Cell & Gene Therapies

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.

Himal Thakur said the required long-term safety study can replicate a clinical trial, which is not the intent.

A Catalyst fix, a pediatric rare disease voucher reauthorization that should prevent future workload imbalances for FDA, and a boost for out-of-state coverage in Medicaid are among Congress’s year end commitments to pediatric and rare disease patients that now seem in doubt.

Clene’s ALS drug CNM-Au8 and UniQure’s gene therapy for Huntington’s disease emerged from US FDA discussions with accelerated approval plans using real-world evidence from compassionate use and natural history data sources.

The incoming EU Health Technology Assessment Regulation will see historical data move “more center stage” for advanced therapies, because directly comparing highly individualized therapies is often unfeasible, an advanced therapies expert says.

The US FDA seeks to better understand and predict insertional mutagenesis in gene therapies like bluebird bio's Skysona, which use integrating viral vectors, but a workshop on integration site analysis highlighted the approach's complexity and limitations.

The European Commission’s proposed pharma legislation overhaul could expand the definition of a gene therapy medicinal product, posing challenges both from a regulatory and public perception perspective.

The company said its financial viability is in jeopardy, along with patient access to Lyfgenia, if US FDA Commissioner Robert Califf does not reverse the denial of a rare pediatric disease priority review voucher.

New US FDA draft guidance attempts to address sponsor confusion about the different types of regulatory meetings under PDUFA, as well as frequently asked questions on nonclinical testing, CMC, pharm/tox and clinical studies.