(Corrects spelling of drug name to Alprolix in paragraphs 7 and
By Deena Beasley
LOS ANGELES, March 7 For 33-year-old video
editor and hemophilia patient Travis Roop, being able to get
life-saving infusions of a blood clotting agent every two weeks,
instead of rushing to treat a bleeding episode, meant he could
jog for the first time.
Roop was enrolled in a clinical trial testing a long-acting
treatment for hemophilia B from Denmark's Novo Nordisk
. The therapy is one of several new blood clotting
agents in development that will help hemophilia patients cut by
half, or more, the number of regular intravenous infusions they
need to prevent traumatic bleeding in their daily lives.
"It was amazing actually. It was a life-changing
experience," said Roop, who lives in the Los Angeles area. "I
always avoided trauma to my left ankle ... With this, I knew it
would take care of itself."
People with hemophilia have a fault in a gene that regulates
the body's production of proteins called clotting factors. This
can cause spontaneous bleeding as well as severe bleeding
following injuries or surgery.
To avoid joint damage and other complications, patients with
severe forms of the disease need regular infusions, lasting 30
minutes or more, of relatively short acting and very expensive
clotting factors. The number of infusions and their volume
depends on the medical status of each patient and their body
Since the gene is carried on the X chromosome, hemophilia is
almost entirely a disease of men. But women can pass the gene to
their offspring. Hemophilia has often been called the "Royal
Disease" since it was carried by Britain's Queen Victoria and
affected many of the ruling families of Europe.
The U.S. Food and Drug Administration is due to decide by
mid-year whether to approve Alprolix, a new long-lasting
hemophilia B clotting factor from Biogen Idec and
partner Swedish Orphan Biovitrum. Novo Nordisk
expects to file next year for regulatory approval of its
long-acting hemophilia B drug, N9-GP.
Some industry experts say these and other new treatments
could help drive down the price of existing hemophilia products,
which can total $300,000 or more a year for a single patient.
That's because Biogen does not yet market any hemophilia
therapies. In the United States, Novo Nordisk sells a drug used
only by patients who have developed antibodies to currently-used
factors. For a factbox, click.
If new entrants to the hemophilia market decide to price
their longer-lasting factors in line with the overall price of
the existing shorter-acting products, the decision for patients
to try out the new factors will be relatively easy, said Dr Guy
Young, director of the Hemostasis and Thrombosis Center at
Children's Hospital Los Angeles.
"If they come in at a similar annual price, then companies
that are making recombinant drugs will be forced to drop their
price," said Dr Young, who has been involved in clinical trials
of long-acting factors.
$11 BILLION MARKET BY 2016
Worldwide, about one in 5,000 men is born with hemophilia A
and one in 25,000 men is born with hemophilia B each year.
The global market for hemophilia drugs, led by hemophilia A
products such as Baxter International's Advate and
Pfizer's Xyntha, totaled $8.5 billion in 2011 and will
grow to $11.4 billion by 2016, according to Morningstar.
Deutsche Bank estimates that Biogen's hemophilia products
can reach annual sales of $1.2 billion by 2017.
Biogen Chief Executive Officer George Scangos said at an
industry conference in January that if insurance companies and
other payors view the long-lasting products as simply more
convenient, they will be priced in line with existing therapies.
If they are viewed as more beneficial, prices will be higher, he
Blood factor concentrates were not developed until the
mid-20th century, and up until that time people with hemophilia
had a life expectancy of less than 30 years. The last major
advance in treatment of the rare disease came in the early 1990s
when bioengineered clotting factors replaced factors derived
from blood plasma.
Factors are numbered using Roman numerals. Hemophilia A is
caused by a lack of clotting factor VIII, while hemophilia B is
caused by a lack of clotting factor IX.
The new longer-lasting hemophilia B products can be given
every 10 days or two weeks, offering significant advantages for
patients, especially young children, who now need infusions
every two or three days, said Dr Marion Koerper, medical
director of the National Hemophilia Foundation and professor of
hematology at the University of California at San Francisco
Children's Hospital. Koerper has not been involved in clinical
trials of longer-acting factors.
"Patients don't always get the doses they are supposed to
get," she said, noting that mornings can be hectic and parents
may wait to infuse a child at night even though factor levels
should be highest when they are running around or at school.
STRONG DEMAND SEEN FOR LONG-ACTING FACTOR IX
Biogen's Alprolix extends the duration of the factor
three-fold, while Novo Nordisk's version offers a five-fold
increase, Dr Young said. That raises the stakes for Biogen to be
first to market its product.
"There is no question that when these drugs get on the
market there will be pretty massive and quick uptake," Young
said. "I predict most (hemophilia B) patients will end up on one
of these new drugs."
Roop, the video editor, said he will seek out Biogen's
therapy if it is the first to become available, but has heard
that Novo Nordisk's, which he has already tried, works better.
For hemophilia A, the advantages of the new products are
less dramatic and uptake will be slower, Dr Young said.
An FDA decision on Biogen's longer-acting hemophilia A
therapy, Eloctate, is expected mid-year.
Baxter expects to file by the end of this year for U.S.
approval of its long-lasting hemophilia A treatment. Bayer
, and CSL Behring also have long-lasting factors in
advanced stages of development. Pfizer Inc's work in the
field is still in the "discovery" stage.
Meanwhile, biotechnology companies like Sangamo BioSciences
and BioMarin Pharmaceutical are working on
methods to replace defective hemophilia genes, allowing patients
to begin producing their own clotting factor.
Biogen's Scangos acknowledged that new progress is being
made in gene therapy, particularly for hemophilia B, but
believes that a safe, effective treatment based on this method
is still at least five years away.
Roop, who has severe hemophilia B, said he was offered the
option of participating in a gene therapy trial, but decided not
to take a chance on "unproven science."
"If I can get some longer-lasting factor, maybe get down to
just once a month or once every three weeks - I would be happy
with that," he said.
Jackson Corral did not benefit during trials of the
longer-lasting treatments. The five-year-old Los Angeles area
resident, who has hemophilia A, took part in a clinical trial of
His mother, Jackie Corral, cannot wait for something to
work. She prepares and infuses Jackson and his 3-year-old
brother Jameson with a factor VIII product every other morning
and always stays within a 20 minute radius of both boys.
"If we did go to twice-a-week infusions that would be
amazing," she said. "That would give me so much more peace."
(Reporting By Deena Beasley; Editing by Michele Gershberg and