Adynovate is an injectable factor replacement therapy developed by Shire for the treatment and prophylaxis (prevention) of bleeding in patients 12 years and older who have hemophilia type-A.

The treatment was first approved by the U.S. Food and Drug Administration (FDA) in 2015 as Adynovate and was granted a recommended marketing authorization in Europe as Adynovi in November 2017.

How Adynovate works

Adynovate is based on Advate, an artificially produced coagulation factor VIII, a protein required for blood clotting that is defective in hemophilia type-A patients.

The difference is that Adynovate is manufactured by attaching polyethylene glycol (PEG), a non-toxic and non-immunogenic polymer to Advate, allowing it to stay in the blood longer.

By miming factor VIII, Adynovate allows blood clots to form, which stops the bleeding.

Adynovate in clinical trials

The safety and pharmacokinetics (movement in the body) of Adynovate was first assessed in a Phase 1 clinical trial (NCT01599819) in 19 patients with severe hemophilia type-A. This was followed by a pivotal Phase 2/3 trial (NCT01736475) in 137 previously-treated patients with severe hemophilia type-A to evaluate the safety, effectiveness, and pharmacokinetics of the drug as a preventative or on-demand treatment.

Published results of the trials showed that the mean half-life of Adynovate was 1.4 times longer than that of Advate. The median annualized bleed rate (ABR) on prophylaxis was 1.9, with about 40 percent bleed-free patients, while 96 percent of bleeding episodes were successfully treated with one or two infusions.

A Phase 3  trial (NCT02210091) evaluated the safety, effectiveness, immunogenicity, and pharmacokinetics of Adynovate in previously treated children younger than 12 years who have hemophilia type-A. Results of the study showed that 38 percent of patients had zero bleeds, and up to 91 percent of all bleeds were successfully treated with one or two injections. No inhibitors were detected during the study, which found a significant reduction in pain as well as an increase in the children’s physical activity.

Another Phase 3 study (NCT01913405) evaluated the effectiveness and safety of the treatment in previously-treated hemophilia-type A patients undergoing surgery. Results of the trial from 15 patients demonstrate “excellent” overall effectiveness both before, during and after surgery in all cases, with no related adverse events. Following surgery, all treatments were rated as “excellent” except for one minor (dental) procedure, which was rated “good.”

The company also is investigating the safety, immunogenicity (ability to trigger an immune response), and hemostatic effectiveness (ability to stop bleeds) of Adynovate in previously untreated and minimally treated patients with severe hemophilia type-A. A Phase 3 clinical trial (NCT02615691) is recruiting 125 boys and girls 5 years and younger in 61 locations in the U.S., Europe, and Asia.

Other details

The most common adverse effects of Adynivate are headache and nausea.


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