ASH Annual Meeting and Exposition
ASH Annual Meeting and Exposition
December 23, 2019
9 min watch

VIDEO: Efgartigimod safe, effective in primary immune thrombocytopenia

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ORLANDO — A 3-week treatment cycle of efgartigimod, a neonatal Fc receptor antagonist, was well tolerated and effective in patients with primary immune thrombocytopenia, according to phase 2 data presented at the ASH Annual Meeting and Exposition.

Primary immune thrombocytopenia (ITP) is a bleeding disorder characterized by a low platelet count that is difficult to treat, according to Adrian C. Newland, CBE, FRCP, FRCPath, professor of hematology at Barts and The London School of Medicine, Queen Mary University of London.

“The basic problem in ITP is that it is predominately an autoantibody-mediated disorder,” he told Healio. “The autoantibodies help destroy platelets by opsonizing them [and] impact megakaryocytes, so they reduce platelet reduction. They also impact platelet function by attaching to the platelets.”

In this video, Newland reviews data from the phase 2 trial in which investigators randomly assigned 38 patients with IPT who were relapsed or refractory to conventional treatments to receive 5 mg of efgartigimod, 10 mg of efgartigimod or placebo. Overall, the treatment was well tolerated with no significant treatment-related adverse events. It was associated with a reduction in bleeding that occurred in conjunction with platelet increments.

An open-label extension analysis showed the 10 mg dose of efgartigimod was effective in patients who did not adequately respond to the 5 mg dose or placebo.

“Our conclusions from that was this was a treatment that worked, it was acceptable for patients, and it was something that we needed to explore further in larger numbers,” Newland said.

The researchers will continue to investigate the 10 mg dose of efgartigimod as well as a subcutaneous formulation in two larger phase 3 trials.

“There is clearly an unmet need in this particular population,” Newland said. “Although we have conventional treatments, they are not all that applicable to what is a very difficult patient group. The majority of patients with ITP are male over 60 who have other comorbidities. So, treatments that are likely to be associated with thrombosis and venous thromboembolism need to be avoided. Therefore, there is clearly an opening for further immunologically based treatments, which have a very exciting potential in this group.”


Newland AC, et al. Abstract 895. Presented at: ASH Annual Meeting and Exposition; Dec. 7-10, 2019; Orlando.

Disclosure: Newland reports serving in consultancy roles for Amgen, Angle, Argenx, Dova, Novartis, ONO, Rigel and Shionogi. He also reports receiving honoraria from Amgen, Angle, Argenx, Dova, Novartis, ONO, Rigel and Shionogi, as well as research funding from Amgen, Novartis and Rigel.