Hypophosphatasia (HPP) was long-considered a bone disease that was almost universally fatal for infants; but something older patients can survive and even grow out of. Upon further examination, literature and case studies revealed that malformed bone was simply the tip of the iceberg; that, in fact, HPP affects multiple body systems throughout the course of a lifetime.

Shifting perception from being a fatal infantile disease to a progressively devastating and lifelong enzyme deficiency, coupled with management guidelines and the promise of a first-ever treatment, drove the need for physicians to suspect, diagnose, and ultimately treat HPP.

For the past 80 years, physicians and patients believed Morquio A (mucopolysaccharidosis IVA) was primarily a skeletal condition. But recent research has shown that the disease resulting from a deficiency of a critical enzyme—causes severe, progressive damage to multiple organ systems.

In anticipation of the launch of VIMIZIM® (elosulfase alfa), BioMarin quickly began to establish a new paradigm in disease perception, through a unique mix of innovative communication strategies and tactics that engaged, intrigued, and motivated.

The launch of VIMIZIM® (elosulfase alfa) is the most successful in BioMarin history.

Cambridge BioMarketing (CB) is a unique and purpose-driven agency—its mission is focused on the rare disease community. As traditionally big pharma agencies and healthcare agency networks move into the rare space, CB realized a need for a bold brand refresh and philanthropic self-promotion.

The brand refresh elevates and clearly states CB’s position as the “rare” agency. Not only rare in its commitment but also as a unique place to work, as evidenced by the “No assholes” policy.

Every Rare Disease Day, CB creates an awareness “event.” Two years ago, that event was wrapping a MBTA Red Line train in zebra stripes, as rare diseases are referred to as zebras in the medical community and created the #SupportTheSearch social media campaign. The campaign generated thousands of posts and thousands of dollars in donations as well as garnering notoriety and good press for the agency.

Sarepta stands at the ready; waiting to introduce the world to the potential of first-in-class RNA based therapeutics. This once-in-a-generation breakthrough comes in the form of eteplirsen, the first exon-skipping therapy, and truly the first-ever disease-affecting therapy, for Duchenne muscular dystrophy (DMD).

In developing pitch strategy and creative, we keyed in on the insight, “The DMD caregiver community is led by intelligent, passionate, vested, and extremely powerful moms.  Their power is drawn from an unfathomable desperation and primal need to protect their sons.” 

Positioning: For moms and boys fighting to survive DMD, Sarepta's Brand X (Fenox), is a best-in-class RNA-based exon-skipping agent that not only slows the progression of DMD, but is their chance to regain hope for the future—for this community of boys, for the first-time ever, it's their time to grow up. 

Inconsistent communication related to MPS disease awareness and education plagues the global market. Some individual markets focus their education efforts on generalizing MPS symptomatology while others choose to educate solely on clinical manifestations of specific subtypes.

 MPSReference serves a twofold purpose: first, to streamline mucopolysaccharidosis (MPS) education across specialists and global regions to help ensure patients across the globe are receiving the best care possible and, second, to help position BioMarin as a leader within the MPS community.

MPSReference is a single dynamic web property made to educate physicians around the globe, to be a resource for all things MPS. Dynamic content is served up based on how visitors self-identify. This is truly a unique site in the rare disease space.