Aptevo Therapeutics Launches New 3000 IU IXINITY Assay Offering Enhanced User Experience and Improved Convenience for Hemophilia B Patients
Expands Options for Patients with Potential for
Reduced Reconstitution Time and Infusion Volume
“Aptevo has a strong reputation in the Hemophilia B 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,” said
“We are excited to continue to deliver on our promise to patients with this latest initiative,” said
With its commercial efforts in the U.S. solidly on track, Aptevo is intensifying its focus on additional market expansion opportunities for IXINITY both within and outside the U.S market. Later this year Aptevo will begin a confirmatory Phase 4 study of IXINITY for potential label expansion in a pediatric setting. The study is expected to enroll approximately 20 Hemophilia B patients under the age of 12 years and will be conducted at several clinical study centers worldwide. Previously reported data showed that IXINITY appeared to be safe and well tolerated in this subject population and were comparable to the results from the overall patient population studied in the pivotal clinical trial of IXINITY.
Mr. Lamothe continued, “Approximately 33% of people treated for Hemophilia B in the U.S. are under the age of 13 years1, representing a sizeable new addressable market, should IXINITY receive a pediatric label expansion. We also continue to look for opportunities to make IXINITY available to patients around the world and are seeking opportunities to align with international distributors to expand our IXINITY footprint more broadly. I’m very excited about the momentum we are building in our IXINITY franchise and look forward to continued future growth of IXINITY in the marketplace.”
IXINITY is currently marketed exclusively in
IXINITY is an intravenous recombinant factor IX therapeutic for use in people 12 years of age or older with Hemophilia B – a hereditary bleeding disorder characterized by a deficiency of clotting factor IX in the blood, which is necessary to control bleeding. IXINITY is currently available in four assay sizes -500 IU, 1000 IU, 1500 IU and 3000 IU vials.
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 U.S. The clinical spectrum may include spontaneous or trauma-induced bleeding into joints, muscles, and soft tissues, resulting in joint damage, reduction in mobility, and severe arthritis, all of which negatively impact health-related quality of life. The primary aim of care is to prevent and treat bleeding by replacing the deficient clotting factor.
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.
Safe Harbor Statement
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, including, without limitation, statements regarding potential milestone payments, Aptevo’s outlook, financial performance or financial condition, Aptevo’s technology and related pipeline, collaboration and partnership opportunities, commercial portfolio, milestones, and any other statements containing the words “believes,” “expects,” “anticipates,” “intends,” “plans,” “forecasts,” “estimates,” “will” and similar expressions are forward-looking statements. These forward-looking statements are based on Aptevo’s current intentions, beliefs and expectations regarding future events. Aptevo cannot guarantee that any forward-looking statement will be accurate. Investors should realize that if underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could differ materially from Aptevo’s expectations. Investors are, therefore, cautioned not to place undue reliance on any forward-looking statement. Any forward-looking statement speaks only as of the date of this press release, and, except as required by law, Aptevo does not undertake to update any forward-looking statement to reflect new information, events or circumstances.
There are a number of important factors that could cause Aptevo’s actual results to differ materially from those indicated by such forward-looking statements, including a deterioration in Aptevo’s business or prospects; adverse developments in research and development; adverse developments in the U.S. or global capital markets, credit markets or economies generally; and changes in regulatory, social and political conditions. Additional risks and factors that may affect results are set forth in Aptevo’s filings with the
2Source: Global Data PharmaPoint: Hemophilia A and B Recombinant Factor Replacement Therapy – Global Drug Forecast and Market Analysis to 2026 (Publication Date:
3Sources: 2017 Annual
Senior Director, Investor Relations and Corporate Communications
Source: Aptevo Therapeutics Inc.