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.
About ALPROLIX®
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
About Bioverativ
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.
REFERENCES
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.
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version on businesswire.com: http://www.businesswire.com/news/home/20171127005234/en/
Bioverativ Inc.Media Contact:Marianne McMorrow, +1
781-663-4376media@bioverativ.comorInvestor Relations Contact:Susan
Altschuller, +1 781-663-4360IR@bioverativ.com
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