BHR Pharma, LLC announced today the European Medicines Agency (EMA), responsible for evaluating medicines developed for use in the European Union, granted an orphan medicinal product designation for treating moderate and severe traumatic brain injury (TBI) to its proprietary intravenous progesterone infusion agent, BHR-100. The agent currently is being evaluated as a neuroprotective treatment for severe traumatic brain injury (TBI) in the global, Phase III SyNAPSe® clinical trial.
BHR Pharma is sponsoring SyNAPSe with the intent to bring the first-ever approved TBI treatment to market. The trial currently has 154 participating sites, including U.S. Level 1 and 2 trauma centers, in 21 countries on four continents.
“Traumatic brain injury or TBI is a global problem that needs a global solution, and with providing an orphan drug designation for BHR-100 in Europe, the EMA acknowledges the importance of supporting the development of this potential treatment in TBI,” said Roland Gerritsen van der Hoop, BHR Pharma Chief Medical Officer and Head of Global R&D. “BHR is committed to bringing this first-of-its-kind treatment to market with the hope of demonstrating progress in a condition with no approved therapies.”
To qualify for orphan designation in Europe, a medicine must meet the following criteria:
- The new medicine must be intended for the treatment, prevention or diagnosis of a disease that is life threatening or chronically debilitating;
- The prevalence of the disease in the EU most not be more than five of every 10,000 people;
- The new medicine must be of significant potential benefit to those affected by the disease.
In 2009, the U.S. Food and Drug Administration granted BHR-100 an Orphan Drug designation also for treating moderate and severe TBI and placed the drug on Fast Track status designed to accelerate its potential approval.
BHR initiated the SyNAPSe trial in June 2010 with a total enrollment target of 1,180 severe TBI patients. The trial is now more than 75 percent enrolled. Last month, the SyNAPSe trial's Independent Data and Safety Monitoring Board (DSMB) released its formal interim analysis, concluding no safety issues exist, there was no reason to stop the study for futility and the trial may continue towards its intended completion. Patient enrollment is set to complete in 2013.
About Traumatic Brain Injury (TBI)
TBI is a serious public health problem that affects millions of people worldwide. There are approximately 66,000 deaths attributed annually to TBI in Europe.(1) According to the World Health Organization, traumatic brain injury is the leading cause of death and disability in children and young adults worldwide and is involved in nearly half of all trauma deaths. Despite significant efforts and more than 75 clinical trials over the past 20 years, there is still no approved treatment for TBI.
About BHR Pharma
Founded in January 2008, BHR Pharma, LLC (www.bhr-pharma.com) is a pharmaceutical research and development company located near Washington, DC, USA. BHR is committed to bringing to market specialty treatments that employ non-oral delivery systems, with an emphasis on unmet and underserved medical needs. The company is a wholly owned subsidiary of Besins Healthcare SA (www.besins-healthcare.com), which markets healthcare products in 93 countries. ) Socin et al. JAMA 273:22 (1995).