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.

Richard Blackburn

“We are very pleased that adult and pediatric organ transplant recipients will soon have access to the only FDA-approved ready-to-use oral liquid formulation of mycophenolate,” Richard Blackburn, CEO of Azurity Pharmaceuticals, said in a news release. “Myhibbin’s ready-to-use formulation provides patients, pharmacists, and caregivers an alternative to other mycophenolate dosage forms.”

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.

Another mycophenolate mofetil product, CellCept, also has a REMS program because of the risks for miscarriage and birth defects. For both Myhibbin and CellCept, birth control is recommended for women.

CellCept is marketed by Genentech as capsule, a tablet and as an oral suspension. The cost for CellCept oral capsule 250 mg is around $957 for a supply of 100 capsules, according to Drugs.com.

The approval of Myhibbin is based on five clinical trials. In these trials, estimated total of 1,557 adult patients received mycophenolate mofetil. Of these, 991 were included in the three renal studies, 277 were included in one hepatic study, and 289 were included in one cardiac study. About 53% of the kidney transplant patients, 65% of the heart transplant patients, and 48% of the liver transplant patients were treated for more than one year.

Three studies were conducted with patients who had kidney transplants: two studies compared two dose levels of oral mycophenolate mofetil with azathioprine and study compared mycophenolate mofetil with placebo. In all three studies, mycophenolate mofetil were administered in combination with cyclosporine (Sandimmune) and corticosteroids. In these trials, ycophenolate mofetil, in combination with corticosteroids and cyclosporine, reduced the incidence of treatment failure within the first 6 months following transplantation

One study enrolled patients who had heart transplantation study and compared mycophenolate mofetil with azathioprine, administered in combination with cyclosporine (Sandimmune) and corticosteroids. This study found that mycophenolate mofetil was as effective at preventing death or re-transplantation as azathioprine at one year.

One study enrolled patients who had liver transplants and compared mycophenolate mofetil with azathioprine, administered in combination with cyclosporine (Neoral) and corticosteroids. In these patients, mycophenolate mofetil demonstrated a lower rate of acute rejection at six months and a similar rate of death or re-transplantation at one year as azathioprine.

Adverse reactions reported in about 20% of patients in the mycophenolate mofetil treatment groups and included bacterial and viral infections, blood and lymphatic disorders, metabolism and nutrition disorders. Leukopenia (low white blood count), anemia (low red blood count), thrombocytopenia (low platelet count) are known risks associated with mycophenolate.