Veloxis Pharmaceuticals, a specialty pharmaceutical company, has submitted a New Drug Application (NDA) to the US 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 programme 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 per cent vs 19.6 per cent with Prograf therapy. The differences between the treatments was well-within the pre-defined 10 per cent 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 US," said William Polvino, MD, 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 III clinical trials, studies 3002 and 3001. Our commercial preparation activities in the US are underway. We are planning to build a sales force for the marketing of Envarsus in the US and adequate financing is in place to support development, differentiation and commercialization activities through the initial launch of Envarsus."

John Weinberg, MD, chief commercial officer of Veloxis added, "The need for kidney transplants is significant with 17,000 kidney transplants performed annually in the US 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 centres across the US and Veloxis anticipates hiring a sales force of approximately 20 representatives to call on the key transplant centres. 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 US, 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.

Tacrolimus is a leading immunosuppression drug used for the prevention of transplant allograft rejection after organ transplantation. Envarsus is an investigational drug that is being developed as a once daily tablet version of tacrolimus, with improved bioavailability, consistent pharmacokinetic performance and reduced peak to trough variability when compared to currently approved tacrolimus products. Transplant patients need to maintain a minimum blood level of tacrolimus for the prevention of transplant allograft rejection, but excessive levels may increase the risk of serious side effects such as nephrotoxicity, tremor, diabetes, high blood pressure, and opportunistic infections. Therefore, tacrolimus levels need to be managed carefully, and transplant patients are typically obliged to make frequent visits to the hospital for monitoring and dose adjustments after receiving a new organ.