Vertex Pharmaceuticals’ new treatment for sickle cell disease can eliminate the damaging pain crises people with the blood condition frequently experience, a life-changing benefit that has excited many about the therapy’s promise.
However, drugs used to prepare patients for the treatment, called Casgevy, carry a high risk of infertility. Vertex has said it will help patients insured on the commercial market access fertility preservation services, but federal rules bar it from doing the same for people covered by Medicaid.
On Monday, the company filed a lawsuit to force a change in that policy, asking the U.S. District Court for the District of Columbia to declare such assistance permissable. The suit challenges a determination made by the U.S. Department of Health and Human Services’ inspector general, which found that Vertex providing fertility support would violate laws designed to prevent illegal kickbacks.
“Even though Medicaid and most other insurers already deny Americans with [sickle cell] fertility coverage, the federal government, through [the inspector general]’s refusal to issue a favorable advisory opinion, has effectively prohibited those patients from receiving free fertility services from others — leaving them with the Hobson’s choice between undergoing a potentially curative treatment or becoming biological parents,” the suit states.
Vertex also seeks to compel the inspector general to issue a written opinion as required by law, rather than the oral determinations it has so far communicated. The company describes the agency’s failure to do so as a “delaying tactic” that has left patients’ lives in regulatory limbo.
Approved for sickle cell by the Food and Drug Administration last December, Casgevy is the first CRISPR gene editing medicine cleared for commercial use. It consists of a patient’s own stem cells, which are modified using the gene editing technology to essentially detour around the genetic mutation that causes the disease. In testing, nearly all treated participants were free of sickle cell crises, which can cause organ damage and a variety of other health problems.
But before the engineered stem cells are infused back into the body, patients must undergo treatment with a chemotherapy regimen that creates room in the bone marrow for the new cells to take root.
While this regimen comes with a number of side effects, among the most significant is the chance it causes infertility. This risk is seen as a major barrier to treatment for those who may be considering Casgevy to become healthy enough to start a family.
The FDA in January also approved Casgevy for severe beta thalassemia, another blood condition. Treatment for those people also requires chemotherapy preconditioning. Vertex set the list price of Casgevy for both diseases as $2.2 million.
The company has not disclosed whether anyone has received Casgevy since its U.S. approval, although it has said several have begun the process. For people who are commercially insured, the company will help pay for the treatments necessary to preserve fertility, such as the collection and storage of eggs or sperm, and in vitro fertilization.
It sought to do the same for the many sickle cell patients covered by Medicaid, and last June asked HHS whether doing so would violate anti-kickback or related statutes. The agency’s inspector general in November and again in January informed Vertex that it would, noting that it “poses more than a low risk of fraud and abuse,” according to the lawsuit.
Vertex claims the agency’s interpretation of the law is “overly broad” as well as “arbitrary and capricious.” The company also cites the actions of the Centers of Medicare and Medicaid Services, which is launching a pilot program designed to improve access to sickle cell gene therapy for people on Medicaid. One requirement of the pilot is that manufacturers provide fertility services, albeit in a “defined scope.”
Along with Casgevy, the FDA has also approved another genetic medicine, Lyfgenia from Bluebird Bio, which uses a different gene modification technique to also treat sickle cell. Preconditioning is also required for Lyfgenia treatment.
Correction: A previous version of this story included the wrong list price for Casgevy.
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- Source: https://www.biopharmadive.com/news/vertex-sue-hhs-casgevy-sickle-cell-fertility-treatment-aks/721417/