The EMA has once again come in behind the U.S. FDA, granting market access to 77 new products in 2023, fewer than half the 157 approvals the FDA processed in the 11 months from January through December 2023.
The most comprehensive analysis of gene dependencies in cancer cells to date has identified 370 “highly enriched” drug targets in defined molecular backgrounds. This latest iteration of the Cancer Dependency Map, published in Cancer Cell, Jan. 11, 2024, builds on an earlier version published in 2019, which was based on 324 cell lines.
Six weeks after the U.S. FDA issued an alert, the EMA is following suit and starting a review of the safety of six approved CAR T-cell cancer therapies, following 23 reports of patients developing secondary cancers. The EMA said such malignancies were considered “an important potential risk” at the time of approval and are included in the risk management plan. Close monitoring is in place, with companies required to conduct long-term safety and efficacy follow-up studies and to file safety update reports.
A landmark, real-world study in the U.K. has demonstrated that combining whole genome sequencing with clinical data enabled tailored cancer treatment and improved outcomes. At one health care center, having DNA sequence data led to changes from usual standard of care in 25% of cases. “Mostly, [patients] got into clinical trials; some got medicines they wouldn’t have got. Others avoided medicines because their genetic make-up suggested that if they were exposed to the medicines, they would be at risk of harm,” said Mark Caulfield, professor of clinical pharmacology at Queen Mary University of London, who is co-author of a paper outlining the findings in Nature Medicine, Jan 11, 2024.
The U.S. FDA is promising to make 2024 a “breakout” 12 months for gene therapies, with a number of initiatives to promote clinical development, approvals and uptake. “This is a great year to focus on gene therapy,” said Peter Marks, director of the Center for Biologics Evaluation and Research at the FDA. “I just want to focus on moving ahead gene therapy,” he told attendees of the J. P. Morgan Healthcare Conference on Jan. 8.
Gene therapy has finally become the “new normal” with serial breakthroughs unlocking “tremendous value” for patients and society, while at the same time the U.S. health care system is shaping up to enable access to these costly treatments, according to Tim Hunt, CEO of the Alliance for Regenerative Medicine.
Newron Pharmaceuticals SpA has reported what it claims are “exceptional” results in the 12-month analysis of a phase II open-label trial of evenamide in treatment-resistant schizophrenia. The glutamate modulator produced benefits “of a kind that have never been reported before,” the company said.
Genome editing specialist Tome Biosciences Inc. now has all the bases covered, after arriving on the scene in December with $213 million funding and three weeks later announcing the acquisition of fellow precision editing company, Replace Therapeutics Inc. for up to $185 million.
After the initial approvals in monogenic inherited diseases, the scope of gene therapy is widening, with new delivery routes, novel vectors, cell-specific targeting and products aiming to treat chronic disorders, all making headway in 2023.
It is not the first malaria vaccine, but R21, recommended for use by the World Health Organization in October, is the first that can be manufactured at modest cost and the sort of scale needed for widespread prevention of the killer disease in Africa.
