Pervasis Therapeutics Inc, a biotechnology company pioneering biologically active cellular therapies to treat vascular and other serious diseases, has received orphan drug designation for Vascugel, a novel allogeneic cell therapy product that may restore natural repair and regeneration pathways in the vasculature, from the US Food and Drug Administration (FDA).

The orphan drug designation is granted for the prevention of arteriovenous fistula or arteriovenous graft failure in patients with end-stage renal disease (ESRD) receiving haemodialysis or preparing for haemodialysis.

The orphan drug designation is a distinguished status granted by the FDA under the orphan drug Act for a product that treats a rare disease or condition. Companies that receive orphan drug designation for their products may receive funding for certain clinical trials, clinical trial design assistance, tax credits, a waiver of Prescription Drug User Fee Act (PDUFA) fees and marketing exclusivity for up to seven years following regulatory approval. The orphan drug Act is designed to encourage companies to develop therapies to treat rare diseases or conditions.

Vascugel is a novel and unique allogeneic cell therapy product under investigation for enhancing repair, extending patency and promoting vascular health. Vascugel builds on concepts of tissue engineering to enable implantation of allogeneic endothelial cells in a controlled state. When placed adjacent to an injured blood vessel, the endothelial cells in Vascugel provide growth regulatory compounds to the underlying blood vessel, which promotes a natural healing process and may prevent excessive inflammation, thrombosis and stenosis. Vascugel has completed two Phase 2 clinical trials in patients with end stage renal disease. The studies achieved the primary safety endpoints, demonstrating fewer thrombotic events, early complication and interventions than placebo patients. Additionally, positive efficacy trends were shown in secondary endpoints.

"We are very pleased to receive orphan drug designation from the FDA for Vascugel," said Frederic Chereau, president and chief executive officer of Pervasis. "The designation is for a broad array of vascular accesses for patients with end-stage renal disease, including placement of an arteriovenous graft or arteriovenous fistula for haemodialysis and revisions to existing accesses. Vascular access failure is a major complication to providing care to patients on haemodialysis with end-stage renal disease. Vascugel is a potential new treatment option that may improve outcomes for these patients, and the first in a portfolio of novel cell-based therapeutics we are developing to treat vascular injury by harnessing the body's natural healing process. We are working closely with the FDA as we move forward with plans for a Phase 3 trial for Vascugel, and are in active discussions with potential partners to bring this product to market."

According to the United States Renal Data System, the number of ESRD patients requiring haemodialysis in 2006 was over 325,000. Medicare data shows that vascular access complications account for up to 25 percent of all haemodialysis patient admissions. The majority of vascular access is achieved through the surgical creation of synthetic arteriovenous graft (AVG) or autogenous arteriovenous fistulae (AVF). Vascular access failure is the single most important cause of morbidity in the haemodialysis population.