Full Press Release Details
Novato, CA, Oct. 27, 2014 (GLOBE NEWSWIRE) -- 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
FDA Office of Orphan Products Development has granted orphan drug
designation for triheptanoin (UX007) for the treatment of glucose
transporter type-1 deficiency syndrome (Glut1 DS). Glut1 DS, also
known as De Vivo disease, is a rare and potentially severely
debilitating disease characterized by seizures, developmental
delay, and movement disorder. Ultragenyx is conducting a Phase 2
study of triheptanoin in patients with Glut1 DS.
"Glut1 deficiency syndrome is a rare condition that can severely
affect patients and their families. Ultragenyx is committed to
addressing this high unmet medical need population and has
initiated a rigorous development plan to do so. The FDA's decision
to grant triheptanoin orphan drug designation for Glut1 deficiency
syndrome is another important step in our plan to help treat
patients with this disease," said Sunil Agarwal, M.D., Chief
Medical Officer of Ultragenyx.
Orphan Drug Designation program provides orphan status to drugs
and biologics that are intended for the safe and effective
treatment, diagnosis or prevention of rare diseases/disorders that
affect fewer than 200,000 people in the U.S. Among the benefits of
orphan designation in the U.S. are seven years of market
exclusivity following FDA approval, waiver or partial payment of
application fees, and tax credits for clinical testing expenses
conducted after orphan designation is received.
About Glut1 DS and Triheptanoin
Glut1 DS is a severely debilitating disease characterized by
seizures, developmental delay, and movement disorder. Glut1 DS is
caused by a genetic defect in the transport of glucose into the
brain. Because glucose is the primary source of energy for the
brain, this disorder results in a chronic state of energy
deficiency in the brain. Studies suggest a range of 3,000 to 7,000
Glut1 DS patients in the United States. There are currently no FDA
approved treatments specific to Glut1 DS.
Triheptanoin, also known as UX007, is a purified,
pharmaceutical-grade, specially designed synthetic triglyceride
compound created via a multi-step chemical process. Triheptanoin is
metabolized to and intended to provide patients with heptanoate,
which can diffuse across the blood-brain barrier and be converted
into glucose. Heptanoate can also be further metabolized to four-
and five-carbon ketone bodies in the liver that also cross the
blood-brain-barrier and provide an additional energy source to the
brain. Heptanoate and five-carbon ketone bodies can also regenerate
new glucose in the brain, which is deficient in these patients.
Ultragenyx is conducting a Phase 2 study in the U.S. and Europe to
evaluate the potential of triheptanoin to treat Glut1 DS patients
who have failed the ketogenic diet and who continue to have
breakthrough seizures.
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
metabolic 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.