Ultragenyx Announces Approval of Mepsevii™ (vestronidase alfa) in Brazil for the Treatment of Mucopolysaccharidosis VII
October 18 2018 - 8:30AM
Mepsevii, an enzyme replacement therapy, is the
first treatment approved in Latin America for this rare
genetic disease
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical
company focused on the development of novel products for serious
rare and ultra-rare genetic diseases, today announced that Brazil’s
National Health Surveillance Agency (ANVISA) has approved Mepsevii™
(vestronidase alfa) for the treatment of
Mucopolysaccharidosis VII (MPS VII; Sly syndrome) for
patients of all ages.
“The approval of Mepsevii in Brazil is an important milestone,
particularly for children with this progressive and debilitating
disorder, and also for Ultragenyx because it marks the first
regulatory clearance for this important medicine outside of the
U.S. and Europe,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive
Officer and President of Ultragenyx. “Occurring less than one year
after Mepsevii was approved in the U.S., this approval validates
our strategic plan to rapidly expand into other regions of the
world, including Latin America.”
Mepsevii was approved by the U.S. Food and Drug Administration
(FDA) in November 2017 for the treatment of pediatric and adult
patients with MPS VII. In August 2018, the European Commission (EC)
approved the Marketing Authorization Application (MAA) for Mepsevii
under exceptional circumstances for the treatment of
non-neurological manifestations of Mucopolysaccharidosis VII. With
this decision, Mepsevii is approved in all 28 EU countries,
Iceland, Liechtenstein and Norway.
About MPS VIIMPS VII is a progressive, rare,
genetic, metabolic lysosomal storage disorder (LSD) caused by the
deficiency of beta-glucuronidase, an enzyme required for the
breakdown of the glycosaminoglycans (GAGs) dermatan sulphate,
chondroitin sulphate and heparan sulphate. These complex GAG
carbohydrates are a critical component of many tissues. The
inability to properly break down GAGs leads to a progressive
accumulation in many tissues and results in multisystem tissue and
organ damage. MPS VII symptoms can include an abnormally coarsened
face, pulmonary disease, cardiovascular complications,
hepatosplenomegaly (in which the liver and spleen swell beyond
their normal size), joint stiffness, short stature, cognitive
impairment and the skeletal disease known as dysostosis
multiplex.
MPS VII is one of the rarest MPS disorders, affecting an
estimated 200 patients in the developed world.
About Mepsevii™ (vestronidase alfa)
INDICATION (IN THE U.S.)Mepsevii is indicated
in pediatric and adult patients for the treatment of
mucopolysaccharidosis VII (MPS VII, Sly syndrome). The effect of
Mepsevii on the central nervous system manifestations of MPS VII
has not been determined.
U.S. IMPORTANT SAFETY INFORMATION
What is the most important information to know about
Mepsevii?
- A severe allergic reaction called anaphylaxis has
occurred with Mepsevii treatment, as early as the first
dose.
- Symptoms of an allergic reaction will be monitored
closely while receiving Mepsevii and for 60 minutes after the
infusion.
- If anaphylaxis is experienced, the Mepsevii infusion
will be immediately discontinued.
What are the possible side effects of
Mepsevii?
- The most common side effects of Mepsevii are:
- Leakage of Mepsevii into the surrounding tissue during
infusion
- Diarrhea
- Rash
- Severe allergic reaction (anaphylaxis)
- Infusion site swelling
- Swelling around the infusion site
- Severe itching of the skin
- One patient experienced a seizure during a fever while taking
MEPSEVII.
Before receiving Mepsevii, doctors should be informed
about all of medical conditions, including if:
- One is pregnant, thinks she may be pregnant, or plans to become
pregnant. There is not enough experience to know if Mepsevii may
harm an unborn baby.
- One is breastfeeding or plans to breastfeed. There is not
enough experience to know if Mepsevii passes into breast milk.
Women should talk with their doctor about the best way to feed
their babies while receiving Mepsevii.
These are not all the possible side effects of Mepsevii. Doctors
should be contacted for medical advice about side effects.
Side effects may be reported to the FDA at (800) FDA-1088
or www.fda.gov/medwatch.
Side effects may also be reported to Ultragenyx at
1-888-756-8657.
Please see full United States Prescribing Information for
additional Important Safety Information including serious side
effects.
About Ultragenyx Pharmaceutical Inc. Ultragenyx
is a biopharmaceutical company committed to bringing to patients
novel products for the treatment of serious rare and ultra-rare
genetic diseases. The company has built a diverse portfolio of
approved therapies and product candidates aimed at addressing
diseases with high unmet medical need and clear biology for
treatment, for which there are no approved therapies.
The company is led by a management team experienced in the
development and commercialization of rare disease therapeutics.
Ultragenyx’s strategy is predicated upon time and cost-efficient
drug development, with the goal of delivering safe and effective
therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the Company's
website at www.ultragenyx.com.
Contact Ultragenyx Pharmaceutical Inc.Investors &
MediaDanielle Keatley415-475-6876
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