Ultragenyx Announces Recombinant Human Beta-Glucuronidase Biologics License Application and Marketing Authorization Applicati...
May 23 2017 - 08:30AM
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical
company focused on the development of novel products for rare and
ultra-rare diseases, today announced that a Biologics License
Application (BLA) submitted to the U.S. Food and Drug
Administration (FDA) and a Marketing Authorization Application
(MAA) submitted to the European Medicines Agency (EMA), for
recombinant human beta-glucuronidase (rhGUS, UX003), an
investigational therapy for the treatment of Mucopolysaccharidosis
VII (MPS VII, Sly syndrome) were accepted for review. The
Prescription Drug User Fee Act (PDUFA) goal date for a decision is
November 16, 2017 and an opinion from the Committee for Medicinal
Products for Human Use (CHMP) is expected in the first half of
2018. rhGUS is an enzyme replacement therapy for the
treatment of MPS VII.
The FDA granted rhGUS Priority Review status, which is available
for drugs that offer major advances in treatment or provide a
treatment where no adequate therapy exists. rhGUS was previously
granted Orphan Drug Designation by the FDA.
“We are pleased that the FDA has granted priority review to the
rhGUS BLA and are looking forward to working with both the FDA and
EMA in the coming months with the goal of bringing this potential
treatment to patients with MPS VII who currently have no other
options,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer
and President of Ultragenyx.
About MPS VII
Mucopolysaccharidosis VII (MPS VII, Sly syndrome), originally
described in 1973 by William Sly, M.D., is a rare genetic,
metabolic disorder and is one of 11 different MPS disorders. MPS
VII is 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
disease.
While its clinical manifestations are similar to MPS I and MPS
II, MPS VII is one of the rarest among the MPS disorders. MPS VII
has a wide spectrum of clinical manifestations and can present as
early as at birth in a severe form called non-immune hydrops
fetalis. There are no approved therapies for MPS VII today. The use
of enzyme replacement therapy as a potential treatment is based on
20 years of research work in murine models of the disease. Enzyme
replacement as a strategy is well established in the MPS field as
there are currently four approved enzyme replacement therapies for
other MPS disorders: MPS I (Aldurazyme®, laronidase), MPS II
(Elaprase®, idursulfase), MPS IVA (Vimizim™, elosulfase alfa), and
MPS VI (Naglazyme®, galsulfase).
About the Phase 3 Study
The Phase 3 randomized, placebo-controlled, blind-start clinical
study, conducted at four sites in the U.S., was designed to assess
the efficacy and safety of rhGUS in 12 patients between 5 and 35
years of age. Patients were randomized to one of four groups. One
cohort began rhGUS therapy immediately, while the other three
started on placebo and crossed over to rhGUS at different
predefined time points in a blinded manner. This novel trial design
generated treatment data from all 12 patients and improved the
statistical power relative to a traditional parallel-group design.
Patients were dosed with 4 mg/kg of rhGUS every other week for up
to a total of 48 weeks, and all groups received a minimum of 24
weeks of treatment with rhGUS.
The primary objective of the study was to determine the efficacy
of rhGUS as determined by the percent reduction in urinary GAG
excretion after 24 weeks of treatment. Secondary efficacy endpoints
include a multi-domain responder index and an individualized
clinical response measure, as well as other clinical outcomes
including pulmonary function, walking, shoulder flexion, fine and
gross motor function, visual acuity, and fatigue. The safety and
tolerability of rhGUS were also assessed.
Agreement has been reached with both the U.S. FDA and EMA on the
Phase 3 study design. Based on the data from the Phase 3 study, we
met with the FDA and the EMA prior to filing the BLA and MAA. The
FDA stated that their evaluation of the pivotal Phase 3 study will
be based on the totality of the data on a patient-by-patient basis.
FDA advised against the declaration of a primary clinical endpoint
in order to allow for more flexibility in the overall efficacy
evaluation, appreciating the difficulty of evaluating a single
clinical endpoint given the heterogeneity and rarity of the
disease. The EMA has agreed that approval under exceptional
circumstances could be possible based on the Phase 3 study with
urinary GAG levels as a surrogate primary endpoint, provided the
data are strongly supportive of a favorable benefit/risk ratio and
that some evidence or trend in improvement in clinical endpoints is
observed.
About Ultragenyx
Ultragenyx is a clinical-stage biopharmaceutical company
committed to bringing to market 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 product candidates with the potential 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 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, uncertainties regarding the acceptance by
the FDA or EMA of the adequacy of the clinical data in our recently
completed Phase 3 rhGUS study, and the clinical validity and
relevance of the endpoints from this study. 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 Securities and Exchange
Commission on May 5, 2017, and its subsequent periodic reports
filed with the Securities and Exchange Commission.
Contact Ultragenyx Pharmaceutical Inc.
Investors & Media
Ryan Martins
844-758-7273
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