Aptevo Therapeutics Doses First Patient in IXINITY Clinical Trial for Potential Pediatric Label Expansion
Clinical Data Intended to be Used to Support Pediatric Label Expansion for IXINITY
Approximately 1/3 of
"Initiation of the clinical study of IXINITY in younger patients is the next step expanding treatment options for patients with hemophilia B," said
A subset of clinical data from the Phase 3 program to support the registration of IXINITY in the
The current Phase 4 clinical trial is expected to enroll up to 22 patients to evaluate the safety, efficacy, pharmacokinetics and immunogenicity of IXINITY over a period of at least 50 exposure days in patients with hemophilia B who are younger than 12 years of age.
"We are very encouraged by the pediatric subset data from the Phase 3 IXINITY program and are excited to begin this latest study of IXINITY," continued
About Hemophilia B
Hemophilia B is a congenital bleeding disorder caused by a deficiency of coagulation factor IX. It affects approximately 1:25,000 male births, with approximately 4,000 persons affected in the
IXINITY is indicated for the control and prevention of bleeding episodes and for perioperative management for adults and children ≥12 years of age with hemophilia B. IXINITY is not indicated for induction of immune tolerance in patients with hemophilia B. IXINITY contains recombinant coagulation factor IX (trenonacog alfa). Trenonacog alfa is a purified single chain glycoprotein derived from Chinese hamster ovary (CHO) cells and has an amino acid sequence that is comparable to the Thr148 allelic form of plasma-derived factor IX. No human or animal proteins are added during any stage of manufacturing or formulation of IXINITY. The recombinant factor IX is purified by a chromatography purification process. The process includes three validated steps for virus inactivation and removal. The process also includes a validated manufacturing step to reduce the presence of CHO proteins in the final drug product.
Indications and Important Risk Information
IXINITY [Coagulation Factor IX (Recombinant)] Lyophilized Powder for Solution for Intravenous Injection is a coagulation factor IX (recombinant) indicated in adults and children ≥ 12 years of age with hemophilia B for control and prevention of bleeding episodes, and for perioperative management. IXINITY is not indicated for induction of immune tolerance in patients with hemophilia B. IXINITY is contraindicated in patients who have known hypersensitivity to IXINITY or its excipients, including hamster protein.
Hypersensitivity reactions, including anaphylaxis, may occur following IXINITY administration. Discontinue use of IXINITY if hypersensitivity symptoms occur and initiate appropriate treatment. Regularly evaluate patients for the development of factor IX inhibitors by appropriate clinical observations and laboratory tests. If expected factor IX activity plasma levels are not attained, or, if bleeding is not controlled as expected with a certain dose, perform an assay that measures factor IX inhibitor concentration. An association between the occurrence of a factor IX inhibitor and allergic reactions has been reported. Individuals with factor IX inhibitors may be at increased risk of severe hypersensitivity reactions or anaphylaxis if re-challenged.
Nephrotic syndrome may occur with IXINITY. Nephrotic syndrome has been reported following attempted immune tolerance induction in hemophilia B patients with factor IX inhibitors and a history of allergic reactions. Thromboembolism may occur when using IXINITY (e.g., pulmonary embolism, venous thrombosis, and arterial thrombosis). Patients may develop hypersensitivity to hamster (CHO) protein as IXINITY contains trace amounts. The most common adverse drug reaction observed in >2% of patients in clinical trials was headache.
Please see full Prescribing Information at www.IXINITY.com.
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Senior Director, Investor Relations and Corporate Communications