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FDA Updates for the Week of May 6: An Approval, a Delay and Two Ad Comm Meetings


The FDA has approved a liquid mycophenolate to prevent organ rejections but delayed the approval of Moderna’s RSV vaccine. Regulators have set dates for advisory committee meetings for midomafetamine for PTSD and for donanemab in Alzheimer’s disease. Additionally, the agency has set review dates for a subcutaneous Opdivo and a pancreatic/lung cancer therapy.

FDA Approves Liquid Mycophenolate to Prevent Organ Rejections

The FDA has approved Myhibbin, a ready-to-use mycophenolate mofetil oral suspension. Mycophenolate mofetil is an immunosuppressant used to prevent organ rejections.

In 2023, there were more 46,000 transplants in the United States, an 8.7% increase over 2022, according to the Health Resources and Service Administration, which is part of the Department of Health & Human Services. This continues a trend of increasing transplants across the country.

Developed by Azurity Pharmaceuticals, Myhibbin is indicated to prevent organ rejection in adult and pediatric recipients 3 months of age and older of allogeneic kidney, heart, or liver transplants, in combination with other immunosuppressants. Myhibbin is expected to be available in the second quarter of 2024. No information has been provided about pricing of Myhibbin.

Myhibbin has a black box warning regarding the risk of lymphoma and other malignancies, first trimester fetal loss and birth defects, and an increased risk of fungal, bacterial and fungal infections. Myhibbin has a pregnancy exposure Risk Evaluation and Mitigation Strategy (REMS) because of the risks of miscarriage and birth defects. The Mycophenolate Pregnancy Registry collects information about pregnancies that occur during treatment with mycophenolate or within six weeks after stopping.

Administrative Issues Delay FDA Decision on Moderna RSV Vaccine

The FDA has indicated that regulators will not be able to complete their review of the biologics license application (BLA) for mRNA-1345, Moderna's investigational respiratory syncytial virus (RSV) vaccine by the Prescription Drug User Fee Act (PDUFA) date of May 12, 2024. The delay is because of administrative issues and there are no issues related to vaccine safety, efficacy or quality that would prevent the approval.

Regulators are working to complete their review by the end of May 2024.

Moderna remains on track for mRNA-1345 to be reviewed at the CDC's Advisory Committee on Immunization Practices (ACIP) June 26-27, 2024, meeting, according to a company news release.

RSV is a common virus that can lead to potentially serious respiratory illness. Older adults, including those with underlying medical conditions such as diabetes and chronic heart and lung disease, are at increased risk of severe RSV illness and drive the majority of RSV hospitalizations. About 177,000 adults 65 years and older are hospitalized in the United States because of RSV.

mRNA-1345 is an investigational RSV vaccine that consists of a single mRNA sequence encoding for a stabilized prefusion F glycoprotein. The vaccine uses the same lipid nanoparticles as in the Moderna COVID-19 vaccines. When it launches, mRNA-1345 will be the only pre-filled, ready to use syringe. It will be indicated for adults ages 60 years of age and older.

The application is supported by data from a phase 3 study of about 37,000 adults. The study met its primary endpoint against RSV-associated lower-respiratory tract disease, with vaccine efficacy of 83.7% in those with two or more symptoms and vaccine efficacy of 82.4% in those with three or more symptoms.

Most adverse events were mild to moderate, and there were no cases of Guillain-Barre Syndrome. These data were published in December 2023 in the New England Journal Medicine.

FDA Sets Date for Advisory Committee Meeting for Midomafetamine for PTSD

The FDA will convene a meeting of the Psychopharmacologic Drugs Advisory Committee on June 4, 2024, to review data for midomafetamine to treat patients with post-traumatic stress disorder (PTSD). PTSD is a mental health condition affecting about 13 million Americans each year, yet currently available treatments provide only modest efficacy; there are high treatment discontinuation rates.

Developed by Lykos Therapeutics, midomafetamine is 3,4-methylenedioxymethamphetamine (MDMA) — commonly known as ecstasy — a class of psychoactive compounds. The FDA’s target action date is Aug. 11, 2024. If approved, this would be the first psychedelic-assisted therapy for post-traumatic stress disorder.

The committee will discuss the overall benefit-risk profile of the product, including the potential public health impact. Committtee members will review results from two phase 3 studies (MAPP1 and MAPP2) evaluating the efficacy and safety of MDMA.

FDA Sets Date of Advisory Committee Meeting for Donanemab in Alzheimer’s Disease

The FDA will convene an in-person meeting of the Peripheral and Central Nervous System Drugs Advisory Committee on Monday, June 10, 2024, to discuss the phase 3 trial of Lilly’s donanemab to treat patients with early symptomatic Alzheimer’s disease.

Regulators had said previously they want to discuss the safety results in donanemab-treated patients and the efficacy implications of the unique trial design of the TRAILBLAZER-ALZ 2 phase 3 study. Specifically, they want to discuss the trial’s use of limited-duration dosing regimen that allowed patients to complete treatment based on an assessment of amyloid plaque and the inclusion of participants based on tau levels.

Donanemab was under review for full approval to treat patients with early Alzheimer’s disease. A decision had been expected by the end of 2023. The FDA had previously said it wouldn’t grant accelerated approval of donanemab to treat patients with early Alzheimer’s disease. In a complete response letter in January 2023, the agency indicated that there too few patients with at least 12 months of data provided in Lilly’s submission. Lilly had resubmitted the application in July 2023 for full approval.

Lilly continues to study donanemab in multiple clinical trials, including TRAILBLAZER-ALZ-3, studying donanemab for the prevention of Alzheimer’s disease.

FDA Assigns Action Date for Subcutaneous Opdivo

The FDA has accepted Bristol Myers Squibb’s biologics license application (BLA) for the subcutaneous formulation of Opdivo (nivolumab) co-formulated with Halozyme’s recombinant human hyaluronidase (rHuPH20). Opdivo formulated with Halozyme’s technology allows for subcutaneous administration, or administration under the skin. Currently Opdivo is administered through an IV infusion.

Bristol Myers Squibb is seeking approval of the subcutaneous formulation for all 22 previously approved adult, solid tumor Opdivo indications across multiple cancers. Opdivo IV is approved as a monotherapy, monotherapy maintenance following completion of Opdivo plus Yervoy (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of Feb. 28, 2025.

FDA Accepts BLA for Pancreatic and Lung Cancer Therapy

The FDA has accepted for priority review Merus’s biologics license application (BLA) for zenocutuzumab (Zeno) to treat patients with non-small cell lung cancer and pancreatic cancer. The FDA’s target action date is in December 2024

The American Cancer Society predicts that in 2024, there will about 234,580 new cases of lung cancer and 66,440 people diagnosed with pancreatic cancer. NRG1 gene fusions are rare subtypes of these tumors, occurring in less than 1% of solid tumors.

Zeno is new type of bispecific antibody that is designed to target both HER2 and HER3 proteins and inhibit NRG1 binding. The company has on its website a video about how this works to impact tumor survival.

If approved, Zeno would be the first targeted therapy for a NRG1+ cancer.

The BLA is supported by data from the phase 1/2 eNRGy trial, an open-label study with multiple expansion cohorts. Phase 1 was a dose escalation study, which is complete. Phase 2 is dose expansion evaluating Zeno with various tumors with NRG1 fusions and is ongoing. The study aims to enroll 250 patients with NRG1 fusions, including non-small cell lung cancer and pancreatic cancer, as well as breast and cholangiocarcinoma (bile duct cancer).

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