FDA Approves Doptelet With Sprinkle Form for Pediatric Immune Thrombocytopenia

Doptelet and new oral sprinkle granules is approved for children one year and older with chronic immune thrombocytopenia, showing 28% durable platelet response in study.

The U.S. Food and Drug Administration (FDA) approved Doptelet (avatrombopag) for the treatment of thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia who have had an insufficient response to a prior therapy, according to a news release from Sobi.

In addition, the approval also includes a new formulation, Doptelet Sprinkle (avatrombopag) oral granules, for use in children ages one to less than six years.

“Doptelet represents a significant advancement in the treatment of children and adolescents with persistent or chronic immune thrombocytopenia,” Dr. Rachael Grace said in the news release. “This therapy offers simple, flexible administration because it is oral, available as a tablet and now as a new pediatric sprinkle formulation and has no food restrictions. The approval of Doptelet for pediatric immune thrombocytopenia offers families a new treatment option that can help address challenges in managing immune thrombocytopenia in pediatric patients.”

Grace is a pediatric hematologist and director, Hematology Clinical Research at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, and lead investigator of the AVA-PED-301 study, which was the basis for the approval.

The study found that 27.8% of patients receiving Doptelet reached the main goal of durable platelet response — meaning their platelet counts stayed at or above 50 billion per liter for at least six of the last eight weeks without needing rescue medication — compared with no patients in the placebo group.

Furthermore, 81.5% of Doptelet patients achieved the alternative platelet response endpoint, while no placebo patients did. By day 8, 55.6% of patients on Doptelet had platelet counts of 50 billion per liter or higher without rescue treatment, compared with none in the placebo group. Doptelet was generally well tolerated.

The most common side effects seen in children with persistent or chronic immune thrombocytopenia were viral infections, nasopharyngitis (common cold symptoms), cough, fever and sore throat.

“Since its introduction in 2019, Doptelet has been a cornerstone therapy for chronic ITP in adults,” Duane Barnes, President of Sobi North America, said in the news release. “This approval not only reinforces our commitment to innovation but also allows us to expand the treatment experience for patients and their families by offering Doptelet in two formulations.”

AVA-PED-301 is a global, randomized, phase 3 study that evaluated the efficacy, safety, and how the drug is processed in children with immune thrombocytopenia

What is Doptelet and How Does it Work?

Doptelet is an orally bioavailable, small molecule thrombopoietin receptor agonist (TPO-RA) that stimulates the proliferation and differentiation of megakaryocytes from bone marrow progenitor cells, resulting in increased production of platelets. DOPTELET does not compete with TPO for binding to the TPO receptor.

The most common adverse reactions (10% or more) in patients with chronic immune thrombocytopenia were headache, fatigue, contusion, epistaxis, upper respiratory tract infection, arthralgia, gingival bleeding, petechiae, and nasopharyngitis.

Serious adverse reaction that occurred more frequently in patients treated with Doptelet (9%; 12 of 128 patients) compared to placebo (5%; 1 of 22 patients) was headache, occurring in 1.6% (2 of 128 patients).

Previous New Drug Application

The FDA previously accepted a new drug application for Doptelet for the treatment of pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia who did not respond to earlier therapy, according to a new release from 2024. Data supporting the submission came from the AVA-PED-301 study, which met its primary endpoint of durable platelet response — achieved in 28% of patients during the last 8 weeks of the 12-week core phase without rescue therapy.

An additional efficacy measure was also met, with 81.5% of patients achieving two or more consecutive platelet counts at or above 50 × 10⁹ per liter without rescue medication over the same period.

References

1. “Sobi Announces U.S. Food and Drug Administration Approves Doptelet® (avatrombopag) for the Treatment of Thrombocytopenia in Pediatric Patients One Year and Older with Persistent or Chronic Immune Thrombocytopenia (ITP).” Press release. Sobi. July 25
2. “Sobi announces FDA acceptance of new drug application for avatrombopag (DOPTELET®) for the treatment of pediatric immune thrombocytopenia. Press release.” Sobi. December 12, 2024.

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