Novel Gene Therapies for Blood Disorders (CRS Report for Congress)
| Release Date |
Oct. 25, 2024 |
| Report Number |
IF12796 |
| Report Type |
In Focus |
| Authors |
Pravin R. Kaldhone |
| Source Agency |
Congressional Research Service |
Summary:
During the past few years, the U.S. Food and Drug
Administration (FDA) has approved gene therapy products
designed to treat blood disorders. Gene therapy works by
modifying a person’s genes to treat or prevent diseases.
While the potential of gene therapy products to treat certain
diseases has been established, their long-term risks are
unclear. Gene therapy products are considered biologics
(biological products) by FDA and regulated by FDA’s
Center for Biologics Evaluation and Research (CBER).
CBER has approved three novel gene therapy products
since 2023 that treat blood disorders. Casgevy is used to
treat sickle cell disease (SCD) and thalassemia, Lyfgenia is
used to treat SCD, and Roctavian is used to treat
hemophilia A. Casgevy and Lyfgenia are the first cell-based
gene therapies for SCD. Casgevy is the first treatment to
use a novel genome editing technology approved by the
FDA. Roctavian is the first gene therapy for adults with
severe hemophilia A.
These three gene therapy products were approved by FDA
utilizing authority in Section 351(a) of the Public Health
Service (PHS) Act. FDA has issued several guidance
documents pertaining to gene therapy products. This In
Focus provides a brief overview of the select blood
disorders, the opportunity gene therapy products provide to
improve care for individuals with these particular blood
disorders, and the approvals required by FDA.
