WALTHAM, Mass.--(BUSINESS WIRE)-- Bioverativ Inc. (NASDAQ: BIVV), a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, today announced that the U.S. Food and Drug Administration (FDA) has approved updated labeling for ALPROLIX® Coagulation Factor IX (Recombinant), Fc Fusion Protein, the leading extended half-life therapy for the treatment of adults and children with hemophilia B. The label update, including the addition of pediatric data showing prophylactic treatment with ALPROLIX results in effective bleed protection with extended dosing intervals, further supports the long-term efficacy and safety profile of ALPROLIX. These updates are based on interim data from the Phase 3 B-YOND open-label extension trial and final data from the Phase 3 Kids B-LONG pediatric study.
ALPROLIX is a recombinant clotting factor therapy developed using Fc fusion technology to prolong circulation in the body and has been studied in more than 150 adult, adolescent, and pediatric patients over 17,000 exposure days as part of its clinical development program. The new label demonstrates additional clinical trial experience with 93 subjects treated prophylactically for more than 104 weeks.
“ALPROLIX has the most clinical trial experience of any hemophilia B extended half-life therapy on the market, and in the three years since approval, we have continued to observe low overall bleed rates, as well as low spontaneous and joint bleeds with extended prophylactic dosing across all populations,” said Rogerio Vivaldi, MD, Executive Vice President and Chief Global Therapeutics Operations Officer at Bioverativ. “The addition of the pediatric annual bleed rates, and adult and adolescent joint bleed data, to the label reflects a growing body of evidence highlighting the clinical benefit of ALPROLIX and our commitment to improving patient outcomes through extended half-life protection.”
The ALPROLIX label update is based on FDA review of results from B-YOND, an open-label, non-randomized extension study of previously-treated adults and adolescents enrolled in the Phase 3 B-LONG study and participants of Kids B-LONG, a Phase 3 study of children (<12 years of age) with severe hemophilia B. In these trials, weekly prophylactic treatment with ALPROLIX resulted in a median spontaneous annualized bleeding rate (AsBR) of zero among children and 1.04 among adults and adolescents, and a median joint annualized bleeding rate (ABR) of zero among children and 1.11 among adults and adolescents. Median overall ABRs for children, and adults and adolescents with weekly prophylactic treatment, were 1.97 and 2.95, respectively. Updated pharmacokinetic data from these studies are also included in the label.
Obstructive uropathy was also added to the label as a common adverse reaction (incidence of greater than or equal to one percent). Obstructive uropathy was reported in two subjects and the condition was resolved in both cases with hydration. Other common adverse reactions include headache and oral paresthesia (an abnormal sensation in the mouth).
About the B-YOND Extension Study
B-YOND enrolled 116 previously-treated males, including 93 participants (81%) who completed B-LONG, and 23 (100%) who completed Kids B-LONG. The primary outcome measure is development of inhibitors. Secondary endpoints include the annualized number of bleeding episodes per subject (including spontaneous joint bleeding rates), ALPROLIX exposure days per participant, ALPROLIX consumption (total IU/kg per subject per year), and the participant’s assessment of response to treatment of a bleeding episode.
ALPROLIX® Coagulation Factor IX (Recombinant), Fc Fusion Protein is a recombinant clotting factor therapy developed for hemophilia B using Fc fusion technology to prolong circulation in the body. It is engineered by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling ALPROLIX to use a naturally occurring pathway to extend the time the therapy remains in the body (half-life). While Fc fusion technology has been used for more than 15 years, Bioverativ and its collaboration partner, Swedish Orphan Biovitrum AB (publ) (Sobi) have optimized the technology and are the first companies to utilize it in the treatment of hemophilia. ALPROLIX is manufactured using a human cell line in an environment free of animal and human additives.
ALPROLIX is approved and marketed by Bioverativ for the treatment of hemophilia B in the United States, Japan and Canada. It is also approved in Australia, New Zealand, Brazil and other countries, and Bioverativ has marketing rights in these regions. It is also authorized in the European Union, Iceland, Liechtenstein, Norway and other countries, where it is marketed by Sobi.
Allergic-type hypersensitivity reactions and development of inhibitors have been observed with ALPROLIX in the treatment of hemophilia B, including in previously untreated patients. For more information, please see the full US prescribing information for ALPROLIX. Note that the indication for previously untreated patients is not included in the EU Product Information.
About Hemophilia B
Hemophilia B is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting.1 The World Federation of Hemophilia estimates that approximately 29,700 people are currently diagnosed with hemophilia B worldwide.2
People with hemophilia B may experience bleeding episodes in joints and muscles that cause pain, decreased mobility and irreversible joint damage. In the worst cases, these bleeding episodes can cause organ bleeds and life-threatening hemorrhages. Infusions of factor IX temporarily replace clotting factors necessary to resolve bleeding and, when used prophylactically, to prevent new bleeding episodes.1
Bioverativ (NASDAQ: BIVV) is a global biopharmaceutical company dedicated to transforming the lives of people with hemophilia and other rare blood disorders through world-class research, development and commercialization of innovative therapies. Launched in 2017 following separation from Biogen Inc., Bioverativ builds upon a strong heritage of scientific innovation and is committed to actively working with the blood disorders community. The company’s mission is to create progress for patients where they need it most and its hemophilia therapies when launched represented the first major advancements in hemophilia treatment in more than two decades. For more information, visit www.bioverativ.com or follow @bioverativ on Twitter.
Bioverativ Safe Harbor
This press release contains forward-looking statements, including statements about the potential benefits, safety profile, and efficacy of ALPROLIX in hemophilia B and potential treatment decisions of physicians. These forward-looking statements may be accompanied by such words as “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “potential,” “project,” “target,” “will” and other words and terms of similar meaning. You should not place undue reliance on these statements. These statements involve risks and uncertainties that could cause Bioverativ’s actual results to differ materially from those reflected in such statements, including, without limitation, risks and uncertainties associated with Bioverativ’s drug development and commercialization activities described in the Risk Factors section of Bioverativ’s filings with the Securities and Exchange Commission. These statements are based on Bioverativ’s current beliefs and expectations and speak only as of the date of this press release. Bioverativ does not undertake any obligation to publicly update any forward-looking statements.
1 World Federation of Hemophilia. About Bleeding Disorders –
Frequently Asked Questions. Available at: http://www.wfh.org/en/page.aspx?pid=637#Difference_A_B.
Accessed on: February 11, 2016.
2 World Federation of Hemophilia. Report on the Annual Global Survey 2016. Available at: http://www1.wfh.org/publications/files/pdf-1690.pdf. Accessed on: November 11, 2017.