IDELVION® for Hemophilia B Now Available in the U.S.
KING OF PRUSSIA, Pa. — 21 March 2016
- In clinical trials, IDELVION maintained factor IX activity levels above 5 percent over 14 days, resulting in a median annualized spontaneous bleeding rate (AsBR) of 0.00
- IDELVION, CSL Behring’s long-acting recombinant albumin fusion protein, delivers on the company’s promise to develop and provide innovative specialty biotherapies that help people with serious medical conditions live full lives
CSL Behring announced today that IDELVION® [Coagulation Factor IX (Recombinant), Albumin Fusion Protein], its novel, long-acting recombinant albumin fusion protein for treating hemophilia B, is now available nationwide. IDELVION is the first and only factor IX therapy that delivers high-level protection with up to 14-day dosing in appropriate patients. This extended dosing interval was achieved while maintaining high levels of factor activity, above 5 percent over 14 days at the highest dose, thereby reducing the number of units needed for monthly prophylaxis therapy.
IDELVION was approved by the U.S. Food and Drug Administration (FDA) on March 4, 2016, for use in children and adults with hemophilia B for: routine prophylaxis to prevent or reduce the frequency of bleeding episodes; on-demand control and prevention of bleeding episodes; and the perioperative management of bleeding (around the time of surgery). FDA approval of IDELVION is based on results from the PROLONG-9FP clinical development program. Data from the global Phase III pivotal study were recently published in the American Society of Hematology’s publication Blood.
“IDELVION provides excellent bleeding control by maintaining factor IX activity levels above 5 percent over a prolonged period of time, resulting in a median annualized spontaneous bleeding rate of zero,” said Jerry Powell, M.D., Medical Director, North America Commercial Operations, CSL Behring. “We are proud to offer this long-acting therapy as a part of our broad portfolio of bleeding disorder products and are particularly excited about the positive impact IDELVION can have on the lives of patients with hemophilia B.”
IDELVION is available in four vial sizes: 250 IU, 500 IU, 1000 IU, and 2000 IU with low diluent volumes of 2.5 mL (250, 500, 1000 IU) or 5 mL (2000 IU). The product may be stored in the refrigerator or at room temperature (2-25°C; 36-77°F).
About Hemophilia B
Hemophilia B is a congenital bleeding disorder characterized by deficient or defective factor IX; nearly all affected patients are male. People with hemophilia B may experience prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. According to U.S. Centers for Disease Control and Prevention, the condition affects approximately one in 25,000 male births.
PROLONG-9FP includes Phase I through Phase III open-label, multicenter studies evaluating the safety and efficacy of IDELVION in children and adults (ages 1 to 61 years) with hemophilia B (factor IX levels ≤2%).The data from PROLONG-9FP showed median annualized spontaneous bleeding rates (AsBR) of zero and factor IX activity levels above 5 percent in patients using IDELVION prophylactically. According to the World Federation of Hemophilia, patients with factor IX activity levels above 5 percent (and below 50 percent) are considered to have mild hemophilia. This result was achieved for both 14-day dosing and 7-day dosing. The data for on-demand therapy showed that 94 percent of bleeds were controlled with one infusion, while 99 percent were controlled with one or two infusions. The most common adverse reaction in clinical trials was headache.
CSL Behring engineered IDELVION to extend the half-life of recombinant factor IX through fusion with recombinant albumin. CSL Behring selected recombinant albumin as its fusion partner for its coagulation factor proteins due to its long physiological half-life.
IDELVION is approved in Canada. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recently recommended granting marketing authorization for IDELVION in the European Union. Regulatory agencies in Australia, Switzerland and Japan are also currently reviewing CSL Behring’s marketing applications for IDELVION.
For more information about IDELVION, including full prescribing information, please visit http://labeling.cslbehring.com/PI/US/Idelvion/EN/Idelvion-Prescribing-Information.pdf. For more information about CSL Behring's recombinant products in development to treat hemophilia, visit http://www.cslbehring.com/products/bleeding-disorders/novel-recombinant-hemophilia-treatments.
Important Safety Information
IDELVION®, Coagulation Factor IX (Recombinant), Albumin Fusion Protein (rIX-FP), is indicated in children and adults with hemophilia B (congenital factor IX deficiency) for:
- On-demand control and prevention of bleeding episodes
- Perioperative management of bleeding
- Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
IDELVION is not indicated for induction of immune tolerance in patients with hemophilia B.
IDELVION is contraindicated in patients who have had life-threatening hypersensitivity to the product or its components, including hamster proteins.
IDELVION is for intravenous use only. IDELVION can be self-administered or administered by a caregiver with training and approval from a healthcare provider or hemophilia treatment center. Higher dose per kilogram body weight or more frequent dosing may be needed for pediatric patients.
Hypersensitivity reactions, including anaphylaxis, are possible. Advise patients who self-administer to immediately report symptoms of hypersensitivity, including angioedema, chest tightness, hypotension, generalized urticaria, wheezing, and dyspnea. If symptoms occur, discontinue IDELVION and administer appropriate treatment.
Development of neutralizing antibodies (inhibitors) to IDELVION may occur. If expected factor IX activity plasma levels are not attained or bleeding is not controlled with appropriate dose, perform an assay to measure factor IX inhibitor concentration. Factor IX activity assay results may vary with the type of activated partial thromboplastin time reagent used.
Thromboembolism (e.g., pulmonary embolism, venous thrombosis, and arterial thrombosis) can occur when using factor IX-containing products. In addition, nephrotic syndrome has been reported following immune tolerance induction in hemophilia B patients with factor IX inhibitors and allergic reactions to factor IX.
The most common adverse reaction (incidence ≥1%) reported in clinical trials was headache.
About CSL Behring
CSL Behring is a global biotherapeutics leader which is driven by its promise to save lives. Focused on serving patients’ needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.
CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL), headquartered in Melbourne, Australia, employs more than 16,000 people with operations in more than 30 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring. For more information about CSL Behring visit www.CSLBehring.com or follow us at www.Twitter.com/CSLBehring.