Hyperion Therapeutics, Inc.
) recently presented multiple pipeline related data at the
International Congress of Inborn Errors of Metabolism (ICIEM) and
the satellite symposium on urea cycle disorders (UCDs) in
The data was related to the company's marketed product
Ravicti. We remind investors that Hyperion acquired worldwide
rights to Ravicti from Ucyclyd Pharma Inc., a subsidiary of
Valeant Pharmaceuticals International, Inc.
), last year for an upfront payment of $6 million. The future
payments will be based on the achievement of regulatory
milestones in indications other than UCD, sales milestones, and
mid-to-high single-digit royalties on global net sales of
Ravicti received the U.S. Food and Drug Administration (FDA)
approval in Feb 2013 for use as a nitrogen-binding agent for
chronic management in adult and pediatric UCD patients of more
than two years of age, who cannot be managed by dietary protein
restriction and/or amino acid supplementation alone. In May 2013,
Ravicti received orphan drug status in the U.S.
Hyperion emphasized on long-term data on ammonia control in
Ravicti treated pediatrics suffering from UCD.
The long-term data was based on two studies on Ravicti
comprising 49 UCD minor patients. The patients enrolled included
23 patients between the age of 2 to 5 years. Patients were
treated for 12 months with Ravicti and the observed ammonia
levels remained below the normal upper limit of 35 umol/L.
Average ammonia levels ranged between 17.2 to 24.8 umol/L.
Patients' physical growth and development were also found to be
Hyperion also presented a pooled analysis of data from two
short-term studies which showed that Ravicti was non-inferior to
its other marketed product Buphenyl. Buphenyl is approved by the
FDA to treat UCD.
Hyperion currently carries a Zacks Rank #3 (Hold). Companies
that look well-positioned include
Jazz Pharmaceuticals Public Limited Company
) with a Zacks Rank #1 (Strong Buy) and a Zacks Rank #2 (Buy),
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