Year: 2014-15

Company: BioMarin

Liaison(s): Javier Femenia Thomas Lester

BioMarin Pharmaceuticals, Inc. was founded in 1997
in Novato, California to develop and commercialize
therapies for rare genetic diseases. Their strategy to
develop first-to-market therapies with an orphan
disease status has been remarkably successful and the
current product portfolio comprises five approved
products as well as multiple pre-clinical and clinical
Given the limited manufacturing capacity of any
industrial plant, the BioMarin TMP team has been
tasked with investigating strategies to increase
BioMarin’s production capacity and capability by
analyzing the feasibility of implementing a number
of changes to the manufacturing process for one of
their products. The benefits of the proposed changes
include decreasing the production time and costs
while building in-house capabilities and increasing
the available resources in BioMarin’s manufacturing
facility. The team has been assessing the technical,
regulatory, and financial issues that BioMarin must
address in order to implement these post-approval
process changes.
Historically, companies have avoided implementing
post approval process changes in order to avoid large
capital outlays and extensive regulatory investments
in additional clinical trials and other comparability
tests. However, companies might gain significantly
from the post approval process changes if they were
to make these investments. Typically, process capacity
and efficiency could be enhanced in additional to other
potential benefits. The team’s task was to investigate
and fully understand the potential risks and benefits of
a number of post approval process changes. With this
information in hand, the BioMarin Pharmaceuticals
TMP Team was expected to outline a recommended
course of action.
To accomplish the project objectives, the team
implemented two semester-phase milestones. The first
milestone was to conduct interviews to understand the
industry’s best practices with respect to manufacturing
processes. As part of this effort the team classified each
manufacturing change and identified critical regulatory
hurdles. As part of this effort the team also provided a
comprehensive risk-benefit analysis and risk mitigation
plan associated with the different manufacturing
changes. Finally, the BioMarin Pharmaceuticals
TMP Team assessed the economic feasibility, and
provided recommendations for post approval process
changes from a technical, regulatory and economical