Rapidly establishing a new orphan-drug standard of care through integrated marketing


Raptor Pharmaceutical Corp. (NASDAQ: RPTP) is a biopharmaceutical company focused on developing and commercializing transformative treatments for people affected by rare and debilitating diseases.  Raptor’s first product, PROCYSBI® (cysteamine bitartrate) delayed-release capsules, received FDA approval in April 2013 for the management of nephropathic cystinosis in adults and children six years and older. It was approved in Europe one year later.

Without treatment, children live to about 9 years of age.

Nephropathic cystinosis (NC) is a rare, inherited condition that affects every cell of the body by causing the buildup of a protein building block called cystine. The buildup of cystine causes kidney and other problems throughout the body. NC may lead to slow body growth and small stature, weak bones, hypothyroidism, blindness, muscle weakness, pulmonary dysfunction, and developing and worsening kidney failure by age 9 or 10. It is estimated that there are currently about 500 people in the United States—and 2,000 worldwide—diagnosed with nephropathic cystinosis.


Today, a new generation of patients is going to college, moving away from home, even getting married.

PROCYSBI is a delayed-release form of cysteamine bitartrate that works by continuously reducing the toxic levels of cystine in the cells. This may help prevent irreversible cellular damage. PROCYSBI can provide continuous control of cystine when taken every 12 hours. PROCYSBI plays an important role in the management of nephropathic cystinosis.

PROCYSBI is life-changing therapy for the entire family.

Proper treatment with PROCYSBI can preserve kidney function, control cystine levels consistently for a full 12 hours, and be taken a number of ways. No middle-of-the-night dosing eased the burden on family life. Studies showed that patients’ social, school and total functioning improved significantly. Suddenly, children with nephropathic cystinosis had more time to play, sleep and just be a kid.

One global brand vision centrally planned and locally executed.

Within four months of launch, PROCYSBI captured more than half of the entire US market and is tracking toward similar success in Europe. As the global agency-of-record for the launch of PROCYSBI, Big Arrow participated in the development of US and EU marketing and communications planning; market research; digital strategy and development; creative development; and production of multichannel professional, patient, caregiver, and advocacy communications.

In February 2015, Raptor was awarded the New Treatment Award for PROCYSBI by the Lysosomal Disease Network at the WORLDSymposium.


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