FDA News

FDA grants orphan drug designation to veltuzumab for ITP

The FDA granted orphan drug designation to veltuzumab — a humanized anti-CD20 antibody — for the treatment of patients with immune thrombocytopenia, according to a press release from the drug’s manufacturer.

Results of a phase 1 study showed low-dose, single-agent veltuzumab (Immunomedics) demonstrated an objective response rate of 55% in 38 response-assessable patients with relapsed immune thrombocytopenia (ITP). Eleven patients (29%) experienced a complete response.

Ten responders achieved durable responses for more than 1 year, including three patients who remained in remission for up to 4.3 years after receiving veltuzumab.

A phase 2 expansion trial has completed patient accrual and researchers plan to follow patients for 5 years.

“We are currently evaluating various options for further clinical development of veltuzumab in ITP and other autoimmune disease indications, including licensing arrangements and collaborations with outside study groups,” Cynthia L. Sullivan, president and CEO of Immunomedics, said in a press release.

The FDA’s 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 FDA granted orphan drug designation to veltuzumab — a humanized anti-CD20 antibody — for the treatment of patients with immune thrombocytopenia, according to a press release from the drug’s manufacturer.

Results of a phase 1 study showed low-dose, single-agent veltuzumab (Immunomedics) demonstrated an objective response rate of 55% in 38 response-assessable patients with relapsed immune thrombocytopenia (ITP). Eleven patients (29%) experienced a complete response.

Ten responders achieved durable responses for more than 1 year, including three patients who remained in remission for up to 4.3 years after receiving veltuzumab.

A phase 2 expansion trial has completed patient accrual and researchers plan to follow patients for 5 years.

“We are currently evaluating various options for further clinical development of veltuzumab in ITP and other autoimmune disease indications, including licensing arrangements and collaborations with outside study groups,” Cynthia L. Sullivan, president and CEO of Immunomedics, said in a press release.

The FDA’s 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.