FDA grants orphan drug designation to ATA230 for cytomegalovirus

The FDA granted orphan drug designation to ATA230 for the treatment of cytomegalovirus viremia and disease in immunocompromised patients, according to the drug’s manufacturer.

ATA230 (Atara Biotherapeutics) is an allogeneic T-cell immunotherapy that targets antigens expressed by cytomegalovirus. The condition causes potentially life-threatening disease in patients with weakened immune systems, including bone marrow and solid organ transplant recipients, as well as individuals with HIV and symptomatic congenital cytomegalovirus viremia infections.

A phase 1 study and two phase 2 clinical trials have investigated ATA230 for patients with cytomegalovirus viremia and disease who are refractory or resistant to antiviral drug treatment.

“We are delighted that the therapeutic potential of our allogeneic T-cell immunotherapies in orphan diseases continues to be recognized by the FDA,” Isaac Ciechanover, MD, CEO and president of Atara Biotherapeutics, said in a company-issued press release.

“We believe that the high unmet medical need of immunocompromised patients with antiviral refractory or resistant cytomegalovirus viremia and compelling ATA230 clinical data provide a strong rationale for continued development,” Ciechanover added. “We look forward to further evaluating ATA230 development plans with the FDA and other global health authorities following the initiation of our ATA129 EBV-PTLD phase 3 studies.”

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.