Ixinity

Ixinity (trenonacog alfa) is a replacement therapy originally developed by Aptevo Therapeutics. The U.S. Food and Drug Administration approved the treatment in 2015 for adults and children ages 12 and older with hemophilia B.

Medexus Pharmaceuticals acquired Ixinity from Aptevo in 2020.

How does Ixinity work?

Hemophilia B is a bleeding disorder caused by the deficiency of one of the factors required for blood clotting, called factor IX (FIX). This results in delayed blood clotting.

Ixinity is a recombinant, or man-made, version of human FIX produced in Chinese hamster ovary cells. When injected into hemophilia B patients, Ixinity replenishes FIX levels and restores the normal blood clotting process.

It is used for controlling bleeding episodes in both adults and children with hemophilia B, and to manage blood loss during surgery. It is also used as a routine prophylactic treatment to reduce the frequency of bleeding episodes in adults.

Ixinity in clinical trials

A multicenter Phase 2/3 trial (NCT00768287) assessed the pharmacokinetics, or movement in the body (which includes its absorption, distribution and excretion), and the safety and efficacy of Ixinity in 77 hemophilia B patients.

Results showed that Ixinity was well tolerated. The most common side effect was headache, which only 2.6% of participants experienced. There were no reports of FIX inhibitors during treatment. Inhibitors are neutralizing antibodies that target a medicine used to treat or prevent bleeding episodes, lowering its effectiveness.

The study also found that Ixinity was effective as a prophylactic treatment. Moreover, one or two infusions of Ixinity resolved 84% of bleeds. Researchers rated its ability to control these bleeds as excellent or good.

Pooled data from two open-label studies testing Ixinity in children under 12 showed the therapy was safe and well-tolerated. In that study, which reviewed 61 bleeding episodes, six (10%) resolved with no infusions of Ixinity, and 44 (72%) resolved after one infusion. Five (8%) bleeding episodes required two infusions, and six (10%) required three to five infusions.

Researchers rated bleed control as excellent in 65% of bleeding episodes, good in 32%, and fair in 3%. The most common side effect was excessive sweating, called hyperhidrosis, while one patient experienced a fever.

Other details

Ixinity is available in bottles containing 250, 500, 1,000, 1,500, 2,000, or 3,000 international units (IU) of recombinant FIX. The maintenance dose depends on the type of bleed or surgery, bleeding severity, and how long it takes for a wound to heal. For routine prophylaxis, the dose is 40 to 70 IU/kg twice weekly.

The most common side effects associated with Ixinity are headache, dizziness, and an altered sense of taste.

Patients who have had allergic reactions to the treatment or one of its components, including hamster proteins, should avoid taking Ixinity.

Anaphylaxis, or severe life-threatening allergic reactions, may occur. Symptoms include swelling underneath the skin (angioedema), chest tightness, drop in blood pressure, fatigue, nausea, vomiting, burning sensation, restlessness, wheezing, and shortness of breath.

Patients may also develop inhibitors against treatment. These patients are at an increased risk of experiencing allergic reactions, including anaphylaxis.

Patients who have developed inhibitors or allergic reactions also have an increased risk for nephrotic syndrome, a kidney disorder that causes the body to pass too much protein in urine. Treatment with Ixinity may also increase the risk of blood clotting.

 

Last updated: Feb. 4, 2022, by Teresa Carvalho MS

 


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