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A Message From Your HAEA President
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Dear HAEA friends
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I wish you and
your loved ones a happy and healthy 2010!
The past few months since our last newsletter have been
filled with a whirlwind of activities and positive developments for HAE
friends!
- Over 500 HAE friends
attended the HAEA's 2009 National Patient conference in Orlando in
late October; and
- Two additional
non-steroidal medicines have been FDA approved for treating HAE
attacks! CSL Behring's Berinert ® brand of C1-inhibitor
concentrate; and Dyax's Kalbitor, a kallikrein inhibitor that will
be delivered in a medical setting by three subcutaneous injections.
Counting the
Cinryze ™ brand of C1-inhibitor concentrate which was approved last
year for preventing HAE attacks, HAE patients now have what the HAE
Association and HAE friends have been working for over the past 10
years--a choice! Namely, three therapies other than
anabolic steroids that can either prevent or treat HAE attacks.
While we are excited that our longstanding goal of
providing HAE patients with a choice of non-steroidal treatments has
been achieved, the HAE Association continues to grow and expand our
ability to help each and every HAE friend. We are steadfast in
our commitment to provide the HAE patient community with
- authoritative,
unbiased information about the latest in HAE therapies and
research, along with
- caring and individualized
assistance from one of our Patient Services Representatives who
continue to work tirelessly to assist each and every HAE friend
with any and all needs related to HAE. In addition, our Patient
Services Representatives stand ready to answer questions about (1)
getting access to and insurance reimbursement for new medications,
and (2) clinical trials for medications still coming to market.
Please be sure to check the Patient Services list below to
find the phone number for your Representative, and don't forget to
check our website often for the latest news on what the HAE Association
is doing on your behalf.
While the hard won approval of three new HAE treatments
brings us closer to the HAEA's overall goal of ensuring every HAE
friend has access to and adequate reimbursement for a treatment that
works for them, much still needs to be done. The HAEA is committed to
directing research that will ultimately cure HAE and we need your help
to make this dream a reality. If you have not already done so, please
join the HAEA's Scientific Registry. Scientists can only unlock the
mysteries that still surround HAE by having sufficient data. Your participation
in the HAEA Scientific Registry will ensure that our children and
generations to come do not have to suffer.
Warm New Year Regards to All,
Tony Castaldo
President, US
HAEA
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CSL Behring's Berinert Receives FDA Approval
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The Berinert brand of C1 Inhibitor - a
medicine
that boasts a 30-year track
record of
remarkable safety and
effectiveness was approved by the
FDA on October 9, 2009 and is now
available to treat acute attacks of HAE.
In a press release announcing the Berinert approval,
CSL Behring's Vice President and General Manager of U.S.
Commercial Operations Robert Lefebvre said, "The FDA approval of
Berinert marks an important milestone in CSL Behring's ongoing
commitment to satisfying the unmet needs of patients with rare and
serious disorders, such as hereditary angioedema, As a leader in
developing safe, effective and high-quality therapies, we are pleased
to add to our rapidly growing portfolio a proven treatment that can
make a positive difference in the lives of HAE patients and their
families."
Please contact an HAE Association Patient Services
Representative (see list below) for more information about this
exciting new option for treating HAE attacks.
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Dyax's Kalbitor Receives FDA Approval
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 Dyax Corp.
announced on Dec.1,
2009
that the FDA granted approval
for KALBITOR® (ecallantide) for the
treatment of acute attacks
of hereditary angioedema (HAE)
in patients16 years of age and older.
In a press release announcing KALBITOR's approval, Dyax CEO Gustav
Christenson said, "The approval of KALBITOR represents an
important milestone in our ongoing commitment to the HAE
community. Furthermore, bringing KALBITOR to market validates our
mission to discover, develop, and commercialize innovative
biopharmaceuticals for unmet medical needs."
The press release also noted that as part of product approval, Dyax
has, together with the FDA, established a Risk Evaluation and
Mitigation Strategy (REMS) program to communicate the risk of
anaphylaxis and the importance of distinguishing between a
hypersensitivity reaction and HAE attack symptoms.
Please contact an HAE Association Patient Services Representative (see
list below) for more information about this exciting new option for
treating HAE attacks.
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US HAEA 2009 National Patient Conference Highlights
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Nearly 500 members of the HAE
community streamed into
beautiful
Orlando, Florida to attend the 2009
National Patient Conference, and
celebrate the HAEA's 10th
Anniversary.
Below are highlights from the conference:
Opening Remarks
by HAEA President Anthony Castaldo
Tony opened up
the conference by emphasizing that the availability of C1 inhibitor
concentrate-the worldwide gold standard HAE treatment--provides an
opportunity for every HAE patient to lead a normal life. He went on to
say, "HAE-related suffering, disability, and death from throat
swelling will become a relic of the past if we continue to unite as a
patient community. "
Tony also said, "The HAE Association is steadfast in demanding
that physicians and insurance companies recognize that HAE patients
have a fundamental right to choose a treatment program that best fits
their individual needs. We now have a variety of products from which to
choose, and we are optimistic that additional HAE treatments are going
to be approved. The HAEA's Patient Services group is dedicated to
continuing and expanding its efforts to provide each and every HAE
friend and their physicians with all of the information necessary to
establish a treatment program to control and hopefully eliminate HAE
attacks."
In closing his remarks, Tony reiterated that the HAEA's philosophy on
our 10th Anniversary is identical to that articulated on our 1st : The
HAEA is a patient services and research organization run by patients.
He went on to say, "Our day-to-day interactions with HAE
friends provides a chance for us to demonstrate the HAEA's "culture"
of caring and kindness, and absolute devotion to doing whatever humanly
possible to help every HAE friend lead a normal life. Tony then invited
everyone to enjoy the weekend and feel the love!
The Conference Keynote Presentation "HAE - A Quickly
Changing Landscape," was delivered by Dr. Bruce Zuraw, Professor
of Medicine in Residence at the University of California, San Diego
School of Medicine
The
Conference Keynote Presentation "HAE - A Quickly Changing
Landscape," was delivered by Dr. Bruce Zuraw, Professor of
Medicine in Residence at the University of California, San Diego School
of Medicine; and Chairman of the HAE Association's Medical Advisory
Board
Professor Zuraw provided conference attendees with a fascinating
history of HAE from its first mention in the medical literature by Dr.
William Osler in 1888 to today where five different companies are
heavily involved in either producing or researching HAE treatments.
The presentation also provided patients with an excellent
overview of what is happening in our bodies when we have an HAE attack
and some preliminary findings from two studies conducted by Professor
Zuraw and the HAEA: 1. the safety and effectiveness of home infusion
with C1 inhibitor concentrate and 2. HAE patient views on the safety
and effectiveness of anabolic steroids (731 patients participated). In
a nutshell, Professor Zuraw noted that the studies provide compelling
data showing that (1) home infusion with C1 inhibitor is safe,
effective, and leads to a better quality of life, and (2) anabolic
steroids are effective, but many patients are looking to discontinue
the therapy because androgen side effects outweigh the drug's benefits,
and non-steroidal therapies are now available.
Professor Zuraw established an important goal for the future-ensuring
that HAE becomes an incidental issue instead of the dominant issue in
the lives of affected patients. This will be accomplished, according to
the Professor, by an HAE Association led effort to improve diagnosis
and patient access to knowledgeable physicians. Professor Zuraw ended
his talk by discussing the importance of the HAE Association's
Scientific Registry. The Professor said that the quickest path to
curing HAE is getting each and every HAE friend to join and continue
participating in the HAEA Registry.
Representatives
from each of the five drug companies provided updates on the status of
their US clinical programs
ViroPharma's C1
inhibitor (Cinrzye) was approved last year by the FDA
as an attack prevention therapy. ViroPharma has put together an
infrastructure, Cinryze Solutions, to make their product available to
every HAE friend that could benefit from the medicine. The HAE
Association is ready to (1) provide you with a direct referral into the
Cinryze Solutions program, and (2) act as your advocate and confidant
and help you work with your doctor, the specialty pharmacy who will
ship your medicine, drug company representatives, and insurance
providers. Please contact the HAEA's Patient Services group for more
information-- Donna Davis donna-davis@haea.org or
Michelle Williamson michellewilliamson@haea.org .
CSL Behring (Berinert brand of C1 inhibitor) has received FDA
approval for their C1 inhibitor infusion for treating acute HAE attacks
(attacks that have begun) on October 12, 2009. CSL has put
together an infrastructure, B.E.N., to make their product available to
every HAE friend that could benefit from the medicine. The HAE
Association is ready to (1) provide you with a direct referral into the
B.E.N. program, and (2) act as your advocate and confidant and
help you work with your doctor, the specialty pharmacy who will ship
your medicine, drug company representatives, and insurance providers.
Please contact the HAEA's Patient Services group for more information--
Donna Davis donna-davis@haea.org or Michelle
Williamson michellewilliamson@haea.org.
Dyax
(Ecallantide) Ecallantide is a recombinant product that
inhibits kallikrein - an enzyme that plays a key role in the biochemical
processes that lead to HAE-related swelling. The company filed for FDA
licensure in September 2008 and the product was approved in early
December 2009.
Shire (Firazyr) Firazyr is an acute attack therapy
that is administered by a subcutaneous injection (under the skin
injection similar to how diabetics take insulin). The product has
already been approved in Europe, and Shire is currently conducting a
clinical trial in the US. If you would like more information
about participating in this clinical trial, please contact the HAEA's
Patient Services group for more information-- Donna Davis donna-davis@haea.org
Pharming NV (recombinant C1 inhibitor) cited successful
clinical trial results for their product, Rhucin-a recombinant human C1
inhibitor protein that is derived from the milk of genetically altered
rabbits. Clinical trials have shown that Rhucin is a safe and effective
treatment for acute HAE attacks. The company is still in the process of
applying for a US license for their product.
HAE International Henrik Boysen, from Denmark,
is an HAE patient as well as the newly appointed Executive Director of
HAE International - an association that serves HAE organizations around
the world. HAE President Anthony Castaldo serves as the President
of HAEI. During lunch on Saturday, Henrik filled us in on initiatives
of the new HAEI which include an updated website that, among other
things, will provide patients who travel abroad with information on
hospitals and physicians who treat HAE in the country to be visited.
US HAE Association Scientific Registry Vice
President Janet Long presented a brief overview of the US HAEA's
Scientific Registry. Dr. Zuraw and the HAEA, working together, designed
a state-of-the-art registry that will produce high quality
"scientific grade" data. The Registry was launched on August
12, 2009 - to promote new research in HAE, to study HAE genetics, and
to increase understanding of the disease - ultimately finding a
cure! Conference attendees were given the opportunity to
join the
Scientific Registry right at the Hotel - by the end of the weekend, 153
new members had signed up, had their blood drawn and submitted DNA
samples!
[If you are interested in participating in this great project, please
go to www.haea.org and simply
click on the Scientific Registry icon.]
Physician Question and Answer Panel US
HAEA Medical Advisor Professor Bruce Zuraw and HAE specialists- Dr. Henry
Li from Wheaton, MD, Dr. Marc Riedl from UCLA and Dr. Leonard Bielory
from Springfield, NJ fielded a wide variety of questions from the
patient community. There is not enough space to post the detailed
questions and answers in this summary. However, the HAE Association is
happy to field any and all questions from each and every member of our
community, and get definitive answers from world class HAE
scientists/physicians. HAE friends can contact their Patient Services
Representative or submit a question through our website at this link: http://www.haea.org/contactus.php
The US HAE Association 2009-2010
Sunday's patients-only session presented on-going programs and plans
that will benefit all HAE patients. The programs discussed
included:
* Celebrating all of
the accomplishments of the US HAE on its 10 year
anniversary.
* Expanding the
HAEA's Patient Services- we are growing to ensure we are well
positioned to serve as the primary problem solver for HAE friends who
are interested in exploring new therapies, or have any HAE-related
issues or needs.
* Rolling out and
recruiting patients for our HAE Scientific Registry.
* Implementing a
robust research infrastructure that includes offering medical research
grants aimed at bettering the lives of HAE patients.
* Conducting
regional meetings, workshops, and lectures for patients and physicians
interested in learning more about HAE.
* Maintaining our
24/7 toll free number to help patients deal with HAE issues that arise.
* Continuously
upgrading our web page to ensure it remains the most authoritative
source on the web for HAE-related information.
Conference attendees were separated into groups by region to meet one
another as well as touch base with their respective Patient Services
Representative.
The Sunday session closed with a video featuring our HAE children
attendees!
This conference was our first to provide a separate program for our HAE
children and youth - art, games, a carnival time and a separate movie
night while the grownups were entertained by the amazing psychic Robert
Channing.
Your US HAE Association continues to be your authoritative source for
HAE information and resources. We continue to send out
newsletters with updates on the status of all HAE medicines - approved
and awaiting approval. The Newsletter will also keep you informed on
Association news and events. The best way to be certain that you are
always up-to-date on new HAE treatments and everything the US HAEA is
doing on your behalf is to make sure that you and all of your affected
family members have signed up as HAE Association members http://www.haea.org/hae_form.php
Remember its free! So, please, if you have not already done so, fill
out the membership form! And keep in mind, we go to great lengths
to ensure your personal information is secure and protected. Access to
information is strictly limited and tightly controlled. We have never
and will never disclose any personally identifiable information to an
outside party.
We
were glad to see all of our HAE friends who helped us celebrate 10
incredible years of the US HAEA. We look forward to seeing
everyone at our next Conference!
Tony
Castaldo, President US HAEA
Janet Long, Vice President
Donna K. Davis, Director of Patient Services and Clinical Programs
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In This Issue
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A
Message
Highlights
from the
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US HAEA Scientific Registry - Join Today!
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The US HAEA Scientific Registry is making great gains,
As noted
above, the Registry gained over 150
new registrants at our Orlando conference. If you
check
our website counter, you will see that we now have almost 375
registrants total.
We are
looking for every person who has been diagnosed with
HAE and all those who are a blood relative of someone with HAE to join
the Registry. So if you have not already, please go to www.haea.org and click on the
Scientific Registry icon!
Encourage your
family members to register too!
What does it mean
to be a member of the Scientific Registry? It means you will
donate a small blood sample and a cheek swab containing your
DNA. These samples are stored in a biorepository in a completely
secure manner - there will be no identifying
information that could connect you to these
samples. Researchers will ask the HAEA for permission to use some
of the collected samples to better understand HAE and, eventually, find
a cure!
You will also be required to answer some online
questionnaires over a five year period. This data will also help
us to discover answers to all the questions we have about how HAE
affects our lives.
So please sign up now and be part of this great
undertaking to improve the life of every HAE patient!
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Meet your HAE Patient Advocates
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 Jenny
Barnes - Patient Services Representative
Jenny is a
native of Virginia who resides in North
She joined the US
HAEA in 2001 after her then five year
old
son, James recieved an HAE (de novo
Jenny dove right
into her HAEA work, becoming the moderator of the Associations's
Listserv online. Jenny's past experience working with non-profit
organizations and social service organizations has provided her with
valuable insights into how to best help HAE patients.
Jenny is a
Patient Service Representative for Region 5 (see below). Day
in and day out, she strives to do whatever possible to make a
difference in the lives of HAE friends- providing information on new
HAE treatment options, to answer questions, and deal
with HAE's inevitable challenges.
Jenny's son Jim
(James) Bain passed away from laryngeal swelling at the age of 19 on
June 6, 2008, and Jenny is passionately devoted to making
sure this tragedy will never be repeated in another HAE family.
John Williamson -
Patient Services/Clinical
A native of
Texas, John became involved with the
HAE
Association nearly a decade ago as his family
shared information they found on the original
Yahoo! chat rooms and message boards. An
early HAE trial provide both
John and
his mother access to medicine they would not have known
existed if not for the HAE Association.
As a boy, John
watched family members suffer with severe HAE attacks and how the
attacks affected quality of life. John's first clearly identified
HAE attack occurred at the age of 11 and with a laryngeal swelling at
age16; his attacks increased in frequency and severity from there.
Access to HAE clinical trials, however, helped John lead a fairly
normal life.
John is grateful
that trials were available to him through the HAE
Association, and now works every day to bring
information on the clinical trials and new treatment options available
to every HAE patient.
John is a Patient
Service Representative for Region 3 (see below) and
works tirelessly every day with our patient community to
provide guidance, explore treatment options, and offer
information on HAE clinical trials still available.
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Your Patient Services Representatives by Region
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(Supervise All
Regions)
Region -1 Michelle Williamson, Assist. Director Of
Patient Service and Clinical Programs
(972) 814-5205
michellewilliamson@haea.org
(Region 1 includes - Alaska, Colorado, Washington DC,
Hawaii, Illinois, Kansas, Kentucky, Missouri, Nebraska, New Mexico and
Nevada)
Region - 2 Lois Perry, Patient Services Representative
(559) 259-0572
loisperry@haea.org
(Region 2 includes - California, Iowa, Indiana, Maine,
Mississippi, Montana, North Dakota, New Hampshire, Oregon, Tennessee,
Virginia, Vermont and West Virginia)
Regions - 3 John Williamson, Patient Services
Representative
(972) 984-0621
john@haea.org
(Region 3 Includes - Arizona, Idaho, Ohio, Pennsylvania,
Rhode Island, South Carolina, Texas, Utah and Wyoming)
Region - 4 Sally Urbaniak, Patient Services Representative
(904) 826-6700
surbaniak@haea.org
(Region 4 includes - Connecticut, Delaware, Florida,
Massachusetts, New Jersey, New York, and Washington)
Region - 5 Jenny Barnes, Patient Services Representative
(252) 585-0763
jennybarnes@haea.org
(Region 5 includes - Alabama, Arkansas, Georgia,
Louisiana, Maryland, Michigan, Minnesota, North Carolina, Oklahoma,
South Dakota and Wisconsin)
********************************************
For Clinical Trial Referrals, Contact Patient Services/Clinical
Programs Representative, John Williamson
(972) 984-0621
john@haea.org
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The
US HAE Association publishes its
newsletters to ensure everyone in our HAE family is informed about
important clinical developments and all HAE Association
activities.
We hope you have
enjoyed this issue!
The United States
Hereditary Angioedema Association
500 Ala Moana
Blvd., Suite 400
Toll free
phone: (866) 798-5598
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