transplant therapy for severe heart failure has faced multiple challenges gaining full approval in Japan, potentially offering lessons for other regenerative medicine developers.