Global Primary Sclerosing Cholangitis Market, by Drug (BT1023, GS-9674, NGM282, OCA, LUM001, and Others) and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) has potential pipeline products and is estimated to reach US$ 140.4 Mn by 2023.
Pharmaceutical companies are focusing on enhancing research and development for introducing novel therapeutics to address unmet patient needs. For instance, the discovery and development of siRNA (small interfering RNA) therapeutics by Sirnaomics, Inc., with dual-target properties and polypeptide nanoparticle (PNP)-enhanced delivery that directly controls the fibrotic and inflammatory activity is expected to have wide applications in many inflammatory and fibrotic diseases.
The support from the public and private organizations such as PSC Support Organization and National Organization for Rare Disorders serves as the hub of rare disease community, providing assistance programs for patients and advancing clinical trials by granting funds for advanced research programs. The UK PSC Support Organization granted US$ 20,000 to the University of Birmingham for supporting the research and investigation of Vascular Adhesion Protein (VAP-1) in primary sclerosing cholangitis (PSC). Also, in 2017, the Falk Foundation funded a European multi-center phase II study led by the Medical University of Vienna in collaboration with the Medical University of Graz and the Medical University of Hannover, to treat primary sclerosing cholangitis.
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Orphan drug designation granted by regulatory agencies such as the Food and Drug Administration (FDA) and European Medicine Agency (EMA) for the treatment of PSC enables manufacturers to accelerate the drug development process for providing new drug treatments to patients in need. For instance, in 2017, Conatus Pharmaceuticals Inc. was granted orphan designation to IDN-7314 by the FDA and EMA. The drug completed two successful pre-clinical trials, of which the second one was completed in 2017. Also, in 2016, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for BTT1023 drug candidate developed by Acorda Therapeutics, which is in Phase 2 clinical trials currently.
Also, pharmaceuticals companies are focusing on mergers and acquisitions to accelerate the development of new treatment options to address the unmet needs of patients suffering from rare diseases by advancing the therapies into clinical trials. For instance, in 2015, Gilead Sciences, Inc. acquired Phenex Pharmaceuticals AG, to gain access to Phenex’s Farnesoid X Receptor (FXR) program comprising small molecule FXR agonists for the treatment of liver diseases including PSC and nonalcoholic steatohepatitis (NASH). Also, in February 2018, Intercept Pharmaceuticals, Inc. collaborated with Sumitomo Dainippon Pharma Co. Ltd., to develop and commercialize Obeticholic Acid (OCA) for chronic liver disease in Japan, China, and Korea.
In 2014, Shire acquired Lumena Pharmaceuticals, to gain access to its late-stage rare disease pipeline assets indicated for gastrointestinal conditions- LUM001 and LUM002, thus providing potential treatment options to patients with rare cholestatic liver disease and adding products to its rare disease portfolio.
Key Takeaways of the Primary Sclerosing Cholangitis Market: