Year: 2015-16

Company: BioMarin

Liaison(s): Rajeev Mahimkar

BioMarin is a biopharmaceutical company that
develops and commercializes innovative therapeutics
for rare genetic diseases. The company was established
in 1997 in San Rafael, California. BioMarin aims to
provide first-in-class or best-in-class treatments for
patients with serious unmet medical needs through
the optimization of powerful biology, the acceleration
of the drug approval process, and a strategic pipeline
development. This strategy has earned the company
the reputation of being one of the fastest and most
innovative drug development companies in the world.
Recurrent gastrointestinal bleeding (GI) due to
Acquired von Willebrand Syndrome (AVWS) is
recognized as a significant clinical complication after
the implantation of left ventricular assist devices
(LVADs). With the intent of developing a potential
therapeutic for patients implanted with LVADs, and
with recurrent GI bleeding, BioMarin is interested in
the adequacy of the current therapeutic approach, the
business opportunity within this clinical space, and
potential clinical trial concepts.
To assess the current state of AVWS in patients with
LVADs, the BioMarin TMP team conducted primary
and secondary research — physician surveys, KOL
interviews, and literature reviews. The results of this
research allowed the team to confirm that recurrent GI
bleeding due to LVADs presents an unmet medical need
and a viable business opportunity for BioMarin. The
team compiled and archived its research findings for
BioMarin. Further, the team proposed a preliminary
format for clinical trials modeled after existing trials
in this space. The report will help BioMarin make
decisions regarding the future direction of this program
and lays the groundwork for clinical study upon
the discovery of a novel therapeutic addressing this