SAN RAFAEL, Calif.,
Feb. 15, 2017 /PRNewswire/ --
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that
the company will present data in four oral and seven poster
presentations at the 13th Annual
WORLDSymposium™ being held February 13-17, 2017 in San Diego, California.
"The annual WORLD Symposium is a great forum for the rare
disease community as it brings together leading industry experts,
each committed to developing and advancing treatment options for
patients," said Hank Fuchs, M.D.,
President Worldwide Research and Development at BioMarin. "We look
forward to sharing our latest research through eleven presentations
that explore both our existing therapies, as well as potential new
therapies for conditions with unmet medical needs."
Listing of Posters and Presentations Related to BioMarin
Products and Programs at the 13th Annual
WORLDSymposiumTM
Oral Presentations
Title
|
Authors
|
Preliminary findings
of a twenty-six week or longer intracerebroventricular infusion
study of BMN 250 administered once every 2 weeks in a canine model
of Mucopolysaccharidosis type IIIB
Presentation:
February 15 at 4:15-4:30 PM
|
Ellinwood NM, DVM,
PhD
|
Long-term safety and
efficacy of intracerebroventricular enzyme replacement therapy with
cerliponase alfa in children with CLN2 disease: interim results
from an ongoing multicenter, multinational extension
study
Presentation:
February 16 at 10:15-10:30 AM
|
Schulz A,
MD
|
Elosulfase alfa
treatment and changes in physical functioning and disability in
Morquio syndrome type A
Presentation:
February 16 at 11:30-11:45 AM
|
Hendriksz CJ,
MD
|
Sub-analysis of
long-term elosulfase alfa treatment outcomes in adults with Morquio
syndrome type A
Presentation:
February 16 at 1:00-1:15 PM
|
Parini R,
MD
|
Poster Presentations
CLN2
|
|
Title
|
Authors
|
Immunogenicity to
cerliponase alfa, an enzyme replacement therapy for patients with
CLN2 disease: results from a Phase 1 / 2 study
Presentation:
February 14 at 4:30-6:30 PM
Poster/Presentation: #55
|
Cherukuri A, Cahan H,
Van Tuyl A, de Hart G, Slasor P, Bray L, Henshaw J, Ajayi T, Jacoby
D, O'Neill C, Schweighardt B.
|
|
MPS
|
|
Title
|
Authors
|
Long-term galsulfase
treatment improves survival of patients with mucopolysaccharidosis
VI (MPS VI, Maroteaux-Lamy Syndrome): 15 year follow up from the
survey study
Presentation:
February 14 at 4:30-6:30 PM
Poster/Presentation: #113
|
Giugliani R, Lampe C,
Guffon N, Ketteridge D, Leao-Teles E, Jones SA, Quartel A, Harmatz
P.
|
Elosulfase alfa
treatment and changes in physical functioning and disability in
Morquio A
Presentation:
February 14 at 4:30-6:30 PM
Poster/Presentation: #140
|
Hendriksz CJ, Parini
R, AlSayed MD, Raiman J, Giugliani R, Mitchell JJ, Burton BK,
Guelbert N, Stewart F, Hughes DA, Matousek R, Hawley SM, Decker C,
Harmatz P.
|
Clinical outcomes
from a sub-analysis of adults with Morquio A in a long-term
extension study of elosulfase alfa treatment
Presentation:
February 14 at 4:30-6:30 PM
Poster/Presentation: #148
|
Hughes D, Giugliani
R, Guffon N, Jones SA, Mengel KE, Parini R, Matousek R, Jurecki E,
Quartel A.
|
Design and rationale
of ongoing observational and treatment studies for BMN 250, a novel
enzyme replacement therapy for Sanfilippo B syndrome
Presentation:
February 15 at 4:30-6:30 PM
Poster/Presentation: #308
|
Shaywitz A, Maricich
SM, Yu H, Kent S.
|
Preliminary safety
and pharmacodynamic response from a Phase 1/2 study of ICV BMN 250,
a novel enzyme replacement therapy for the treatment of Sanfilippo
B syndrome (MPS IIIB)
Presentation:
February 15 at 4:30-6:30 PM
Poster/Presentation: #LB-12
|
de Castro Lopez MJ,
Muschol N, Clearly M, Shaywitz AJ, Cahan H, Grover A, Maricich S,
Melton A, Pinkstaff J, Smith L, Couce ML.
|
Development of an
assay to measure LAMP2 in canine brain and cerebrospinal fluid
samples
Presentation:
February 15 at 4:30-6:30 PM
Poster/Presentation: #320
|
Soon RK, Tripp M,
Chandriani S, Wait J, Pinkstaff J, Russell C, Prill H, Lawrence R,
Crawford B, Ellinwood NM, Pryer N, Jesaitis L, Melton A.
|
Vimizim Indication
Vimizim (elosulfase alfa) is indicated for patients with
Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
Vimizim Important Safety Information
Life-threatening allergic reactions, known as anaphylaxis,
can occur during Vimizim (elosulfase alfa) infusions. Typical signs
of anaphylaxis include cough, rash, throat tightness, hives,
flushing, changes in skin color, low blood pressure, shortness of
breath, chest pain, and gastrointestinal symptoms such as nausea,
abdominal pain, retching, and vomiting. Contact your doctor or get
medical help right away if these symptoms occur during or after
Vimizim infusions. If you have a respiratory illness, you may be at
risk for a sudden worsening of your condition, and you may require
additional monitoring.
Vimizim is a prescription medicine. Before treatment with
Vimizim, it is important to discuss your medical history with your
doctor. Tell your doctor if you are sick or taking any medication
and if you are allergic to any medicines. Also tell your doctor if
you are pregnant, planning to become pregnant, or are a nursing
mother. Your doctor will decide if Vimizim is right for you. If you
have questions or would like more information about Vimizim,
contact your doctor.
Anaphylaxis can occur during any Vimizin infusion and up to
three hours after any infusion, and hypersensitivity reactions have
been observed as early as 30 minutes from the start of infusion but
as late as six days after infusion.
Serious and severe reactions can happen with Vimizim treatment,
including life-threatening allergic reactions (anaphylaxis), hives,
swelling, cough, shortness of breath, and flushing. You should
receive medication such as antihistamines before Vimizim infusions
to reduce the risk of reactions. If a reaction occurs, the infusion
should be slowed or stopped and you may be given additional
medication. If a severe reaction occurs, the infusion should be
stopped immediately and you will receive appropriate medical
treatment.
If you have acute febrile or respiratory illness at the time of
Vimizim infusion you may be at higher risk of life-threatening
complications from hypersensitivity reactions. If you use
supplemental oxygen or continuous positive airway pressure (CPAP)
you should have it available during your infusion in the event of a
sudden reaction, or extreme drowsiness/sleep from
antihistamines.
Spinal cord damage may occur due to the natural MPS IVA disease
process. Signs of spinal cord injury include back pain, numbness
and paralysis, and loss of bladder and bowel control. Contact your
doctor immediately if you develop any of these symptoms.
The most common side effects reported during Vimizim infusions
included fever, vomiting, headache, nausea, abdominal pain, chills,
and fatigue. These are not all of the possible side effects with
Vimizim. Talk to your doctor if you have any symptoms that bother
you or that do not go away.
Call your doctor for medical advice about side effects.
You may report side effects to FDA at 1-800-FDA-1088.
For more information, call BioMarin Patient and Physician
Support (BPPS) at 1-855-MORQUIO (1-855-667-7846).
Please see accompanying full Prescribing Information, including
important warning, or visit www.VIMIZIM.com.
Indication
NAGLAZYME® (galsulfase) is indicated for patients with
Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome).
NAGLAZYME has been shown to improve walking and stair-climbing
capacity.
Important Safety Information
Life-threatening anaphylactic reactions and severe allergic
reactions have been observed in some patients during NAGLAZYME
(galsulfase) infusions and up to 24 hours after infusion. If these
reactions occur, immediate discontinuation of NAGLAZYME is
recommended and appropriate medical treatment should be initiated,
which may include resuscitation, epinephrine, administering
additional antihistamines, antipyretics or corticosteroids. In
patients who have experienced anaphylaxis or other severe allergic
reactions during infusion with NAGLAZYME, caution should be
exercised upon rechallenge; appropriately trained personnel and
equipment for emergency resuscitation (including epinephrine)
should be available during infusions.
As with other enzyme replacement therapies, immune-mediated
reactions, including membranous glomerulonephritis have been
observed. In clinical trials, nearly all patients developed
antibodies as a result of treatment with NAGLAZYME; however, the
analysis revealed no consistent predictive relationship between
total antibody titer, neutralizing or IgE antibodies, and
infusion-associated reactions, urinary glycosaminoglycan (GAG)
levels, or endurance measures.
Caution should be exercised when administering NAGLAZYME to
patients susceptible to fluid volume overload because congestive
heart failure may result. Consider a decreased total infusion
volume and infusion rate when administering NAGLAZYME to these
patients.
Consideration to delay NAGLAZYME infusion should be given when
treating patients who present with an acute febrile or respiratory
illness. Sleep apnea is common in MPS VI patients and antihistamine
pretreatment may increase the risk of apneic episodes. Evaluation
of airway patency should be considered prior to the initiation of
treatment. Patients using supplemental oxygen or continuous
positive airway pressure (CPAP) during sleep should have these
treatments readily available during infusion in the event of an
infusion reaction, or extreme drowsiness/sleep induced by
antihistamine use.
Pretreatment with antihistamines with or without antipyretics is
recommended prior to the start of infusion to reduce the risk of
infusion reactions. If infusion reactions occur, decreasing the
infusion rate, temporarily stopping the infusion, or administering
additional antihistamines and/or antipyretics is recommended.
During infusion, serious adverse reactions included laryngeal
edema, apnea, pyrexia, urticaria, respiratory distress, angioedema,
and anaphylactoid reaction; severe adverse reactions included
urticaria, chest pain, rash, abdominal pain, dyspnea, apnea,
laryngeal edema, and conjunctivitis. The most common adverse events
(≥ 10%) observed in clinical trials in patients treated with
NAGLAZYME were rash, pain, urticaria, pyrexia, pruritus, chills,
headache, nausea, vomiting, abdominal pain and dyspnea. The most
common adverse reactions requiring interventions are
infusion-related reactions.
Spinal/cervical cord compression is a known and serious
complication that is expected to occur during the natural course of
MPS VI. Signs and symptoms of spinal/cervical cord compression
include back pain, paralysis of limbs below the level of
compression, and urinary or fecal incontinence. Patients should be
evaluated for spinal/cervical cord compression prior to initiation
of NAGLAZYME to establish a baseline and risk profile. Patients
treated with NAGLAZYME should be regularly monitored for the
development or progression of spinal/cervical cord compression and
be given appropriate clinical care.
To report SUSPECTED ADVERSE REACTIONS contact BioMarin
Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or
go to www.fda.gov/medwatch.
Please see full Prescribing
Information.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for patients with serious and
life-threatening rare and ultra-rare genetic diseases. The
company's portfolio consists of five commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.biomarin.com.
Contact:
|
|
Investors:
|
Media:
|
Traci
McCarty
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
(415)
455-7451
|
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SOURCE BioMarin Pharmaceutical Inc.