Alexis Kuhn, PharmD, BCOP, on Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
The pediatric oncology pharmacist at Mayo Clinic discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
“These therapies are obviously very new, very exciting, and very complicated. As pharmacists, there are a lot of areas that we do need to pay attention to and be aware of the nuances [of] not only for the individual agents, but for all of the concomitant therapies that our patients may be on prior to receiving these therapies.”
Over just the past few months, the clinical community has seen multiple FDA approvals of gene therapy products for hemoglobinopathies. In December 2023, Vertex Pharmaceuticals' and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel, marketed as Casgevy) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia) were both approved for sickle cell disease indications.1 Just over a month later, in January 2024, the FDA approved exa-cel for an additional indication in patients with transfusion-dependent beta thalassemia (TDT).2 These new products, the first gene therapies to be approved for hemoglobinopathies, are rapidly changing the treatment landscape for patients with SCD and TDT. As such, it is critically important for doctors and clinical pharmacists to stay up to date with the latest information regarding these therapies in order to prescribe them safely.
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REFERENCES
1. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 7, 2023. Accessed February 23, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
2. Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia. News release. January 16, 2024. Accessed February 23, 2024. https://news.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-casgevytm-exagamglogene
3. Kuhn A. Geneie in a bottle: Gene therapy for hemoglobinopathies. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas.
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