Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical
company focused on the development of novel products for rare and
ultra-rare diseases, today announced that the U.S. Food and Drug
Administration (FDA) has approved MEPSEVII™ (vestronidase alfa),
the first medicine approved for the treatment of children and
adults with Mucopolysaccharidosis VII (MPS VII, Sly syndrome).
MEPSEVII is an enzyme replacement therapy designed to replace the
deficient lysosomal enzyme beta-glucuronidase in MPS VII patients.
“The approval of MEPSEVII is a pivotal moment for
Ultragenyx and for patients suffering from ultra-rare genetic
diseases for which the investment and development of treatments has
not happened yet,,” said Emil D. Kakkis, M.D., Ph.D., Chief
Executive Officer and President of Ultragenyx. “Our development
program sought to create a new paradigm in study design and
endpoint evaluations to help accommodate the difficulties of
studying extremely heterogeneous ultra-rare diseases to fulfill the
promise that the science we have all invested in over many years
actually becomes something available for patients. I would like to
thank the patients and their families for their participation in
the clinical program.”
MPS VII is a 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 a multi-system tissue and organ damage. MPS VII is one
of the rarest MPS disorders, with an estimated 200 patients in the
developed world.
"I am thrilled beyond belief to see this treatment
advance after more than 40 years of work and anticipation. Thanks
to Ultragenyx for making it happen,” said William S. Sly, Chairman
Emeritus, Department of Biochemistry at St. Louis University. “I
hope that this treatment will follow the other successful examples
of enzyme therapy for LSDs and help improve the lives of
patients with this rare disease."
MEPSEVII™ (vestronidase alfa-vjbk) was evaluated by
the FDA with Priority Review, which is reserved for drugs that
offer major advances in treatment or provide a treatment where no
adequate therapy exists.
With this approval, the FDA issued a Rare Pediatric
Disease Priority Review Voucher, which confers priority review to a
subsequent drug application that would not otherwise qualify for
priority review. The rare pediatric disease review voucher program
is designed to encourage development of new drugs and biologics for
the prevention or treatment of rare pediatric diseases.
MEPSEVII will be available to patients in the U.S.
later this month. In order to support patients, Ultragenyx has
launched UltraCare™, a comprehensive support service that will
provide ongoing support to patients and caregivers. UltraCare will
help patients obtain coverage and assist with financial support for
both medication and administration of medication. Dedicated
in-house UltraCare Guides are available Monday through Friday from
9 a.m. to 8 p.m. Eastern Standard Time at 888-756-8657 to assist
patients their families.
In Europe, the European Medicines Agency (EMA) is
currently reviewing the Marketing Authorization Application (MAA)
for vestronidase alfa, and an opinion from the Committee for
Medicinal Products for Human Use (CHMP) is expected in the first
half of 2018.
Investor Conference Call
Ultragenyx will host a conference call today at 5:00 pm Eastern
Time to discuss the approval. The live and replayed webcast of the
call will be available through the company's website at
www.ultragenyx.com. To participate in the live call by phone, dial
(855) 797-6910 (USA) or (262) 912-6260 (international) and enter
the passcode 3274408. The replay of the call will be available for
one year.
INDICATION
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.
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 Prescribing Information for
additional Important Safety Information including serious side
effects.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed
to bringing to patients novel therapies 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 and investigational therapies to
address diseases for which the unmet medical need is high, the
biology for treatment is clear, and 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 regarding Ultragenyx's plans or expectations regarding
the availability of MEPSEVII, future regulatory interactions and
the potential timing and success of filings for regulatory
approvals, 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 our 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 the company in general, see Ultragenyx's Quarterly
Report on Form 10-Q filed with the SEC on November 3, 2017, and its
subsequent periodic reports filed with the SEC.
Contact Ultragenyx Pharmaceutical Inc.Investors
& MediaDanielle Keatley415-475-6876
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