Treatment of chemotherapy-induced thrombocytopenia! Doplet, an oral thrombopoietin receptor agonist, has been approved as an orphan drug by FDA!

December 30, 2019 / BIOON / -- dova pharmaceuticals, a wholly-owned subsidiary of Swedish orthophan biovitrum AB (SOBI), is a pharmaceutical company specializing in the development and commercialization of thrombocytopenia drugs Recently, dova announced that the U.S Food and Drug Administration (FDA) has granted doplet (avatrombopag) orphan drug qualification (odd) to treat chemotherapy-induced thrombocytopenia (CIT) Currently, a phase III study to evaluate the efficacy of the drug in patients with CIT is in progress CIT is a potential serious complication of chemotherapy, which can lead to the decrease of platelet level, the decrease of chemotherapy dose, the delay of chemotherapy dose or the change of chemotherapy scheme For cancer patients receiving therapeutic chemotherapy, changing chemotherapy regimen due to thrombocytopenia may impair their long-term prognosis In the United States, about 10% of cancer patients experience CIT every year, which may require changes in the chemotherapy regimen At present, there are no approved drugs for CIT in the United States or the European Union Orphan drug (Orphan Drug) is a kind of medicine used for prevention, treatment and diagnosis of rare diseases Rare diseases are a general term for diseases with very low incidence rate, also known as orphan diseases In the United States, rare diseases refer to the types of diseases with a population of less than 200000 Incentives for drug research and development of rare diseases include various incentives for clinical development, such as tax credits related to clinical trial costs, FDA user fee relief, FDA assistance in clinical trial design, and a 7-year market monopoly period for the approved indications after the drug is launched "Chemotherapy-induced thrombocytopenia (CIT) is a serious disease and an important obstacle for patients to adhere to chemotherapy regimens," said Milan zdravkovic, chief medical officer and head of research and development of SOBI At present, the treatment of CIT with avatrombopag is in the stage of phase III clinical development We are very pleased that FDA has granted avatrombopag orphan drug qualification in cit indications " Avatrombopag, the active drug component of doplet, is the second generation, once a day oral thrombopoietin receptor agonist (tpo-ra), which can simulate the effect of TPO, which is the main regulator of normal platelet production In the United States, doplet was approved by FDA in May 2018 for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who plan to undergo surgery In June this year, the FDA approved the extension of doplet for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who did not respond to previous therapies In the European Union, doplet was approved in June to treat severe thrombocytopenia in adults with CLD who plan to undergo invasive surgery Original source: FDA grants avorombopag orphan drug design for the treatment of chemotherapy induced trichocytotopia