FDA Approves Myhibbin for Prophylaxis of Organ Rejection in Adult, Pediatric Transplant Recipients
Mycophenolate mofetil oral suspension is the only FDA-approved ready-to-use liquid formulation.
The FDA has approved mycophenolate mofetil oral suspension (Myhibbin), an antimetabolite immunosuppressant indicated for prophylaxis of organ rejection in both adults and pediatric recipients (aged 3 months and older) of allogenic kidney, heart, or liver transplants, in combination with other immunosuppressant therapies.1
According to the Health Resources and Services Administration, there are over 130,000 adults and children on the national organ transplant waiting list, with more than 46,000 organ transplant surgeries performed in 2023.2 The kidneys, heart, and liver are among the most commonly transplanted organs in the country, representing a significant patient population poised to benefit from the approval.3
Mycophenolate mofetil oral suspension is approved for use in patients receiving heart, liver, or kidney transplants. | image credit: vchalup - stock.adobe.com
“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,” said Richard Blackburn, Azurity Pharmaceuticals CEO.1 “Myhibbin’s ready-to-use formulation provides patients, pharmacists, and caregivers an alternative to other mycophenolate dosage forms.
A number of clinical trials contributed to FDA approval.4 For kidney transplants, 3 de novo studies compared 2 dose levels of oral mycophenolate mofetil (1 g and 1.5 g, both twice daily) with azathioprine or placebo (2 studies and 1 study, respectively) to prevent acute rejection episodes. The primary efficacy endpoint across all 3 studies was the proportion of patients in each treatment group who experienced treatment failure within the first 6 months following transplantation.
Heart transplant data were evaluated from a double-blind, randomized, comparative, parallel-group, multicenter study of 578 primary de novo heart transplant recipients. Patients received either mycophenolate mofetil 1.5 g twice daily or azathioprine 1.5 mg/kg/d to 3 mg/kg/day plus cyclosporine and corticosteroids. The first primary efficacy endpoint was the proportion of patients who had at least 1 endomyocardial biopsy-proven rejection with hemodynamic compromise, were re-transplanted, or died, within 6 months of surgery; the second primary efficacy endpoint was the proportion of patients who died or were re-transplanted during the first 12 months after transplantation.
Liver transplant data were evaluated from a double-blind, randomized, comparative, parallel-group, multicenter study of primary hepatic transplant recipients. Similar to the heart transplant study, 565 participants received either mycophenolate mofetil 1.5 g twice daily or azathioprine 1 mg/kg/day to 2 mg/kg/day plus cyclosporine and corticosteroids. Primary endpoints were the same.
Mycophenolate mofetil oral suspension contains a boxed warning for embryofetal toxicity, malignancies, and serious infections.5 The use of this medication during pregnancy is associated with increased risks of pregnancy loss and congenital malformations; it should be avoided if safer treatment options are available. The drug has also been associated with an increased risk in the development of lymphoma and other skin malignancies.
Patients who receive immunosuppressant therapies have an increased susceptibility to bacterial, viral, fungal, and protozoal infections, including opportunistic infections and viral reactivation of hepatitis B and hepatitis C. Health care providers should consider reducing the dose of, or discontinuing treatment with, mycophenolate mofetil oral suspension in patients who develop new infections or who reactivate viral infections.
The most common adverse reactions, experienced in 20% or more of clinical trial participants, include diarrhea, leukopenia, infection, and vomiting.
Dosing for adult and pediatric patients is as follows:
- Adult patients
- Kidney transplant: 1 g, twice daily
- Heart Transplant: 1.5 g, twice daily
- Liver Transplant: 1.5 g, twice daily
- Pediatric patients:
- Kidney transplant: 600 mg/m2 twice daily, up to a maximum of 2 g daily
- Heart transplant: 600 mg/m2 twice daily, up to a maximum of 900 mg/m2 twice daily (maximum daily dose of 3 g or 15 mL oral suspension)
- Liver transplant: 600 mg/m2 twice daily, up to a maximum of 900 mg/m2 twice daily (maximum daily dose of 3 g or 15 mL oral suspension)
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