Market Overview

U.S. Label Update for ALPROLIX® Adds Data Reinforcing Efficacy and Safety of Long-Term Prophylactic Treatment in Adults, Adolescents and Children with Hemophilia B


. (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

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

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


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
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:
Accessed on: February 11, 2016.
2 World Federation of
Hemophilia. Report on the Annual Global Survey 2016. Available at:
Accessed on: November 11, 2017.

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