FDA Approves Avatrombopag for Chronic Immune Thrombocytopenia

The US Food and Drug Administration (FDA) has approved avatrombopag (Doptelet) as a treatment for insufficiently-treated adult patients with chronic immune thrombocytopenia (ITP).

The supplemental New Drug Application (sNDA) submitted by Dova Pharmaceuticals for the oral therapy is its second approved indication in as many years. Last May, the FDA approved avatrombopag as a novel therapy for the treatment of thrombocytopenia in patients with chronic liver disease who are scheduled for medical or dental procedure.

The thrombopoietin receptor agonist (TPO-RA), to be administered with food, is designed to improve platelet counts in patients burdened by thrombocytopenia and its various comorbidities.

Its benefit for patients with ITP is evidenced in a pivotal phase 3 study which observed treated patients over 6 months. At 8 days of therapy, a majority of treated patients reported an improved platelet count of at least 50,000 per µL. The therapy was also significantly improved in maintaining platelet counts through 6 months of care versus placebo.

Data pooled from 1000-plus patients treated across 24 Doptelet clinical development program studies supported its safety and tolerability for the sNDA. Dove also pulled supportive efficacy data from a pair of phase 2 trials, as well as another couple of phase 3 trials.

The company is anticipating a marketing launch of Doptelet for its ITP indication in mid-July next month. In the meantime, Caroline Kruse, president and chief executive officer of the patient advocacy organization Platelet Disorder Support Association, advised that ITP patients work with their clinicians to understand what therapies would best complement their lifestyle and goals to improved ITP care.

Such a luxury of choice is uncommon in the field of thrombocytopenia.

“That’s why having additional treatment options are so important,” Kruse said in a statement. “We are thrilled to have a new, oral TPO-RA available for adult patients with ITP. Every new treatment provides more choices and new hope to our community.”