FDA Approves Biogen Idec’s ALPROLIX™, the First Hemophilia B Therapy to Reduce Bleeding Episodes with Prophylactic Infusions

  FDA Approves Biogen Idec’s ALPROLIX™, the First Hemophilia B Therapy to
  Reduce Bleeding Episodes with Prophylactic Infusions Starting at Least a
  Week Apart

-Only Approved Treatment to Demonstrate Prolonged Clotting Factor Circulation
                                 in the Body-

                      -Approved for Adults and Children-

Business Wire

CAMBRIDGE, Mass. CAMBRIDGE, Mass. -- March 28, 2014

Today Biogen Idec (NASDAQ: BIIB) announced that the U.S. Food and Drug
Administration (FDA) has approved ALPROLIX^™ [Coagulation Factor IX
(Recombinant), Fc Fusion Protein], the first recombinant, DNA derived
hemophilia B therapy with prolonged circulation in the body. ALPROLIX is
indicated for the control and prevention of bleeding episodes, perioperative
(surgical) management and routine prophylaxis in adults and children with
hemophilia B. The therapy is shown to reduce bleeding episodes with
prophylactic (protective) infusions starting at least a week apart.

The approval of ALPROLIX is the first significant advance in hemophilia B
treatment in more than 17 years. The therapy is clinically proven to reduce
bleeding episodes with a favorable safety and tolerability profile. It is
developed using a process called Fc fusion and is the first hemophilia therapy
to demonstrate prolonged circulation in the body, which has been shown in
adults and adolescents with hemophilia to extend the time between prophylactic
infusions.

“The FDA approval of ALPROLIX is a significant milestone for the hemophilia B
community, and represents an important first step in our commitment to
transform the care of people with hemophilia,” said George A. Scangos, Ph.D.,
chief executive officer of Biogen Idec. “ALPROLIX offers people with
hemophilia B the ability to prevent or reduce bleeding episodes with
prophylactic infusions starting at least a week apart. We believe this new
therapy will help more people with hemophilia and their caregivers realize the
benefits of this treatment approach.”

The National Hemophilia Foundation (NHF) recommends routine prophylaxis as
optimal for the treatment of people with severe hemophilia. Studies show
people with severe hemophilia who follow this type of regimen experience fewer
bleeding episodes and their related risks. Complications of bleeding episodes
may range from severe swelling and pain to arthritis, joint damage and
physical disability.^1,2,3

“Hemophilia has a significant impact on people whom it affects, throughout
their lives,” said Patrick F. Fogarty, M.D.,^4 assistant professor of medicine
at the Hospital of the University of Pennsylvania, and director, Penn
Comprehensive Hemophilia and Thrombosis Program. “ALPROLIX addresses a
critical need by allowing people with hemophilia B to maintain factor levels
with prophylactic infusions once weekly or once every 10 days. We hope this
will facilitate use of prophylactic therapy.”

Hemophilia B is a rare, chronic, inherited disorder in which the ability of a
person’s blood to clot is impaired, which can lead to recurrent and extended
bleeding episodes. Therapies for hemophilia B can be administered either on a
schedule to help prevent or reduce bleeding episodes (prophylaxis), or to help
control a bleeding episode when it occurs (on-demand). According to NHF
guidelines, traditional hemophilia B therapy requires prophylactic infusions
two or more times a week.

“The hemophilia community is excited about new therapy options for the
prophylactic management of hemophilia B,” said Val Bias, chief executive
officer of the National Hemophilia Foundation. “This approval is a significant
step forward because it provides an important new option and expanded choice
for the hemophilia B community.”

The approval of ALPROLIX is based on results from the global, Phase 3 B-LONG
study, as well as interim pharmacokinetic (measurement of the presence of the
therapy in a person’s body over time) and safety data from the Phase 3 Kids
B-LONG study. B-LONG study results showed that adults and adolescents with
severe hemophilia B achieved prevention or reduction of bleeding episodes with
prophylactic infusions at least a week apart. The study included two
prophylaxis regimens – the weekly prophylaxis arm and the
individualized-interval prophylaxis arm, in which the dosing interval started
at once every 10 days. The overall median dosing interval with
individualized-interval prophylaxis was 12.5 days; during the last six months
of the study, the median interval was 13.8 days. More than 90 percent of all
bleeding episodes were controlled by a single ALPROLIX infusion.

Noparticipants in the B-LONG study developed inhibitors (neutralizing
antibodies that may interfere with the activity of the therapy) to ALPROLIX.
There were no reports of vascular clots or serious allergic reactions. Across
the routine prophylaxis and on-demand therapy arms, adverse reactions were
reported in 8.4 percent of participants. These adverse reactions included
headache, oral paresthesia (abnormal sensation in the mouth), dizziness,
dysgeusia (taste alteration), breath odor, fatigue, infusion site pain,
palpitations, obstructive uropathy (an obstructing clot in the urinary
collecting system) and hypotension (low blood pressure). Each event occurred
in two or fewer study participants.

An interim analysis from an ongoing, multi-center Phase 3 pediatric study –
Kids B-LONG –showed no inhibitors were detected, and the increase in half-life
(a measure of the time therapy remains in the body) seen with ALPROLIX was
consistent with data reported in adults and adolescents.

ALPROLIX was recently approved by Health Canada for the treatment of
hemophilia B and is currently under review by regulatory authorities in
several other countries, including Australia and Japan.

Biogen Idec plans to make ALPROLIX commercially available to people with
hemophilia B in the United States in early May. To help address the hemophilia
B community’s needs, Biogen Idec is committed to providing equitable access to
therapy, as well as a variety of personalized assistance and resources through
MyALPROLIX^™ Services. For more information on ALPROLIX, prescribing
information and financial assistance programs, please visit www.ALPROLIX.com
or www.biogenidec.com.

About the B-LONG Study

B-LONG was a global, open-label, multi-center Phase 3 study that evaluated the
efficacy, safety and pharmacokinetics of ALPROLIX in 123 males aged 12 years
and older with hemophilia B. The study involved 50 hemophilia treatment
centers in 17 countries on six continents.

The overall median annualized bleeding rates (ABR), or projected rate of
bleeding episodes per year, reported in the study were 3.0 for the weekly
prophylaxis arm, 1.4 for the individualized-interval prophylaxis arm and 17.7
for the on-demand treatment arm. For 12 study participants undergoing 14 major
surgical procedures, treating physicians rated the ability of ALPROLIX to
control bleeding as “excellent” or “good” in 100 percent of these surgeries.

Common adverse reactions (incidence of greater than or equal to 1 percent)
from the B-LONG study were headache and oral paresthesia (an abnormal
sensation in the mouth).

About ALPROLIX

ALPROLIX™ [Coagulation Factor IX (Recombinant), Fc Fusion Protein] is the
first recombinant, clotting factor therapy with prolonged circulation in the
body. It is indicated for the control and prevention of bleeding episodes,
perioperative (surgical) management and routine prophylaxis in adults and
children with hemophilia B. ALPROLIX is not indicated for immune tolerance
induction therapy, which is a treatment for people with inhibitors, and should
not be used in individuals with a known history of serious allergic reactions.
ALPROLIX was developed by fusing factor IX to the Fc portion of immunoglobulin
G subclass 1, or IgG[1] (a protein commonly found in the body). It is believed
that this enables ALPROLIX to use a naturally occurring pathway to prolong the
time the therapy remains in the body. While Fc fusion has been used for more
than 15 years, Biogen Idec is the only company to apply it in hemophilia.

Common adverse reactions (incidence of greater than or equal to 1 percent)
from the B-LONG study were headache and oral paresthesia (an abnormal
sensation in the mouth). For complete prescribing information, go to
www.ALPROLIX.com.

For members of the media interested in more information and additional
resources, please visit www.biogenidec.com/us_media_corner.

About Hemophilia B

Hemophilia B occurs in about one in 25,000 male births annually, and more
rarely in females, affecting about 4,000 people in the United States. The
World Federation of Hemophilia global survey conducted in 2012 estimates that
approximately 28,000 people are currently diagnosed with hemophilia B
worldwide. It is caused by having substantially reduced or no factor IX
activity, which is needed for normal blood clotting. People with hemophilia B
experience bleeding episodes that cause pain, irreversible joint damage and
life-threatening hemorrhages. Prophylactic infusions of factor IX temporarily
replace clotting factors necessary to control bleeding and prevent new
bleeding episodes.

About the Biogen Idec and Sobi Collaboration

Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the
development and commercialization of ALPROLIX for hemophilia B. Biogen Idec
leads development, has manufacturing rights, and has commercialization rights
in North America and all other regions in the world excluding the Sobi
territory. Sobi has the right to opt in to assume final development and
commercialization in Europe, Russia, the Middle East and Northern Africa.

AboutBiogen Idec

Through cutting-edge science and medicine,Biogen Idecdiscovers, develops and
delivers to patients worldwide innovative therapies for the treatment of
neurodegenerative diseases, hemophilia and autoimmune disorders. Founded in
1978,Biogen Idecis the world’s oldest independent biotechnology company.
Patients worldwide benefit from its leading multiple sclerosis therapies. For
product labeling, press releases and additional information about the company,
please visit www.biogenidec.com.

Biogen Idec Safe Harbor

This press release contains forward-looking statements, including statements
about the potential and therapeutic impact of ALPROLIX. 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 actual results to
differ materially from those reflected in such statements, including
uncertainty of success in our commercialization of ALPROLIX, which may be
impacted by, among other things, slower than anticipated acceptance of
ALPROLIX by patients and the medical community, intense competition in the
hemophilia market, the effectiveness of our sales force and marketing efforts,
problems with the manufacturing process for ALPROLIX, the occurrence of
adverse safety events, difficulties in obtaining or changes in the
availability of reimbursement for our products, our failure to obtain
regulatory approvals in other jurisdictions, including in the event other
companies receive marketing approval of their treatments before approval of
our treatments, our failure to protect our intellectual property and other
proprietary rights, product liability claims and the other risks and
uncertainties that are described in the Risk Factors section of our most
recent annual or quarterly report and in other reports we have filed with the
U.S. Securities and Exchange Commission (SEC). Any forward-looking statements
speak only as of the date of this press release and we assume no obligation to
update any forward-looking statements, whether as a result of new information,
future events or otherwise.

^1 Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years'
experience of prophylactic treatment in severe haemophilia A and B. J Intern
Med 1992;232:25-32.

^2 Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus
episodic treatment to prevent joint disease in boys with severe hemophilia. N
Engl J Med 2007;357:535-44.

^3 Panicker J, Warrier I, Thomas R, Lusher JM. The overall effectiveness of
prophylaxis in severe haemophilia. Haemophilia 2003;9:272-8.

^4 Editor’s Note: Dr. Fogarty has received researched support, and fees for
consulting, from Biogen Idec.

Contact:

Biogen Idec
Media Contact:
Todd Cooper, +1-781-464-3260
public.affairs@biogenidec.com
or
Investor Relations Contact:
Ben Strain, +1-781-464-2442
IR@biogenidec.com
 
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