20:51 , Sep 29, 2017 |  BioCentury  |  Regulation

Beyond compassion

Patient advocates, with support from FDA , are honing new arguments to persuade drug companies to routinely provide pre-approval access to investigational drugs. Rather than appealing to companies’ compassion, or threatening to blacken their reputations...
07:00 , Jun 9, 2016 |  BC Innovations  |  Strategy

Sanofi's post-reconstruction era

Lauren Martz, Senior Writer  Five years after taking the helm as Sanofi 's president of global R&D, Elias Zerhouni is rounding out his strategy to revamp the division and rebuild the pipeline with the appointment...
08:00 , Jan 19, 2015 |  BC Week In Review  |  Company News

Baylor Research Institute, Swedish Orphan Biovitrum deal

Swedish Orphan received non-exclusive rights to Baylor’s patents covering the treatment of systemic onset juvenile idiopathic arthritis (sJIA) using IL-1 beta antagonists. The companies declined to disclose financial terms. Swedish Orphan markets inflammation and autoimmune...
08:00 , Nov 10, 2014 |  BC Week In Review  |  Clinical News

Triheptanoin regulatory update

FDA granted Orphan Drug designation to triheptanoin from Ultragenyx to treat glucose transporter type-1 deficiency syndrome (De Vivo disease). The product is in Phase II testing for the indication in patients who failed a ketogenic...
07:00 , Sep 8, 2014 |  BC Week In Review  |  Company News

University of Queensland, Ultragenyx deal

The UniQuest Pty. Ltd. commercialization arm of the University of Queensland granted Ultragenyx exclusive, worldwide rights to IP related to the treatment of refractory epilepsy and other seizure-related and neurologic disorders with Ultragenyx's triheptanoin (...
07:00 , Jun 16, 2014 |  BC Week In Review  |  Company News

Abzena, Baylor Research Institute deal

Abzena's Antitope Ltd. subsidiary will develop a cell line for the institute's preclinical vaccine targeting dendritic cells to treat head and neck and cervical cancer. Antitope will use its Composite CHO technology, which enables the...
07:00 , Mar 17, 2014 |  BC Week In Review  |  Clinical News

Triheptanoin: Phase II started

Ultragenyx began a double-blind, placebo-controlled, international Phase II trial to evaluate oral triheptanoin given 4 times daily in up to 50 patients ages 3-17 years with glucose transporter type-1 deficiency syndrome who are not on...
08:00 , Feb 17, 2014 |  BC Week In Review  |  Clinical News

Triheptanoin: Phase II started

Ultragenyx began an open-label, international Phase II trial to evaluate triheptanoin daily for about 24 weeks in about 30 patients ages 6 months to 35 years with long-chain fatty acid oxidation disorders. The trial includes...
08:00 , Nov 25, 2013 |  BC Week In Review  |  Company News

AstraZeneca management update

AstraZeneca plc (LSE:AZN; NYSE:AZN), London, U.K.   Business: Pharmaceuticals   Hired: Yong-Jun Liu as head of research of AstraZeneca's MedImmune LLC unit, effective Jan. 6, 2014, currently VP and CSO of the Baylor Research Institute...
07:00 , Oct 7, 2013 |  BC Week In Review  |  Clinical News

Triheptanoin: Compassionate use program data

A retrospective medical record review of a compassionate use program in 20 evaluable patients with long-chain fatty acid oxidation disorders showed that triheptanoin reduced overall mean hospitalizations per year by 36% (1.26 vs. 1.94 events...