FDA Accepts Veloxis' Envarsus NDA For Kidney Transplant Rejection

Hørsholm-based Veloxis Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted for standard review its new drug application (NDA) for Envarsus for the prevention of organ rejection in adult patients with kidney transplants.

Envarsus is an investigational drug developed as a once daily tablet version of tacrolimus, a leading immunosuppression drug used to prevent transplant allograft rejection after organ transplant. Envarsus has improved bioavailability, consistent pharmacokinetic performance, and reduced peak to trough variability against existing tacrolimus product.

Organ transplant patients are required to maintain a minimum blood level of tacrolimus to prevent transplant allograft rejection. On the other hand, excessive levels of tacrolimus may increase risk of nephrotoxicity, tremor, high blood pressure, diabetes, opportunistic infections, and other serious side effects.

The drug is based on Veloxis’ proprietary MELTDOSE technology. The technology has received regulatory acceptance through the previous FDA approval of Fenoglide. MELTDOSE makes it possible to decrese intra-individual variability to improve efficacy or side effect profiles and reduce number of adverse events. The company said that these benefits, together with reduction in peak-to-trough ratio and administration frequency, will lead to better transplant outcomes especially in non-compliant organ transplant patients. Veloxis credits its technology for the ability to manufacture a product candidate with a once-daily administration schedule such as Envarsus compared to the twice-daily schedule of currently marketed Prograf.

The NDA for Envarsus was submitted on December 2013 and was based on the two Phase III studies 3001 and 3002. In the trials, the drug demonstrated on par performance with twice daily tacrolimus (Prograf) based on a composite endpoint of treatment failure at one year. The clinical program comprised of 25 studies and involved more than 1,000 patients.

The FDA has set a target review date of October 3, 2014 for Envarsus’ NDA under the Prescription Drug User Free Act (PDUFA). The company had previously been granted Orphan Drug Designation by the FDA for prophylaxis of organ rejection in patients who have received allogeneic kidney transplants.