FDA grants orphan drug designation to posoleucel for virus-associated hemorrhagic cystitis
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The FDA granted orphan drug designation to posoleucel for the treatment of virus-associated hemorrhagic cystitis, according to the agent’s manufacturer.
Virus-associated hemorrhagic cystitis — an inflammatory disease that affects the bladder — is a frequent complication of hematopoietic stem cell transplant that can prolong hospitalization and increase mortality risk.
Posoleucel (Viralym-M, AlloVir) is an allogeneic, virus-specific T-cell therapy designed for the treatment and prevention of several viral pathogens, including BK virus, polyomavirus JC virus, cytomegalovirus, human herpesvirus-6, Epstein-Barr virus and adenovirus.
The FDA previously granted posoleucel its regenerative medicine advanced therapy designation, intended for therapies that show preliminary evidence to address unmet clinical need.
“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,” Ercem Atillasoy, MD, chief regulatory and safety officer at AlloVir, said in a company-issued press release. “We look forward to working with the FDA and regulators around the globe as we advance this therapy for patients in need.”
AlloVir is currently enrolling patients in a phase 3 clinical trial of posoleucel for the treatment of virus-associated hemorrhagic cystitis. The multicenter, double-blind, placebo-controlled study aims to assess the efficacy and safety of posoleucel for patients after allogeneic HSCT.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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