This Week at FDA: Reactions to FDA’s Bayesian methods guidance, GDUFA science report, and more

Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug, and medical device regulation and what we’re reading from around the web. This week, biostatistics experts took to JAMA to discuss the benefits and potential pitfalls of FDA’s recent draft guidance on Bayesian methods, FDA published a report on its GDUFA science program, and the agency released its OMUFA facility fee rates for FY 2026.

JAMA published three articles from academic experts this week reacting to FDA's new guidance on using Bayesian statistical methods in premarket applications. FDA Commissioner Marty Makary has been a strong proponent of using Bayesian methods to evaluate products, and the agency published a draft guidance in January outlining its thinking. Since then, industry and other stakeholders have chimed in with mostly positive comments and asked for specific clarifications.

Andrew Gelman, a statistician at Columbia University, and his co-authors lauded FDA's efforts and said Bayesian statistics are  "well suited to the FDA’s mission and principles." Similarly, J. Jack Lee, a biostatistician at the University of Texas MD Anderson Cancer Center, and his co-authors said the importance of the guidance cannot be overstated and shows FDA's commitment to modernizing clinical research.

Scott Evans, a Biostatistician at the Milken Institute School of Public Health at George Washington University, and his co-authors, however, had a different take. They argued that framing the guidance as modernizing statistical methods misses the mark and endangers the tried-and-true methods of clinical trial objectivity. They noted that FDA's proposal to expand the use of Bayesian methods could compromise the current methods used to ensure the replicability, integrity, and reliability of clinical trial results.

The Center for Drug Evaluation and Research (CDER) published its 2025 Generic Drug User Fee Amendments (GDUFA) Science and Research Report. It noted that last year it issued 925 product-specific guidances (PSG) for developing generic drugs and generating evidence for abbreviated new drug applications (ANDA).

FDA has published its over-the-counter monograph drug facility fee rates for the FY 2026. The agency noted the base revenue for FY 2026, without certain adjustments, is $36.5 million.

Drugs & Biologics

Denali Therapeutics' Avlayah (tividenofusp alfa-eknm) was approved by FDA to treat certain patients with Hunter syndrome (Mucopolysaccharidosis type II or MPS II), a rare inherited lysosomal disease. The drug is administered as a weekly intravenous infusion for presymptomatic or symptomatic pediatric patients with Hunter syndrome weighing at least 5 kg prior to advanced neurologic impairment.

FDA approved Rocket Pharmaceuticals' Kresladi (marnetegragene autotemcel) as the first gene therapy to treat severe Leukocyte Adhesion Deficiency Type I (LAD-I), a rare inherited immune deficiency disease caused by mutations in the ITGB2 gene. The drug was indicated for children with severe leukocyte adhesion deficiency I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant.

Corcept Therapeutics’ glucocorticoid receptor antagonist Lifyorli (relacorilant), in combination with nab-paclitaxel, was approved to treat adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens, at least one of which included bevacizumab. The drug is contraindicated for patients who need corticosteroids for a lifesaving indication and includes warnings and precautions for neutropenia and severe infections, adrenal insufficiency, exacerbation of conditions treated with glucocorticoids, and embryo-fetal toxicity.

Medtech

The Center for Devices and Radiological Health (CDRH) reported that 115 devices have so far enrolled in its Total Product Lifecycle Advisory Program (TAP) pilot. Five health technology offices and divisions are currently part of the pilot, and starting on 1 May, it will expand to include the Division of Renal, Gastrointestinal, Obesity and Transplant Devices (DHT3A) and the Office of Surgical and Infection Control Devices (OHT4).

Navajo Manufacturing Company has issued a class I recall for its Handy Solutions Neck & Shoulders Heating Pads due to concerns they may burn users and their surroundings, according to FDA. The company has asked people to dispose of the heating pads and request replacements from a different manufacturer.

Erbe USA has issued a class I recall for certain lots of its Flexible Cryoprobes due to concerns they could rupture or burst during use, according to FDA. The probes are used to destroy tissue during interventional procedures, and so far, the company has reported five serious injuries related to the affected devices.

CDRH issued an early alert to consumers for certain B. Braun Medical Streamline Airless System Hemodialysis Bloodlines and B3 Low Volume Bloodlines due to initial testing that found a change to the tubing resin could cause small air bubbles sticking to the inside of the arterial bloodline that could ultimately lead to delayed treatment when transferring blood between a patient’s vascular access device and a hemodialyzer during hemodialysis. The company has issued recommendations to mitigate the issue, and the agency said it is still investigating the problem.

FDA has reclassified optical diagnostic devices for detecting melanoma and electrical impedance spectrometers from class III to class II devices with special controls. The agency also renamed the device to software-aided adjunctive diagnostic devices for use on skin lesions by physicians trained in the diagnosis and management of skin cancer.

FDA also proposed reclassifying mycobacterium tuberculosis cell-mediated immunity tests and immune response enzyme-linked immunospot tests from class III devices to class II devices with special controls. The devices are used to help diagnose mycobacterium tuberculosis infections and based on recommendations of the Microbiology Devices Panel and lack of safety signals, the agency said it is proposing to down-classify the products.