Ultragenyx Announces Positive CHMP Opinion for Mepsevii™ (vestronidase alfa) For the Treatment of Mucopolysaccharidosis VI...
June 29 2018 - 9:00AM
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that
the Committee for Medicinal Products for Human Use (CHMP), the
scientific committee of the European Medicines Agency (EMA), has
adopted a positive opinion recommending the marketing authorization
under exceptional circumstances of Mepsevii™ (vestronidase alfa)
for the treatment of non-neurological manifestations of
Mucopolysaccharidosis VII (MPS VII; Sly syndrome). Mepsevii is an
enzyme replacement therapy designed to replace the deficient
lysosomal enzyme beta-glucuronidase in patients with MPS VII, a
progressive and debilitating rare genetic disease.
The CHMP's positive opinion will now be reviewed by the European
Commission (EC), which has the authority to approve medicines for
the European Union (EU). The EC decision is expected in the third
quarter of 2018 and will apply to all 28 countries of the European
Union, Switzerland, Norway, Iceland and Liechtenstein.
“This positive CHMP opinion takes us closer to bringing Mepsevii
to patients across Europe who are living with this ultra-rare,
highly debilitating disease and currently have no approved
treatment options,” said Emil D. Kakkis, M.D., Ph.D., Chief
Executive Officer and President of Ultragenyx. “This important
regulatory milestone underscores our commitment to developing
innovative medicines for patients around the world with both rare
and even ultra-rare diseases for which the investment and
development of the science into first-ever medicines has not yet
happened.”
The CHMP considered that the totality of the data provided for
this debilitating orphan disease warranted a recommendation for
approval under exceptional circumstances. This type of
authorization can be granted for medicines that offer new or
improved treatment options for patients with no or only limited
alternatives, in cases where the company is not able to provide
comprehensive data. In the case of Mepsevii, data on the efficacy
and safety are limited due to the extreme rarity of the disease,
and the marketing authorization requires collection and submission
of further data on the clinical efficacy and safety, which will be
reviewed by the Committee.
Mepsevii was approved by the U.S. Food and Drug Administration
for the treatment of children and adults with MPS VII in November
2017.
About MPS VII
MPS 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 sulfate, chondroitin sulfate and
heparan sulfate. 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 multi-system 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).
Limitations of Use
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 I should know
about Mepsevii?
- A severe allergic reaction called anaphylaxis has
occurred with Mepsevii treatment, as early as the first
dose.
- Your doctor will monitor you closely for symptoms of an
allergic reaction while you are receiving Mepsevii and for 60
minutes after your injection.
- Your doctor will immediately discontinue the Mepsevii
infusion if you experience anaphylaxis.
- Your doctor should talk to you about the signs and symptoms of
anaphylaxis and about getting medical treatment if you have
symptoms after leaving the doctor's office or treatment
center.
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, tell your doctor about all of
your medical conditions, including if you:
- are pregnant, think you may be pregnant, or plan to become
pregnant. There is not enough experience to know if Mepsevii may
harm your unborn baby.
- are breastfeeding or plan to breastfeed. There is not enough
experience to know if Mepsevii passes into your breast milk. Talk
with your doctor about the best way to feed your baby while you
receive Mepsevii.
These are not all the possible side effects of Mepsevii. Call
your doctor for medical advice about side effects.
You may report side effects to the FDA at (800) FDA-1088
or www.fda.gov/medwatch. You may also report side effects to
Ultragenyx at 1-888-756-8657.
Please see full U.S. Prescribing Information for
additional Important Safety Information including serious side
effects.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing
to patients novel products for the treatment of rare and ultra-rare
diseases, with a focus on serious, debilitating genetic diseases.
Founded in 2010, the company has rapidly 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.
Forward-Looking Statements
Except for the historical information contained herein, the
matters set forth in this press release, including statements
relating to future regulatory interactions, the potential timing
and success of filings for regulatory approvals and potential
indications for product candidates are forward-looking statements
within the meaning of the "safe harbor" provisions of the Private
Securities Litigation Reform Act of 1995. Such forward-looking
statements involve substantial risks and uncertainties that could
cause our clinical development programs, future results,
performance or achievements to differ significantly from those
expressed or implied by the forward-looking statements. Such risks
and uncertainties include, among others, the uncertainties inherent
in the clinical drug development process, such as the regulatory
approval process, the timing of regulatory filings, and other
matters that could affect sufficiency of existing cash, cash
equivalents and short-term investments to fund operations and the
availability or commercial potential of our drug candidates.
Ultragenyx undertakes no obligation to update or revise any
forward-looking statements. For a further description of the risks
and uncertainties that could cause actual results to differ from
those expressed in these forward-looking statements, as well as
risks relating to the business of Ultragenyx in general, see
Ultragenyx's Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission on May 8, 2018, and its
subsequent periodic reports filed with the Securities and Exchange
Commission.
Contact:Ultragenyx Pharmaceutical Inc.Investors
& MediaDanielle
Keatley+1-415-475-6876
dkeatley@ultragenyx.com
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