Benzinga | Oct 4, 2021 07:05AM EDT

AlloVir Announces The FDA Has Granted Orphan Drug Designation To The Co.'s Posoleucel, An Allogeneic, Off-the-Shelf, Multi-Virus Specific T-Cell Therapy, For The Treatment Of Virus-Associated Hemorrhagic Cystitis

AlloVir, Inc. (NASDAQ:ALVR), a late clinical-stage cell therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to posoleucel (Viralym-M, ALVR105) for the treatment of virus-associated hemorrhagic cystitis (HC). Posoleucel is an investigational, allogeneic, off-the-shelf, multi-virus specific T cell therapy under development for the treatment and prevention of serious diseases caused by six devastating viral pathogens: BK virus (BKV) and the related polyomavirus JC virus (JCV), cytomegalovirus (CMV), human herpes virus-6 (HHV-6), Epstein Barr virus (EBV) and adenovirus (AdV).

An inflammatory disease of the bladder, virus-associated HC is a serious complication of hematopoietic stem cell transplantation (HSCT) that can significantly prolong hospitalization and increase mortality and for which there are currently no approved or effective antiviral treatment options. The company's Phase 3 study of posoleucel for the treatment of virus-associated HC is ongoing and enrolling adult and pediatric patients following allogeneic HSCT (allo-HSCT).

"This Orphan Drug Designation acknowledges the urgent need for new treatment options for patients who have undergone hematopoietic stem cell transplantation and are at risk for developing viral infections and hemorrhagic cystitis," said Ercem Atillasoy, M.D., Chief Regulatory and Safety Officer, AlloVir. "We look forward to working with the FDA and regulators around the globe as we advance this therapy for patients in need."

In addition to this ODD, posoleucel has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, and Orphan Medicinal Product (OMP) and PRIority MEdicines (PRIME) designations from the European Medicines Agency (EMA). Posoleucel is one of the first seven investigational therapies to receive both PRIME and RMAT designations.

ODD is granted by the FDA to drugs or biological products intended for the treatment of diseases or conditions that impact fewer than 200,000 people in the U.S. The designation acts as a stimulus for the development of drugs for rare diseases through several incentives, including research and development tax credits, waiver of filing fees and the potential for a seven-year marketing exclusivity period after FDA approval. Drugs with this designation undergo the same rigorous scientific evaluation as other investigational medicines seeking FDA approval.