U LT R AGEN Y X PH A R M AC E U T I CA L I N C .
Seeks Innovative Treatments for Patients Who Have Rare and Ultra-Rare Genetic Diseases
BY ULTRAGENYX
“There is nothing more powerful than treating patients who have never been treated before. Nothing is more uplifting and compelling than being in the room and treating a patient for the first time. I’ve been trying to recreate that moment over and over again.” - Dr. Emil Kakkis, CEO and President. Rare diseases affect more people than cancer and AIDS combined. There are over 7,000 rare diseases affecting 30 million Americans. In fact, one in every 10 people is affected by rare diseases, half of whom are children. Of those children, 30% will not live to see their 5th birthday with rare diseases accounting for 35% of deaths in the first year of life. Notwithstanding the prevalence of rare diseases and their devastating effects, only 5% of rare diseases have an FDAapproved drug treatment. Ultragenyx believes that long-term innovation in healthcare is critical for the many patients with rare diseases who are
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MICHBIO | michbio.org
still waiting for an approved treatment and is dedicated to the goal of providing treatments where none currently exist. Founded in April 2010 by Dr. Emil Kakkis with headquarters in Novato, California, and offices and laboratories in Brisbane (CA), Cambridge (MA), Waltham (MA), Woburn (MA), Miami (FL) and Basel (Switzerland), the company employs 796 people globally, and aims to address diseases with high unmet medical need and clear biology for treatment, for which there are few or no approved therapies to treat the underlying disease. Ultragenyx has a diversified pipeline that includes 2 approved therapies (Mepsevii® and Crysvita® − patients in Michigan are being treated with these therapies) and ~20 programs in clinical or pre-clinical development across multiple modalities (small molecule, biologic, gene therapy and mRNA). Mepsevii® is an enzyme replacement therapy approved in November 2017 for pediatric and adult patients who have mucopolysaccharidosis type VII (MPS VII). MPS VII is an ultra-rare disease, affecting ~200 people in the developed world, that is progressively debilitating and life threatening.
