Through our Venable FDA Pulse series, we report on critical developments within the Food and Drug Administration (FDA or Agency) that may affect the Agency's structure and stakeholder interactions. This week's updates are as follows:
- FDA Announces Autism-Related Initiatives
- FDA Releases New Draft Guidance on Malaria Treatments
- CBER Director Prasad Resumes Additional Roles
- Pediatric Priority Review Voucher Legislation Moves through Congress
- FDA Rescinds the LDT Rule
FDA Announces Autism-Related Initiatives: Acetaminophen Label Change and Leucovorin Approval
In the midst of a busy week, the largest developments center on the administration’s implementation of its priority goals of treating and preventing autism. In a major shift, on September 22, FDA announced that it has initiated the label change process for acetaminophen products “to reflect evidence suggesting that the use of acetaminophen by pregnant women may be associated with an increased risk of neurological conditions such as autism and ADHD in children.” The press release highlights that recent evidence, including multiple large-scale cohort studies, “has suggested a correlation between acetaminophen use during pregnancy” and autism and ADHD diagnoses. Even with this label change initiative, FDA notes that “a causal relationship has not been established and there are contrary studies in the scientific literature.”
On the same day, FDA also announced that it began the approval process for a previously withdrawn new drug application (NDA) for leucovorin calcium tablets, intended to treat patients with cerebral folate deficiency (CFD). The press release states that in making its decision, FDA reviewed literature published between 2009 and 2024, which supported “a finding that leucovorin calcium can help individuals suffering from CFD.” FDA notes that individuals with CFD have been observed to have developmental delays, with autistic symptoms, seizures, and movement and coordination difficulties. Notably, during a press conference, FDA Commissioner Marty Makary remarked that FDA filed a Federal Register notice changing the label of prescription leucovorin so it could “be available to children with autism.”
At the same press conference, President Trump also made remarks that suggested his administration would make changes to the recommended vaccine schedule and how many vaccines can be administered in a doctor’s visit.
Much has already been written about these actions from various public health and policy perspectives. Focusing on the regulatory implications, these changes demonstrate the administration’s willingness to revisit previous marketing decisions to address perceived public health threats, even when the scientific consensus is not entirely clear. Potential changes to childhood vaccination schedules will have ramifications for product developers, particularly in light of recent actions to limit COVID-19 vaccines to high-risk groups, combined with heightened requirements for placebo-controlled trials and robust post-marketing studies to demonstrate safety and effectiveness.
We will continue to monitor developments related to the acetaminophen label change process, leucovorin approval, and any proposals regarding vaccine administration.
FDA Releases New Malaria Draft Guidance
On September 23, FDA released a draft guidance document, Malaria: Developing Drugs for Treatment. As the title suggests, the guidance is intended to assist sponsors in developing drugs or biologics to treat malaria by outlining several considerations and recommendations. These recommendations address various topics, including early-phase drug development and assessments of efficacy and safety, trial design and population, as well as labeling. FDA is accepting public comments on the guidance until December 22, 2025.
CBER Director Prasad Resumes Additional Roles
Earlier this month, FDA updated the leadership profile of Dr. Vinay Prasad, M.D., M.P.H., to reflect that he has returned to the position of chief medical and scientific officer, in addition to his post as director of the Center for Biologics Evaluation and Research (CBER). As we previously discussed, Dr. Prasad resigned from the Agency at the end of July amid controversy regarding his decision-making and political criticism; however, he returned to FDA shortly thereafter in August. Until recently, it was confirmed only that he had come back to his position as CBER director, and it remained unclear whether he had also reassumed the chief medical and scientific officer role.
Pediatric Priority Review Voucher Legislation Moves through Congress
Last week the U.S. House of Representatives Committee on Energy and Commerce voted to advance to the full House H.R. 1262, also known as the Give Kids a Chance Act of 2025. Among other things, the bill would renew FDA’s rare pediatric disease priority review voucher (PRV) program―which began to sunset after December 2024―through September 30, 2029. This PRV program is designed to incentivize the development of treatments for rare pediatric diseases by awarding a transferable and sellable voucher to a sponsor who receives approval for their rare pediatric disease drug or biological product. The voucher can be used to receive priority review for a separate product.
It Is Now Official: FDA Rescinds the LDT Rule
On September 19, FDA published in the Federal Register a notice of a final rule rescinding, as of the date of publication, the Laboratory Developed Tests (LDT) rule. The LDT rule, which was finalized in May 2024, was vacated by a federal court in the Eastern District of Texas in March 2025. To reflect the court’s order, FDA has removed the phrase “including when the manufacturer of these products is a laboratory” from 21 CFR 809.3(a). This change changes 21 CFR 809.3(a) to its wording prior to the effective date of the LDT rule. With the recission now finalized, the future of LDT regulation is unclear. We further examine the future of LDT regulation here.