By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1
During the Biotech Showcase at JPM 2024, ARM’s CEO, Tim Hunt, delivered ARM 2024 State of the Industry Briefing, titled, Achieving a New Normal. Hunt began his presentation by stating that while we all know capital markets are not where we want them to be, he would focus his discussion on the potential 2024 could have on the cell and gene therapy sector. He shared that breakthroughs are becoming the new norm and that ARM’s “5x5x5” designation (five gene therapies for five rare diseases were FDA approved within five years from 2017 to 2023) came true. In 2024, there are 11 CGTs up for FDA approval. If even five of those are approved, 10 therapies will have been approved in two years.
“The data tells a compelling story, and these products are digestible by healthcare systems,” Hunt explained. “Look at the data. By 2030, gene therapy revenue could reach $7.5 billion.” For context, Hunt shared that’s about 1/10 of one percent in total healthcare spending in the U.S. and one-third of prescription drug spending in 2030. This is comparable to what Medicare paid for one diabetes drug in 2022. “We spend $1.6 million for a heart transplant, of which there are 3,500 per year, and $1 million for an allogeneic bone marrow transplant, for which 9,950 were performed in 2020,” Hunt said.
At the conclusion of Tim Hunt’s presentation, CBER’s Dr. Peter Marks took the stage to provide an update on CAR T and rare disease cell therapeutics and how the FDA intends to address existing challenges. He shared that as of the end of 2023, 22 T-cell malignancy cases have been reported and that sequencing is not available for all of them. He stated that the onset was soon enough after administration that it seems like there’s a causal relationship. Dr. Marks was referring to the statement the FDA made back in November that it was investigating serious risks of T-cell malignancies in patients following treatment with CAR T-cell therapies. Since 2017, six CAR T-cell therapies have been approved by the FDA and all are for the treatment of blood cancers, including lymphomas and some forms of leukemia. Said cancer therapies are made by companies such as Gilead Sciences, Johnson & Johnson, and Novartis. The FDA made its announcement following reports of T-cell malignancies in patients who received B-cell maturation agent (BCMA)- or CD19-directed autologous CAR T-cell immunotherapies, the FDA is investigating the risks. The risk of developing secondary malignancies is a class warning for these therapies, as it is for all gene therapy agents with lentiviral or retroviral vectors.
“That said, there were 27,000 individuals in the U.S. who were treated, and so the Risk / Benefit profile is still incredibly beneficial,” Dr. Marks stated. He explained that the FDA has asked providers to contact them if new T-cell malignancies arise and that as the Agency learns more, it will adapt and update its information.
Dr. Marks concluded his presentation stating that the real complexity is in the cost to manufacture. “We have not followed the monoclonal antibody model; here you have a once-and-done therapy. And so, new models are needed for manufacturing. Patients cannot go to the local health clinic to stop in for a little gene therapy. This is a new business model. How we, a as a society, pay for this is a challenge,” stated Dr. Marks.
Dr. Marks’ presentation at JPM 2024 may have concluded, but on January 25th at 12:45 p.m. EST, he and Dr. Nicole Verdun, Super Office Director, CBER, FDA will join me to discuss the evolving regulatory landscape in 2024 for cell and gene therapies. During our presentation, we will cover improved communication with sponsors, increased speed availability of cell and gene therapies by enabling manufacturing processes and standards development, and much more. Don’t miss it. As always, registration is free.