FDA Press Releases

FDA Encourages Sponsors to Pursue New Testosterone Therapy Indication for Low Libido

The FDA announced it is taking an initial step to expand treatment options for men with low libido by encouraging sponsors of approved testosterone replacement therapy (TRT) products to express interest in pursuing a potential new indication for idiopathic hypogonadism. Following a preliminary review of published clinical literature and a December 2025 expert panel discussion, FDA identified data suggesting TRT may be safe and effective for certain men with low libido related to low testosterone without an identifiable cause.

Draft Guidance on Safety Assessment of Genome Editing Using Next-Generation Sequencing

The FDA's Center for Biologics Evaluation and Research issued draft guidance recommending next-generation sequencing methods for assessing off-target editing risks and genome integrity in human gene therapy products using genome editing technologies. The guidance applies to both ex vivo and in vivo products and provides recommendations on sequencing strategies, sample selection, analysis parameters, and reporting. Sponsors submitting investigational new drug applications or Biologics License Applications for genome editing therapies should incorporate these safety assessment methods into nonclinical studies. The FDA is accepting public comments for 90 days.

FDA Reminds 2,200 Sponsors to Disclose Trial Results

The FDA sent messages on March 30, 2026, to more than 2,200 companies and researchers associated with over 3,000 registered clinical trials that do not appear to have submitted required results information to ClinicalTrials.gov. The agency seeks voluntary compliance before considering further regulatory action, including potential Pre-Notices and Notices of Noncompliance.

FDA Approves Kresladi, First Gene Therapy for Severe LAD-I

The FDA has approved Kresladi (marnetegragene autotemcel), the first gene therapy for severe Leukocyte Adhesion Deficiency Type I (LAD-I) in pediatric patients. This accelerated approval offers a new treatment option for a rare inherited immune deficiency, targeting the underlying genetic cause of the disease.

FDA Approves Avlayah for Neurologic Manifestations of Hunter Syndrome

The FDA has approved Avlayah (tividenofusp alfa-eknm) for the treatment of neurologic manifestations of Hunter syndrome in certain pediatric patients. This marks the first drug approval specifically targeting the neurological complications of this rare genetic disorder.

FDA Seeks Comment on Priority Voucher Pilot Program

The FDA has published a Federal Register Notice seeking public comment on its Commissioner's National Priority Voucher pilot program. The agency also announced a public hearing on June 12, 2026, to gather stakeholder feedback on various aspects of the program's implementation and effectiveness.

FDA Approves Higher Dose Wegovy (semaglutide) for Weight Loss

The FDA has approved a new higher dose (7.2 mg) of Wegovy (semaglutide) injection, known as Wegovy HD, for weight management in certain adults. This approval was granted under the Commissioner's National Priority Voucher pilot program, aiming to expedite treatments for national health priorities.

FDA Draft Guidance on Alternatives to Animal Testing in Drug Development

The FDA has issued draft guidance to assist drug developers in validating new approach methodologies (NAMs) as alternatives to animal testing in drug development. This guidance supports the FDA's roadmap to reduce animal testing and bring safe, effective drugs to market sooner.

FDA Launches Unified Adverse Event Analysis Platform AEMS

The FDA has launched a new unified platform, the Adverse Event Monitoring System (AEMS), to analyze adverse event reports for all regulated products. This modernization aims to improve data accessibility, transparency, and surveillance capabilities, replacing fragmented legacy systems and is expected to save $120 million over five years.

FDA Approves Tec-Dara for Relapsed Multiple Myeloma

The FDA has approved Tec-Dara (teclistamab and daratumumab hyaluronidase-fihj) for relapsed or refractory multiple myeloma. This approval was granted under the Commissioner's National Priority Voucher (CNPV) pilot program, marking a significant acceleration in the review process.

FDA Meeting on State Drug Importation Program

The FDA held a meeting with several states to discuss the Section 804 Importation Program (SIP), which allows states to import certain prescription drugs from Canada. This meeting is part of the FDA's implementation of an executive order aimed at lowering drug prices for American consumers.

FDA Streamlines Biosimilar Development Guidance

The FDA issued draft guidance to streamline biosimilar development by recommending the reduction of unnecessary clinical pharmacokinetic testing when scientifically justified. This initiative aims to save developers up to 50% in PK study costs, potentially lowering drug prices and increasing patient access to affordable biologic treatments.

FDA Approves Wellcovorin for Cerebral Folate Deficiency

The FDA has approved Wellcovorin (leucovorin calcium) tablets for the treatment of cerebral folate deficiency in adult and pediatric patients with a confirmed FOLR1 gene variant. This marks the first FDA-approved treatment for this rare genetic condition.