Cell & Gene Therapies
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.
CBER Director Peter Marks said recent leaps in gene therapy science have not been matched by more affordable manufacturing technology and that there is a need to set a cost-effectiveness target for the field.
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.
Many platform designation requests have been from sponsors eager to cite other sponsors’ products, but CBER Director Peter Marks said in an interview with the Pink Sheet his office likely is years away from accepting those applications.
Vertex’s fertility preservation assistance program would provide up to $70,000 in financial support for in vitro fertilization and other services to patients prescribed Casgevy.
The head of CBER's Office of Therapeutic Products also touted hiring achievements and new communication plans with sponsors at an Alliance for Regenerative Medicine meeting.
Strong concerns about the imminent joint clinical assessments remain, but there are signs that communications between the health technology assessment coordination group and stakeholders could be improving.