Vertex Sues U.S. Over Fertility Service Limits for Casgevy Patients

Vertex Pharmaceuticals has introduced a groundbreaking treatment for sickle cell disease, known as Casgevy, which promises to eliminate the painful crises experienced by individuals with this blood disorder. This new therapy has sparked significant excitement due to its potential life-changing benefits. However, the drugs used in preparing patients for Casgevy present a high risk of infertility. While Vertex has committed to helping patients with commercial insurance access fertility preservation services, federal regulations prevent similar support for those covered by Medicaid.

In response, Vertex filed a lawsuit on Monday, aiming to change this policy. The company is asking the U.S. District Court for the District of Columbia to allow such assistance, challenging a decision by the U.S. Department of Health and Human Services (HHS) inspector general. The inspector general had previously determined that Vertex providing fertility support would violate laws meant to prevent illegal kickbacks.

Vertex's lawsuit argues that even though Medicaid and many other insurers already deny fertility coverage for Americans with sickle cell disease, the federal government's refusal to grant a favorable advisory opinion effectively prohibits these patients from receiving free fertility services. This, according to Vertex, forces patients to choose between a potentially curative treatment and the ability to become biological parents.

The company is also seeking to compel the inspector general to issue a written opinion, as required by law, rather than the oral determinations it has so far provided. Vertex claims that the agency's failure to provide a written opinion is a delaying tactic that leaves patients' lives in regulatory uncertainty.

Casgevy was approved by the Food and Drug Administration (FDA) last December and is the first CRISPR gene-editing medicine cleared for commercial use. The treatment involves modifying a patient's own stem cells with gene-editing technology to bypass the genetic mutation causing sickle cell disease. Clinical trials showed that almost all participants treated with Casgevy were free from sickle cell crises, which can lead to organ damage and other health issues.

However, before the modified stem cells are infused back into the patient, a chemotherapy regimen is required to create space in the bone marrow for the new cells. This regimen has several side effects, with infertility being one of the most significant. This infertility risk poses a major barrier for patients considering Casgevy, especially those who wish to start a family.

In January, the FDA also approved Casgevy for severe beta thalassemia, another blood disorder that requires similar chemotherapy preconditioning. Vertex set the price of Casgevy at $2.2 million for both conditions. While the company has not disclosed whether any patients have received Casgevy since its approval, it has indicated that several patients have begun the treatment process.

For patients with commercial insurance, Vertex will assist in covering the costs of fertility preservation treatments, such as egg or sperm collection and storage, as well as in vitro fertilization. The company sought to extend this support to Medicaid-covered sickle cell patients and asked HHS in June whether such assistance would violate anti-kickback statutes. The inspector general responded in November and January, stating that it would indeed pose a high risk of fraud and abuse.

Vertex argues that the agency's interpretation of the law is excessively broad and arbitrary. The company also points to the Centers for Medicare and Medicaid Services' pilot program aimed at improving access to sickle cell gene therapy for Medicaid patients. This program requires manufacturers to provide fertility services, though within a defined scope.

In addition to Casgevy, the FDA has approved another genetic medicine, Lyfgenia from Bluebird Bio, which treats sickle cell disease using a different gene modification technique. Lyfgenia also requires a preconditioning regimen similar to Casgevy.