FDA News

FDA grants orphan drug designation to Doptelet for chemotherapy-induced thrombocytopenia

The FDA granted orphan drug designation to avatrombopag for the treatment of chemotherapy-induced thrombocytopenia.

Avatrombopag (Doptelet, Dova Pharmaceuticals) — an oral thrombopoietin receptor agonist — already is approved in the United States for treatment of thrombocytopenia among adults with chronic liver disease who are scheduled to undergo a procedure. It also is approved for treatment of adults with chronic immune thrombocytopenia who had an insufficient response to a prior treatment.

A phase 3 study is underway to evaluate the agent for treatment of chemotherapy-induced thrombocytopenia, which results in low platelet levels and can lead to chemotherapy dose reductions or delays.

An estimated 10% of patients with cancer in the United States experience this condition.

“Chemotherapy-induced thrombocytopenia is a potentially serious complication of chemotherapy with no available treatments,” Mahmood Ladha, president and head of Dova Pharmaceuticals, said in a company-issued press release. “The granting of orphan drug designation highlights the significant need for a drug that could improve chemotherapy-induced low platelet counts and thereby enable patients to better adhere to their chemotherapy regimen.”

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.

The FDA granted orphan drug designation to avatrombopag for the treatment of chemotherapy-induced thrombocytopenia.

Avatrombopag (Doptelet, Dova Pharmaceuticals) — an oral thrombopoietin receptor agonist — already is approved in the United States for treatment of thrombocytopenia among adults with chronic liver disease who are scheduled to undergo a procedure. It also is approved for treatment of adults with chronic immune thrombocytopenia who had an insufficient response to a prior treatment.

A phase 3 study is underway to evaluate the agent for treatment of chemotherapy-induced thrombocytopenia, which results in low platelet levels and can lead to chemotherapy dose reductions or delays.

An estimated 10% of patients with cancer in the United States experience this condition.

“Chemotherapy-induced thrombocytopenia is a potentially serious complication of chemotherapy with no available treatments,” Mahmood Ladha, president and head of Dova Pharmaceuticals, said in a company-issued press release. “The granting of orphan drug designation highlights the significant need for a drug that could improve chemotherapy-induced low platelet counts and thereby enable patients to better adhere to their chemotherapy regimen.”

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.