There remains to be a huge area of unmet needs in the arena of medicine. This is especially true when it comes to rare diseases and ultra-rare illnesses. Because there are not that many patients who are suffering from these diseases, not that many pharmaceutical companies invest in the development of novelty therapeutics.
Ultragenyx Pharmaceutical, Inc. is one of the few companies in the United States that have taken on the challenge of pushing boundaries and providing hope to patients with orphan diseases. The company is registered as a clinical-stage biopharmaceutical venture that is headquartered in Novato, California. Incorporated in June 2011, the business engages in the identification, acquisition, development and commercialization of novelty therapeutics intended for rare and ultra-rare diseases. It has put emphasis on debilitating genetic diseases. The pipeline products of the company are categorized under two treatments: biologics and small molecule.
Their product portfolio includes KRN23 (UX023), rhGUS (UX003), rhPPCA (UX004), Triheptanoin (UX007) and SA-ER (UX001). KRN23 is a fully human monoclonal antibody that is intended as treatment or patients with X-linked hypophosphatemia (XLH). This therapeutic is administrated via subcutaneous injection. rhGUS is an intravenous medication that is intended as a treatment for mucopolysaccharidosis 7 (MPS 7). rhPPCA is a therapeutic that is being developed for galactosialidosis. Trihepatnoin is a replacement therapy for patients who are suffering from long-chain fatty acid oxidation disorders (LC-FAOD). SA-ER is an orally administered medication for hereditary inclusion body myopathy (HIBM). The majority of these novelty therapeutics is intended to address the needs of patients, who are left with no therapeutic options.
With the advancement of the medications being developed by Ultragenyx Pharmaceuticals, patients with debilitating rare diseases are given hope of a better tomorrow. The business is traded publicly on the NASDAQ using the ticker symbol RARE. It is listed as one of the components of the Wilshire 5000 Index.