FDA Approves Dova’s Drug for Restoring Low Blood Platelets

Dova Pharmaceuticals of Durham has received approval from the U.S. Food and Drug Administration for its first commercial drug, a tablet for treating thrombocytopenia, or low blood platelet counts, which can cause dangerous bleeding.

The approval of avatrombopag, to be marketed as Doptelet, allows the drug’s use in patients with chronic liver disease who are scheduled to undergo a minor medical or dental procedure – such as a biopsy, colonoscopy or tooth extraction – to prevent excessive bleeding.

The drug also should allow most patients to avoid a platelet transfusion prior to a procedure by increasing platelet counts to a safe level of 50,000 per microliter or higher, said Alex C. Sapir, president and chief executive officer of Dova.

“We are delighted FDA has approved Doptelet, which represents a significant milestone for Dova, physicians, and most importantly, patients,” Sapir said in a news release. Dova will launch the drug in June with a “full complement of sales, marketing and reimbursement-support resources,” he said.

Dova began hiring a sales force in January to prepare for the launch.

There are no other approved treatments for thrombocytopenia prior to medical procedures in chronic liver disease patients, the company said.

Oral dosing minimizes risk, helps patients manage

“Given the need for patients with chronic liver disease to routinely undergo multiple, invasive procedures, the availability of an oral agent that can lead to a measured increase in platelets, to minimize the need for platelet transfusions and risk of bleeding, will facilitate the clinical management of these patients,” said Norah Terrault, M.D, M.P.H., professor of medicine in the Division of Gastroenterology at the University of California San Francisco. Terrault was the principal investigator for the Phase 3 trials for avatrombopag.

The FDA approval came after the drug met all primary and secondary endpoints for safety and efficacy in two global Phase 3 trials.

“Patients with chronic liver disease who have low platelet counts and require a procedure are at increased risk of bleeding,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Doptelet was demonstrated to safely increase the platelet count. This drug may decrease or eliminate the need for platelet transfusions, which are associated with risk of infection and other adverse reactions.”

Doptelet is first of its kind in U.S. for chronic liver disease

Doptelet is a second-generation, once-daily, orally administered thrombopoietin (TPO) receptor agonist. It is the first TPO agonist to be approved in the United States for the chronic liver disease indication.

TPO, the body’s main regulator of platelet production, is made in the liver and stimulates bone marrow production of platelets, the colorless blood cells that help blood clot. Liver damage in patients with chronic liver disease reduces TPO production, resulting in decreased platelet production and thrombocytopenia.

An estimated 70,000 chronic liver disease patients have a platelet count of less than 50,000 per microliter. They typically require one to three invasive diagnostic and therapeutic procedures per year, and each carries a risk of bleeding. If not effectively treated, thrombocytopenia can lead to serious uncontrolled bleeding, resulting in prolonged hospitalizations and other post-procedure complications.

Dova is developing other potential applications of Doptelet including more invasive surgical procedures and chemotherapy-induced thrombocytopenia.

The total addressable market for Doptelet is estimated at $2.5 billion, Dova said in a January presentation at the 36^th annual J.P. Morgan Healthcare Conference in San Francisco.

Dova, founded in 2016, raised about $79 million in an initial public offering of stock in 2017 and completed a secondary offering earlier this year that raised about $75 million. Its shares are traded on the NASDAQ stock market.

The company has about 120 employees.