FDA Expands Indication for Platelet Booster Avatrombopag (Doptelet)

Megan Brooks

June 28, 2019

The US Food and Drug Administration has approved an expansion of the use of the platelet booster avatrombopag (Doptelet, Dova Pharmaceuticals) to include adults with thrombocytopenia and chronic immune thrombocytopenia (ITP) that has not responded adequately to previous treatment.

Avatrombopag is a second-generation oral thrombopoietin receptor agonist that stimulates platelet production. It is already approved in the United States and Europe as a therapy to increase platelet counts in adults with thrombocytopenia and chronic liver disease who are to undergo a planned medical or dental procedure.

ITP is a rare, autoimmune bleeding disorder that affects roughly 60,000 adults in the United States. It is characterized by a reduction in the number of platelets, which leads to excessive bruising and severe bleeding.

Supportive efficacy data for the ITP supplemental new drug application came from two phase 2 ITP clinical trials and two phase 3 trials for the treatment of thrombocytopenia in patients with chronic liver disease. The safety and tolerability of avatrombopag are supported by data for 128 patients with ITP and for more than 1000 patients who were treated in 24 studies for multiple indications, the company said in a news release. The studies were conducted in the clinical development program for the drug.

ITP patients and clinicians should work together to "choose a therapy that supports their lifestyle and aims to achieve the best possible result to treat their ITP. That's why having additional treatment options are so important," Caroline Kruse, president and CEO of the Platelet Disorder Support Association, a patient advocacy organization dedicated to ITP patients, said in the release.

"We are thrilled to have a new, oral TPO-RA [thrombopoietin receptor agonist] available for adult patients with ITP. Every new treatment provides more choices and new hope to our community," said Kruse.

The company said the pricing of avatrombopag will be in line with similar agents used to treat ITP and that it will continue to offer patients assistance and copay programs. It expects to launch the drug next month.

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