FDA approves Novartis’ Zortress to prevent organ rejection after liver transplantation in adults

Novartis announced today that the US Food and Drug Administration (FDA) has approved Zortress^® (everolimus) for the prophylaxis of organ rejection in adult patients receiving a liver transplant. Zortress is the first mammalian target of rapamycin (mTOR) inhibitor approved for use following liver transplantation. It is also the first immunosuppressant approved by the FDA in over a decade for use following liver transplantation.

"Novartis has been a leading innovator in the transplant field for 30 years, and this FDA approval for liver transplantation marks an important milestone for patients and their transplant physicians in the US," said David Epstein , Division Head of Novartis Pharmaceuticals. "This second indication for Zortress in just three years in the US follows the recent European approval, further underscoring Novartis' continued commitment to bringing much needed treatment options to the transplant community worldwide."

The approval was based on the largest liver transplant study to date, which showed that Zortress plus reduced tacrolimus led to comparable efficacy and 10mL/min higher renal function as measured by estimated glomerular filtration rate (eGFR) for Zortress compared to standard tacrolimus at 12 months.

"While prevention of acute organ rejection is a key priority for physicians following liver transplantation, managing other health risks, including impaired renal function associated with calcineurin inhibitor treatment, is also critical," said John Fung , M.D., Ph.D., Director, Transplantation Center, Cleveland Clinic Foundation, Cleveland, OH. "With the approval of Zortress, the first mTOR inhibitor approved for liver transplant patients, we have a new treatment option that has the potential to address the unmet medical need for maintaining renal function without compromising acute rejection rates – and that's important for patients and physicians."

A large independent registry study of nearly 70,000 patients who received a non-renal solid organ transplant between 1990 and 2000 showed that the incidence of chronic renal failure was greater in liver transplant recipients than in recipients of all other solid organ transplants, except intestinal transplants. Calcineurin inhibitors (CNIs), such as tacrolimus, are part of the standard-of-care treatment regimen for immunosuppression in liver transplantation, but they can contribute to adverse reactions, including impaired renal function. Zortress works by binding to a protein called mTOR, and acts synergistically with CNIs, offering an opportunity to lower CNI exposure.

European Health Authorities approved Certican^® (everolimus) for the prophylaxis of organ rejection in adult patients receiving a liver transplant in the fourth quarter of 2012. In most EU member countries, Certican is also approved in kidney and heart transplantation. In the US, Zortress is already approved for use in adult kidney transplant patients.