US government denies Bluebird fertility support for Zynteglo patients

The U.S. government has decided against allowing Bluebird bio to offer fertility support to individuals insured federally while undergoing treatment with their gene therapy, Zynteglo. This decision was outlined in a negative opinion by the Department of Health and Human Services (HHS) inspector general, issued on Monday. Bluebird's proposed support intended to offer up to $22,500 for fertility preservation treatments, but this was flagged as potentially conflicting with anti-kickback regulations.

The opinion from the inspector general is advisory and has not definitively stated that Bluebird's program violates the rules. However, the government will not grant the company prospective immunity from legal repercussions. This decision is a setback for Bluebird's efforts to expand the use of Zynteglo, which is approved in the U.S. for treating beta thalassemia, an inherited blood disorder. Despite Zynteglo’s significant benefits, it requires preparatory chemotherapy, which poses a high risk of infertility. Bluebird intended its support program to help those who might otherwise avoid the treatment due to fertility concerns.

The anti-kickback rules apply to patients with federal insurance like Medicaid, but not to those with commercial insurance. Bluebird expressed dismay, highlighting the inequity between patients with different types of insurance coverage. They emphasized that no patient should have to choose between lifechanging therapy and the ability to have children. Bluebird also sought to extend this program to patients with cerebral adrenoleukodystrophy receiving its gene therapy, Skysona.

The HHS inspector general pointed to a lack of comprehensive data on costs and healthcare access to assess the risk of fraud and abuse associated with Bluebird’s plans. While acknowledging the promise of cell and gene therapies, the inspector general noted that these treatments are still novel, with much unknown about optimal access arrangements.

This unfavorable opinion follows a similar situation involving Vertex Pharmaceuticals, which recently sued the federal government after the inspector general blocked its fertility support plans for patients receiving its gene therapy, Casgevy. Like Zynteglo, Casgevy is approved for sickle cell disease and beta thalassemia and requires chemotherapy preconditioning.

A potential solution for gathering more data is a pilot program by the Centers for Medicare and Medicaid Services, intended to test different payment models for gene therapies for sickle cell disease, including Casgevy. Bluebird’s treatment for sickle cell, Lyfgenia, which was approved alongside Casgevy last December, is also part of this pilot, which mandates limited fertility support from manufacturers.

Both Bluebird and Vertex have criticized this requirement, arguing it contradicts the inspector general’s stance on their fertility programs. Bluebird expressed surprise that the Office of Inspector General (OIG) does not see infertility as a treatment barrier, especially given the HHS’s own focus on fertility preservation in their Cell and Gene Therapy Access Model.

Zynteglo, Casgevy, and Lyfgenia are all derived from a patient’s stem cells, which are genetically modified before being reinfused to produce a specific therapeutic effect. Chemotherapy is used to create space in the bone marrow for these engineered cells to engraft and develop into functional blood cells.