SAN DIEGO, Dec. 3, 2016 /PRNewswire/
-- Global biotherapeutics leader CSL
Behring today announced new results from its Phase III clinical development program evaluating
IDELVION® [Coagulation Factor IX (Recombinant), Albumin Fusion Protein], the company's novel, long-acting
recombinant albumin fusion protein for the treatment of hemophilia B. The results, from a pooled analysis of clinical studies
from the global PROLONG-9FP program, assessed the relationship between estimated factor IX activity levels and clinical bleeding
risk in adult hemophilia B patients treated with IDELVION using prophylaxis or on-demand (episodic) treatment. Analysis of
factor IX activity versus efficacy showed that adult patients achieving sustained factor IX activity levels above 5 or 10
percent, have approximately 80 percent lower risk of bleeding events over one year compared with patients having factor IX
activity levels below these thresholds. The findings were presented at the 58th annual meeting of the American Society
of Hematology (ASH) in San Diego, December 3-6.
"This new analysis of Phase III IDELVION clinical trials shows a strong association between high and
prolonged factor IX levels and efficacy of IDELVION in reducing bleeding risk in adult
hemophilia B patients," said John Roberts, Ph.D., Director of Clinical Pharmacology,
CSL Behring, and lead investigator. "These findings provide additional clinical rationale for targeting
and maintaining factor IX trough activity levels above 5 or 10 percent to optimize treatment outcomes, aligning with World
Federation of Hemophilia clinical guidelines for the management of hemophilia."
In March 2016, IDELVION was approved in the U.S. The
long-acting therapy is also approved in the European Union, Japan, Australia, Switzerland and Canada. In the U.S., IDELVION is indicated 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). Regulatory approvals of IDELVION were
based on results from the global PROLONG-9FP clinical development program, which included five Phase I through Phase III
open-label, multicenter studies evaluating the pharmacokinetics (PK), safety and efficacy of IDELVION in children and
adults with hemophilia B.
"CSL Behring's research presentation at ASH 2016 demonstrates our commitment to advance coagulation science and optimize
treatment outcomes for people living with hemophilia B," said Jerry Powell, M.D., Medical
Director, North America Commercial Operations, CSL Behring. "IDELVION is the only available recombinant factor IX therapy
that maintains high factor levels using 14-day dosing intervals, helping to provide excellent bleeding control for hemophilia B
patients. Furthermore, IDELVION has been shown to maintain high steady-state factor IX levels (21 percent at 7 days and 13
percent at 14 days, on 7- and 14-day dosing regimens, respectively)."
Key Study Findings
Bleeding risk reduction in relation to predicted factor IX levels in hemophilia B patients receiving
IDELVION (abstract #1411): This abstract reported on a pharmacostatistical analysis
utilizing pooled PK and efficacy data from the pivotal Phase III trials from PROLONG-9FP. A total of
478 bleeding episodes in 57 adult patients with hemophilia B (factor IX levels ≤2 percent) were evaluated.
Investigators estimated factor IX activity levels utilizing available PK data and population PK methods for patients
receiving IDELVION prophylaxis treatment (7-day, 10-day and 14-day regimens) and on-demand treatment.
The majority of bleeding episodes occurred during the on-demand phase of the studies.
The results suggest a statistically significant relationship between factor IX exposure
and bleeding risk, as measured by the time from the start of treatment to the first major bleeding event.
Patients taking IDELVION for one year who maintained cumulative factor
IX trough levels above 5 percent and above 10 percent were predicted to significantly reduce their
bleeding risk by greater than 80 percent, compared with patients having lower trough levels of factor IX activity.
Sustained cumulative factor IX activity above 2 percent did not provide significant risk reduction over
one year. Patients with daily factor IX trough levels above 5 percent and 10
percent were also predicted to reduce their everyday bleeding risk by greater than 77 percent on average.
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 the U.S. Centers for Disease Control and Prevention, the condition affects
approximately one in 25,000 male births.
About IDELVION®
IDELVION is approved in the United States, European Union, Japan, Australia, Switzerland and Canada. For more information
about IDELVION, including full prescribing information, please visit www.IDELVION.com/prescribing-information.
IDELVION Important Safety Information
IDELVION®, Coagulation Factor IX (Recombinant), Albumin Fusion Protein (rFIX-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 17,000 people with
operations in more than 30 countries. For more information about CSL Behring visit www.CSLBehring.com or follow us
at www.Twitter.com/CSLBehring.
Contact:
Greg Healy
CSL Behring
Office: 610-878-4841
Mobile: 610-906-4564
Greg.Healy@CSLBehring.com
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