FDA Removes REMS Requirements for All CAR-T Therapies

On June 27, 2025, the US Food and Drug Administration (FDA) removed the Risk Evaluation and Mitigation Strategies (REMS) requirements for approved B-cell maturation antigen (BCMA)- and CD19-directed autologous chimeric antigen receptor (CAR) T cell immunotherapies, including Abecma^® (idecabtagene vicleucel) and Breyanzi^® (lisocabtagene maraleucel) from Bristol Myers Squibb, Yescarta^® (axicabtagene ciloleucel) and Tecartus^® (brexucabtagene autoleucel) from Gilead Sciences, Carvykti^®  (ciltacabtagene autoleucel) from Johnson & Johnson and Kymriah^® (tisagenlecleucel-T) from Novartis. Of note, Autolus’ Aucatzyl^® (obecabtagene autoleucel) was approved in November 2024 without a REMS requirement. REMS is a drug safety program that the FDA can require for medications with serious safety concerns to ensure the benefits of the medication outweigh its risks. The REMS program was originally established to manage risks such as cytokine release syndrome and neurological toxicities. It required dispensing locations to be specially certified and have immediate access to tocilizumab for complications. However, the FDA has determined that these requirements are no longer necessary, as the risks can be adequately managed through existing product labeling, including boxed warnings and medication guides. This decision aims to improve access to CAR-T treatments, especially for patients in rural areas, by reducing logistical barriers and easing restrictions on post-infusion monitoring and driving. This change is expected to increase CAR-T therapy uptake and expand its administration to community cancer centers, potentially doubling its current use. Continuous safety monitoring and post-marketing studies will remain in place. More information may be found here.

Contraindications Added to Livmarli and Bylvay

The FDA announced safety-related changes to the prescribing information for ileal bile acid transport (IBAT) inhibitors Livmarli^® (maralixibat – Mirium Pharmaceuticals) and Bylvay^® (odevixibat – Albireo) on June 25, 2025. Used to treat pruritus in patients with Alagille syndrome (ALGS) or progressive familial intrahepatic cholestasis (PFIC), the updated labeling includes a contraindication for those with prior or active hepatic decompensation events and expanded warnings and precautions about hepatotoxicity and bleeding risks due to fat-soluble vitamin (FSV) deficiency. The revisions recommend monitoring liver function and provide guidance on when to discontinue use. Updated prescribing is here for Livmarli and Bylvay.

New Gammagard Low IgA Formulation

On June 30, 2025, the FDA granted approval to Takeda’s Gammagard Liquid ERC^® [immune globulin infusion (human)] a ready-to-use liquid immunoglobulin therapy with low IgA content (≤2 µg/mL in a 10% solution), for intravenous (IV) or subcutaneous (SC) use in patients aged two years and older with primary immunodeficiency (PI). PI comprises over 550 rare and chronic disorders caused by genetic mutations, often inherited, in which the immune system is either missing parts or does not function properly. Takeda also announced they will discontinue its first-generation low-IgA product, Gammagard S/D^®, by the end of 2027. Gammagard Liquid ERC provides a new treatment option that simplifies administration by eliminating the need for reconstitution. Commercialization in the US is projected to begin in 2026. Here is the prescribing information.

Gamifant’s New Indication Marks First Approval for Macrophage Activation Syndrome in Still’s Disease

Sobi announced the FDA approval of Gamifant^® (emapalumab-lzsg) on June 28, 2025, for treating hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with inadequate response or intolerance to glucocorticoids, or with recurrent MAS. MAS is a severe complication of rheumatic disorders and a secondary form of HLH. It is characterized by severe inflammation and can potentially lead to multiple organ failure and death. Approval was based on pooled data showing a 54% complete response rate and 82% achieving clinical MAS remission at week eight. Initially approved in 2018 to treat an extremely rare hereditary condition, primary HLH, Gamifant is an interferon gamma (IFNγ)–blocking antibody, helping to control hyperinflammation and reducing reliance on high-dose glucocorticoids. This marks the first FDA-approved treatment for MAS in Still’s disease. Updated prescribing information can be found here.

First Generic to Lotemax SM Approved

Lupin Announced the FDA approval of loteprednol etabonate ophthalmic gel, 0.38% on July 1, 2025, the first generic to Lotemax^® SM (Bausch & Lomb). A corticosteroid used to treat post-operative inflammation and pain following ocular surgery, Lupin will hold 180 days of generic drug exclusivity. Annual US sales for Lotemax SM are approximately $29 million.

FDA Approves First Complera Generic

Gilead’s three drug combination for treating HIV-1 infections, Complera^® (rilpivirine, emtricitabine and tenofovir disoproxil fumarate) received its first generic on May 20, 2025. Although Mylan has not released an announcement, multiple sources are reporting Mylan launched the non-nucleoside reverse transcriptase inhibitor, rilpivirine, with the two-drug combination, emtricitabine and tenofovir disoproxil fumarate, both of which are nucleoside reverse transcriptase inhibitors on May 27, 2025. US sales for Complera in 2024 were $91 million.

FDA Mandates New Labeling for ADHD Stimulants

On June 30, 2025, the FDA announced revisions to the labeling of all extended-release stimulants used to treat attention-deficit/hyperactivity disorder (ADHD), including certain formulations of amphetamine and methylphenidate, to warn about the risk of weight loss and other adverse reactions in patients younger than six years of age. Although not FDA approved for this age group, these medications can be prescribed off-label. The FDA’s analysis of clinical trial data revealed that children under six years of age have higher plasma exposures and increased rates of side effects, including significant weight loss (at least 10% decrease in the Centers for Disease Control and Prevention (CDC) weight percentile), compared to older children. The FDA is requiring manufacturers to include a Limitation of Use section in the prescribing information to reflect these findings. Parents and health care professionals are advised to monitor weight loss and consider alternative treatments if adverse effects occur. The updated prescribing information will ensure consistent messaging across the drug class. The FDA Drug Safety Communication can be found here.

FDA Expands Indications for Imaging Agent Neuraceq

On July 1, 2025, Life Molecular Imaging announced the FDA approved updated labeling for Neuraceq^® (florbetaben F18 injection) for the selection of patients for amyloid-directed therapies and the inclusion of quantitative analysis for positron emission tomography (PET) scans. This expanded indication now allows Neuraceq to be used in both diagnostic assessments and the identification of appropriate candidates for FDA-approved amyloid-targeting therapies. The updated label also supports broader use for monitoring therapy and tracking progression to Alzheimer’s disease (AD). Neuraceq binds selectively to amyloid plaques in the brain, enabling detection through PET imaging, which aids clinicians in determining if AD contributes to a patient’s cognitive symptoms. Updated prescribing information may be found here.

Ready-to-Use Vancomycin Formulation Approved

Hikma Pharmaceuticals announced on July 2, 2025, that the FDA approved Tyzavan^™ (vancomycin injection), a ready-to-infuse formulation of vancomycin. A glycopeptide antibacterial, Tyzavan is approved for the treatment of septicemia, infective endocarditis, skin and skin structure infections, bone infections and lower respiratory tract infections in patients aged one month and older. This new formulation simplifies the administration process by being the only FDA-approved vancomycin product available at room -temperature, eliminating the need for compounding, thawing, activation or dilution. This may be especially helpful in treating sepsis as survival rate decreases by 15% after 87-113 minutes. Tyzavan will be available in seven presentations (0.5g–2g) and has a shelf life of 16 months without refrigeration. Launch and cost are not yet known. Prescribing information will be available on the Drugs@FDA drug database at www.fda.gov.

Updates to Navigating the Diverging Paths of COVID-19 Vaccine Recommendations Issues Document

As discussed in the previous Issues Document, the recent policy shifts by the FDA and the Centers for Disease Control and Prevention (CDC) regarding coronavirus disease 2019 (COVID-19) vaccines have sparked confusion. The Advisory Committee on Immunization Practices met June 25-26, 2025, to discuss and vote on vaccine related topics. This document provides specific updates from the meeting, as well as their potential impact on public health. The updated Issues Document is attached.