Based on feedback from the hemophilia B community, Aptevo Therapeutics announced that a 3,000 international unit (IU) vial assay of Ixinity is now available for patients preferring the convenience of a larger assay over the smaller IUs on the market for this intravenous recombinant factor IX.
The company also announced plans to open a Phase 4 study of Ixinity in children this year in the hope of bringing the treatment to pediatric patients. Ixinity is now approved in the U.S. for hemophilia B patients ages 12 and older to manage or prevent bleeding bouts, and before or during surgery.
Aptevo announced plans for a larger vial last fall. The therapy is available in three other assay sizes: 500 IU, 1,000 IU and 1,500 IU vials. The larger assays require fewer vials for dosing, meaning more convenience for storage and travel.
Depending on the patient’s dose, larger sizes also need less time to reconstitute for IV infusion. And, they lower overall infusion volume — a point that Aptevo saw as a key patient benefit, especially for those with severe hemophilia who typically require higher doses.
“Aptevo has a strong reputation in the Hemophilia community for listening to and responding to the needs of the community, and we’re pleased to once again deliver on our commitment to patients,” Mike Adelman, a company senior vice president, commercial operations, said in a press release.
Last year, in response to community and partner feedback, the company released a markedly smaller Ixinity package size.
Hemophilia B is caused by a deficiency in clotting factor IX. Ixinity contains recombinant coagulation factor IX, and is used to replace the clotting factor.
The planned global Phase 4 trial will enroll about 20 hemophilia B patients under 12 years old. Roughly 33 percent of hemophilia B patients in the United States are under age 13, the company said.
Previously released results show that Ixinity is safe and well-tolerated in younger children, with outcomes comparable to those of the general patient population evaluated in the pivotal Phase 2/3 clinical trial (NCT00768287) of Ixinity, which was open to patients as young as 5.
An open-label Phase 3 study (NCT01271868) in 11 hemophilia B pediatric patients, up to age 12, evaluated Ixinity’s pharmacokinetics (properties), safety and efficacy. Its findings suggested that the therapy was safe and well-tolerated, and may be an effective treatment for this age group.