Veloxis Pharmaceuticals seeks FDA approval to market Envarsus for prevention of organ rejection

Veloxis Pharmaceuticals A/S (OMX: VELO), today announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval for the marketing and sale of Envarsus^® (formerly LCP-Tacro™) for the prevention of organ rejection in kidney transplant recipients. The submission is based on the entire data set from the company's clinical development program that comprised 25 studies and enrolled over 1000 patients, including two pivotal clinical trials, studies 3001 and 3002. In the two pivotal trials, Envarsus^® given once-daily met the primary endpoint of demonstrating comparable efficacy and safety compared to twice-daily tacrolimus (Prograf^®, Astellas Pharma, Inc.). In the largest study, Study 3002, Envarsus^® was shown to have a primary efficacy failure rate of 18.3% vs 19.6% with Prograf therapy.  The differences between the treatments was well-within the pre-defined 10% non-inferiority margin. The study was conducted under a Special Protocol Agreement with the FDA.  Envarsus^® has been granted Orphan Drug status by the FDA for prophylaxis of rejection for kidney transplant recipients.

"The NDA filing marks another significant milestone for Veloxis as we move closer in bringing Envarsus^® to patients in the U.S.," said William Polvino, M.D., president and chief executive officer of Veloxis. "Envarsus' once daily formulation provides an opportunity for an improved treatment experience for transplant patients while offering comparable efficacy and safety to the twice-daily tacrolimus, as was demonstrated most recently by our successful Phase 3 clinical trials, studies 3002 and 3001. Our commercial preparation activities in the U.S. are underway. We are planning to build a sales force for the marketing of Envarsus^® in the U.S. and adequate financing is in place to support development, differentiation and commercialization activities through the initial launch of Envarsus^®."

John Weinberg, M.D, chief commercial officer of Veloxis added, "The need for kidney transplants is significant with 17,000 kidney transplants performed annually in the U.S and about 180,000 kidney transplant recipients living in the US. Most patients are prescribed tacrolimus to prevent rejection of their transplanted kidney. Transplants are performed at approximately 250 highly specialized centers across the U.S. and Veloxis anticipates hiring a sales force of approximately 20 representatives to call on the key transplant centers. We look forward to the opportunity to bring this new treatment option to transplant patients and physicians once Envarsus^® has been approved."

In May 2013, Veloxis received the acceptance of its European Marketing Authorisation Application Filing, based on the Phase I and II studies, as well as the pivotal trial, Study 3001. Outside the U.S., Veloxis plans to launch and commercialize Envarsus^® through partnering arrangements. In Europe, Turkey and the CIS countries, Envarsus^® will be commercialized by Veloxis' partner Chiesi Farmaceutici.