Uptake of Sickle Cell Gene Therapy Is Lagging Behind Expectations | AMCP Nexus 2024
Only a small group of patients have started the process to receive the new gene therapies for sickle cell disease, says Kevin Niehoff, Pharm.D., of IPD Analytics.
When the FDA announced that it had approved Vertex’s Casgevy (exagamglogene autotemcel) and Bluebird Bio’s Lyfgenia (lovotibeglogene autotemcel) on Dec. 8, 2023, expectations soared for the first two FDA-approved gene therapies for sickle cell disease.
Other FDA-approved gene therapies are for extremely rare diseases. Sickle cell disease is common in comparison. In the press release announcing the approval, the FDA described sickle cell disease as a group of inherited blood disorders affecting approximately 100,000 people.
But the Casgevy and Lyfgenia have gotten off to slow start after their approvals and entry into the real world of care for people with sickle cell disease, according to Kevin Niehoff, an associate director at IPD Analytics who specializes in gene therapies. Niehoff was a panelist today at a session on the sickle cell disease gene therapy at the 2024 Academy of Managed Care Pharmacy (AMCP) Nexus meeting in Las Vegas. He spoke with Managed Healthcare Executive (MHE) in a video interview prior to the meeting,
Niehoff told MHE that Vertex has reported that 20 patients had initiated Casgevy treatment as of mid-2024 and Bluebird has reported just four. The companies report initiated treatments, partly because the treatment necessary to get ready for the infusion of the gene therapy is long.
Niehoff noted the gene therapies for hemophilia had a similarly “low and slow uptake.”
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