At NPS Pharma, the most important thing they make is a difference in the lives of patients with rare diseases.
People living with rare diseases have incredible courage. They have to, because they have few, if any, treatment options and are often overlooked because of the rarity of their disorders. Globally, there are approximately 7,000 rare disease and treatments exist for less than 500 of them. They can do better than this. They must do better than this.
Their sole focus at NPS Pharma is rare diseases, and they are building a global pipeline of first-in- or best-in-disease therapies. Patients are at the center of everything they do. Each of us brings a personal passion to fulfilling their mission: To pioneer and deliver innovative therapies that transform the lives of patients with rare diseases worldwide. Their values and culture are the most important drivers of their success and will support us in achieving their vision of creating a world where every person living with a rare disease has a therapy. Integrity, respect, excellence, personal ownership, teamwork, entrepreneurial spirit and fun are the values that guide their work each day and what they expect from each other. They are the fabric of the NPS Pharma culture.
They develop therapies for patients with significant unmet medical needs. In 2013, they launched their first product in the U.S. called Gattex® (teduglutide [rDNA origin]) for injection, which was also approved in Europe where they are launching it this year in select countries under the trade name Revestive®.
NPS Pharma is currently conducting a global registration study for teduglutide in pediatric patients, because children have considerable, unique needs.
NPS Pharma is also developing rhPTH [1-84], an investigational medicine being studied for the treatment for Hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of parathyroid hormone. Hypoparathyroidism is the only classic endocrine disorder without a parathyroid hormone replacement therapy. Their Biologics License Application is currently under FDA review and they are planning to file a European Marketing Authorization Application for rhPTH[1-84] this year.
Their lead pipeline candidate is NPSP795, a small molecule being developed for the treatment of Autosomal Dominant Hypocalcemia or ADH. There is no approved therapy for this ultra-rare, life-long genetic disorder that affects both adults and children. They have a Phase 2a proof-of-concept study underway to evaluate NPSP795 for the treatment of adults with ADH.
Today, NPS Pharma is a global rare disease company with approximately 300 employees and operations in the U.S., Canada, Europe, Latin America and Japan. They are passionate about transforming the lives of patients with rare diseases through science. Every day, they connect with people living with rare diseases. Every day, they connect with physicians, researchers, universities, policy makers and companies throughout the globe working in rare diseases. Together they can make a difference.