5 takeaways:
➀ There are now 10 FDA-approved cell-based gene therapies in the United States. Six of them are chimeric antigen receptor T-cells, also known as CAR T-cells, that aid in attacking certain blood cancers, said Dr. Peter Marks, director of the Center for Biologics Evaluation and Research at the Food and Drug Administration. Two are stem cells that are genetically modified and given as part of a stem cell transplant type program. And two are directly administered gene therapies, one put into the eye for a type of hereditary blindness, and the other given systemically for a neuromuscular disorder that kills very young children. In clinical trials, 14 of 15 children with that fatal disease were developing normally after the treatment. “It would be rare to see them alive, and to see them running around is unheard of,” Marks said. Insert [Transcript | Video]
➁ Genomics companies want to make a profit. In biotech drug development circles, there are several strategies to help speed up safe patient access. They include: the Right to Try Act, Emergency Use Authorizations, Priority Review Voucher Programs, Accelerated Approval, the Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations. But the central issue is commercial viability, Marks said. “There are various gene therapies that have come on the market and have gone off the market in Europe because just the sizes of the populations are too small for commercial viability with our current model.”
➂ With new therapies, there are always tensions between speed and safety. There are tensions, Marks said, between providing treatment access to patients without enough safety and efficacy data, or making patients wait too long and suffer needlessly. Researchers must try to recognize all potential benefits, risks and uncertainties. “Our goal here in bringing these gene therapies forward is to make sure that they do meet our standard for efficacy and that they are safe, so that when patients get these, they can be assured that they’re going to bring benefit. Because if that’s not the case, we’re not doing them a favor.”
➃ Standardizing technologies could change the gene therapy game. While 10 approved therapies for rare diseases may not sound like a lot, Marks said there’s a “very robust global pipeline” for new gene-based therapies. Research cooperation and technology transfer could speed up development and even bring down the costs, Marks said. That’s because there’s a “backbone” of gene therapy: the mechanism that delivers a missing protein to where it’s needed. In theory, that mechanism doesn’t have to change for each treatment — like a razor blade handle that can swap out different blades, Marks said. “The idea is that potentially you could move from one gene therapy to another and not have to rework the entire vector up each time, in terms of the manufacturing information, and toxicology information.”
➄ Building global capacity for gene therapy is crucial. While diseases like sickle cell anemia or hemophilia may be treatable in high-income countries, patients in poorer nations often cannot get the care they need. Gene therapy may be the only way for patients in low- and middle-income countries to get effective treatment, Marks said. FDA officials will be meeting with the World Health Organization to discuss improving global access to cell tissue and gene therapies, Marks said. There’s an urgent need to combine data and caseloads to help surmount the perception that there aren’t enough cases in low- and medium-income countries to make access commercially viable.
This program was sponsored by Fondation Ipsen. NPF is solely responsible for the content.
