Cell & Gene Therapies
US FDA Commissioner nominee Martin Makary’s 6 March Senate confirmation hearing should offer insight on his alignment with HHS Secretary Robert F. Kennedy Jr.'s views and willingness to insulate the FDA from an unusual level of micromanagement by the department and White House.
The FY 2024 fourth quarter had the highest number of formal meetings cancelled because written responses were sufficient since the beginning of FY 2018.
The European Medicines Agency has recommended in favor of approving four new products, and has also reaffirmed its positive opinion for the Alzheimer’s disease drug, Leqembi.
The US FDA is expected to refine warnings recently added to CAR-T cell therapies about the risk of secondary malignancies after reassuring new data, which is a positive sign for the future of the therapies in autoimmune disorders.
Health technology assessment agencies are willing to pay more than usual for advanced therapies that treat rare or severe diseases, but an advanced therapy for a common condition would not qualify for such a modifier, a former senior figure at England’s NICE says.
The European Medicines Agency is set to issue opinions this week on whether pan-EU marketing approval should be granted to a number of new products.
While CSL Behring has successfully negotiated reimbursement contracts for its one-time gene therapy Hemgenix in several European countries, it says there are still “barriers to innovative contracting solutions” in some member states that need to be addressed.
CSL Behring explains how it worked with authorities in Denmark, Austria, England, Scotland, Spain and Switzerland to secure innovative access arrangements suitable to each nation’s “unique needs” for its one-time gene therapy, Hemgenix.
The "interwoven" nature of the offices within the agency likely means Medicare and Medicaid benefits may be disrupted by staff cuts despite assurances from the administration, Brooks-LaSure warned.
The US FDA is receiving more requests for regenerative medicine advanced therapy designation and granting more of them, according to recently reported agency data.
Brazil’s drug regulator ANVISA has also published its first post-marketing authorization monitoring reports on advanced therapies Yescarta, Zolgenma, Kymriah, Carvykti and Luxturna.
The world’s first CRISPR gene editing therapy, Casgevy, has been made available to patients with sickle cell disease in England, adding to access arrangements in the US, Austria, Bahrain, Germany, Luxembourg, Italy and Saudi Arabia.
While Japan looks set to further improve policies to support ventures and ease market entry this year, these will contrast with ongoing reimbursement price controls.
The cell and gene therapy field has reached an inflection point in the US as the second Trump Administration begins and advocates argue the sector could fit well with the “MAHA” agenda.
Regenerative medicines using expedited review pathways dominate novel approvals at the Center for Biologics Evaluation and Research, while the Center for Drug Evaluation’s higher volume comes with lower first-cycle approval rates and more standard reviews.
Marketing authorization applications for five new advanced therapy medicinal products were filed with the European Medicines Agency in 2024, the highest number in any year since 2020. The Pink Sheet looks at these and two other ATMPs that might get the thumbs up from the European Medicines Agency in 2025.
Amid complaints about the challenges of following gene therapy patients for up to 15 years, OTP Director Nicole Verdun said the FDA is considering how to conduct long-term postmarket studies more efficiently.
China has granted its first approval for a stem cell therapy, to domestic firm Platinum Life Biotech’s umbilical cord-derived mesenchymal stem cell product for corticosteroid-failed acute graft-versus-host disease with gut involvement.
If successful, the new CMS cell and gene therapy access model eventually could expand to oncology or hemophilia products, an Avalere Health expert said.
“Significant progress” has been made in setting up the UK’s Rare Therapies Launch Pad pilot scheme, which will construct a “new regulatory pathway tailored specifically for ultra-rare diseases,” a senior scientist involved in the project says.