BC Week In Review | Jul 11, 2016
Clinical News

Glycerol phenylbutyrate regulatory update

Horizon submitted an sNDA to FDA for Ravicti glycerol phenylbutyrate for chronic management of urea cycle disorders (UCDs) in patients 2 months of age to <2 years of age who cannot be managed by dietary...
BC Week In Review | Jan 18, 2016
Clinical News

Ornithine phenylacetate: Phase I data

An open-label, crossover Phase I trial in healthy volunteers showed that 3 extended-release formulations of oral solid-dose OCR-002 led to mean maximum plasma concentrations (Cmax) of phenylacetate of 50-90 ug/mL at 4-9 hours post-dose vs....
BC Week In Review | Dec 7, 2015
Clinical News

Ravicti glycerol phenylbutyrate regulatory update

The European Commission approved Ravicti glycerol phenylbutrate from Horizon to treat urea cycle disorders (UCDs) in patients ages >=2 months. In February 2013, FDA approved the pre-prodrug of phenylacetic acid for chronic management of patients...
BioCentury | Oct 26, 2015
Emerging Company Profile

Building bacteria

Synlogic Inc. is engineering bacteria that purge the gut of toxic metabolites caused by rare inborn errors of metabolism including urea cycle disorders and phenylketonuria. The company expects its products will be the first to...
BC Week In Review | Oct 5, 2015
Clinical News

Ravicti glycerol phenylbutyrate regulatory update

EMA’s CHMP recommended approval of Ravicti glycerol phenylbutyrate from Horizon as an adjunctive therapy for chronic management of patients aged >=2 months with urea cycle disorders (UCDs). In February 2013, FDA approved the pre-prodrug of...
BC Week In Review | Sep 28, 2015
Clinical News

Ornithine phenylacetate: Phase I started

Ocera began an open-label, crossover Phase I trial to compare extended-release formulations of oral solid-dose OCR-002 vs. an immediate-release oral solution of OCR-002 and Ravicti glycerol phenylbutrate in healthy volunteers. An IV formulation of the...
BC Extra | Sep 25, 2015
Company News

CHMP recommends host of therapies

EMA's CHMP issued opinions backing marketing authorization for a basket of compounds on Friday, including recommendations for cancer products from Amgen Inc. (NASDAQ:AMGN) and the Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHHBY) as well as...
BC Week In Review | Apr 6, 2015
Company News

Hyperion Therapeutics Inc, Horizon Pharma deal

Last June, EMA accepted for review an MAA for Ravicti to treat UCD; Hyperion expects a decision from EMA’s CHMP in late 2015 or early 2016. Hyperion plans to submit an sNDA for Ravicti to...
BC Extra | Mar 31, 2015
Company News

Horizon buying Hyperion for $1.1B

Horizon Pharma plc (NASDAQ:HZNP) will acquire rare disease company Hyperion Therapeutics Inc. (NASDAQ:HPTX) for $46 per share, or $1.1 billion in cash. The price represents an 8% premium to Hyperion's Friday close of $42.74. Horizon...
BC Week In Review | Mar 9, 2015
Clinical News

Glycerol phenylbutyrate: Phase IV started

Hyperion began the open-label, U.S. Phase IV HPN-100-009 trial to evaluate oral Ravicti 3-6 times per day for 6-24 months in about 24 patients ages <2. Hyperion said the study is a postmarketing requirement. FDA...
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