The Alliance for Regenerative Medicine predicts up to 17 cell and gene therapy approvals this year and defends high prices, while FDA’s Peter Marks expresses concern about manufacturing costs.
Pictured: Gene therapy concept: iStock, Ilya Lukichev
2023 was a breakthrough year for cell and gene therapies, with seven FDA approvals in the U.S. and one in the European Union, according to the Alliance for Regenerative Medicine. Looking to 2024, the Washington, D.C.–based advocacy organization predicted on Monday in its annual Cell & Gene State of the Industry Briefing that the sector could see up to 17 approvals in the U.S. and EU.
Regulators in the U.S. gave the greenlight in 2023 to five gene therapies for rare genetic diseases and in 2024 could see an additional five approved by the FDA, as well as the first-ever approval of adoptive cell therapy for solid tumors and the first U.S. approval of an allogeneic T-cell therapy.
Other notable milestones expected in 2024 include additional therapies to treat hemophilia A and hemophilia B and dystrophic epidermolysis bullosa.
“We’re optimistic that we’ll see a lot of these pulled through to the finish line,” Tim Hunt, CEO of the Alliance for Regenerative Medicine (ARM), said at the Biotech Showcase 2024 investor conference in San Francisco, co-located with the 42nd Annual J.P. Morgan Healthcare Conference.
Hunt called last month’s respective FDA approvals of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia, competing gene therapies for sickle cell disease, the “seminal moment in the history of biotechnology.”
Casgevy got the regulatory go-ahead for the first CRISPR-based gene editing therapy. However, it is an approval unlikely to be followed up by another such drug anytime soon, according to GlobalData.
“Given the relative immaturity of the CRISPR drugs pipeline, which features very few late-stage products alongside a low likelihood of approvals, it is unlikely to see another drug approval in the near future,” Jasper Morley, drugs intelligence analyst at GlobalData, said in a statement.
Speaking at Monday’s ARM event, Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said that gene therapy is currently at a “critical juncture” due to a combination of factors, including manufacturing challenges, issues with clinical development timelines and different global regulatory requirements.
Marks contends that current manufacturing platforms limit gene therapy production. “The problem is the setup cost for these small gene therapies is just way too high in proportion to the return on investment—so we have to figure out a way to get over that,” he said.
The Cost of Gene Therapies
The National Bureau of Economic Research estimates that more than 1 million patients will receive treatment with gene therapies, costing more than $25 billion annually by the end of 2034, raising concerns about how the U.S. healthcare system could support these growing costs.
The prices for the two latest FDA-approved gene therapies drive the point home. Casgevy has a U.S. list price of $2.2 million per patient, while the wholesale acquisition cost of Lyfgenia in the U.S. is $3.1 million.
Despite their multimillion-dollar price tags, gene therapies have the potential to save the U.S. healthcare system money, according to ARM’s Hunt.
“This can be a little controversial. I know there’s sticker shock when you price the product at $2 million to $3 million. But if you look at the data, take the emotion and set it aside, it tells a very compelling story,” Hunt said. “We believe that these gene therapies are clearly affordable.”
According to Hunt, the lifetime cost of the standard of care for most rare diseases is more expensive than those of approved gene therapies. He noted that the lifetime cost for patients with severe Hemophilia A is over $21 million, those with severe sickle cell disease pay between $4 million to $6 million and patients with transfusion-dependent thalassemia $5.4 million.
Hunt pointed to an Institute for Clinical and Economic Review (ICER) report which found that gene therapies—including BioMarin’s Roctavian (Valoctocogene roxaparvovec) and CSL Behring’s Hemgenix (etranacogene dezaparvovec)—for hemophilia A and B showed the potential for substantial cost offsets due to elimination of the need for expensive prophylactic treatment.
“In the case of these two gene therapies, more than 99% of the traditional cost-effectiveness ‘value’ is generated by cost offsets of eliminating prophylactic treatment that itself is widely considered to be far too expensive to be cost-effective,” according to ICER.
Hunt asserts that ICER “confirmed the high-cost offsets from durable gene therapies in hemophilia, sickle cell and other rare diseases.” He acknowledged that the ICER report is “one data point, but a lot of commercial payers are there as well,” adding that “these products are ultimately digestible by healthcare systems even before taking into account cost offsets.”
Greg Slabodkin is the News Editor at BioSpace. You can reach him at greg.slabodkin@biospace.com. Follow him on LinkedIn.
