An unnamed source clarified that Sandra Retzky has not been fired from the FDA, but it remains unclear where she was reassigned.
Sandra Retzky, formerly the top orphan drug regulator at the FDA, is no longer serving as director of the Office of Orphan Products Development.
The departure, first reported by Endpoints News on Wednesday citing an unnamed source, continues the high-level staffing shuffle at the Department of Health and Human Services since Secretary Robert F. Kennedy Jr. took over the agency in February. Endpoints’ source clarified that Retzky had not been fired, though it remains unclear where she was transferred.
Retzky first took the reins of the FDA’s orphan drug department in August 2021, according to her LinkedIn profile, overseeing the unit’s work of promoting and supporting the development of medical products—such as drugs, biologics and devices—for rare diseases. The department also engages with the broader rare disease community, including patient groups, academia and professional organizations.
Part of the department’s work is to grant orphan drug status to products that are being developed for diseases affecting fewer than 200,000 patients in the U.S. Additionally, the office helps drug companies determine if their products qualify for certain awards, such as the FDA’s orphan drug and rare pediatric disease designations.
Retzky would have been witness to a number of major policy moves in recent weeks. Most recently, the Centers for Medicare and Medicaid Services finalized Inflation Reduction Act guidelines earlier this month, cementing broader exemptions for orphan drugs. Under the initial IRA rules, orphan drugs are exempted from pricing negotiations as long as they remain approved for only one indication.
CMS’ new rules, meanwhile, stipulate that the countdown for qualifying for the negotiation program will start only after a drug is approved for a non-orphan indication.
A few weeks earlier, the House Committee on Energy and Commerce cleared a bill that could restart the program that hands out priority review vouchers to drugmakers that secure approvals for a rare pediatric disease drug. The program, first enacted in 2012, was meant to incentivize companies to invest in rare childhood diseases, but after Congress failed to renew the program in September 2024, the agency was forced to wind it down.
Retzky’s departure also follows a series of leadership exits at the FDA. In June, Nicole Verdun was ousted from her role as the director of the agency’s Office of Therapeutic Products, which oversees cell and gene therapies. Her former boss, Peter Marks, was also forced out of the director post at the Center for Biologics Evaluation and Research in March.
This staffing shakeup extends past the FDA, too. In August, former CDC Director Susan Monarez was fired after just 28 days on the job, a move she alleges was driven by her refusal to agree to Kennedy’s request to pre-approve certain recommendations by the agency’s vaccine advisers. Similarly, Jeanne Marrazzo, former head of the National Institutes of Allergy and Infectious Diseases, was removed from her post in what she claims is retaliation for a whistleblower report that she filed, decrying the HHS leadership’s “hostility towards vaccines.”