Swedish Orphan Biovitrum AB (publ), also known as Sobi, conducts clinical research in rare diseases, including the multinational PACIFICA Phase 3 trial evaluating pacritinib in adults with myelofibrosis and severe thrombocytopenia. The company’s materials focus on helping health care professionals assess eligibility and follow the study’s randomized comparison with physician’s-choice therapy.
SERB PharmaceuticalsSERB Pharmaceuticals develops, manufactures, and supplies specialist medicines for rare diseases and medical emergencies, with a particular strength in antidotes and other critical-care treatments. Beginning in the 1950s as Société d’Études et de Recherches Biologiques, the company now supplies more than 70 medicines through direct operations and distribution partners in over 100 countries. Its fully integrated platform includes a commercial-scale polyclonal antibody manufacturing capability and four manufacturing facilities across three continents.325412CONSHOHOCKEN, PA
Clinigen GroupClinigen Group helps pharmaceutical and biotechnology companies move medicines through clinical development and into patients, combining clinical-trial supply management with managed access and global partnering. With regional teams across North America, Europe, Africa, Japan and Asia Pacific, the company has accelerated access to medicines in more than 130 countries. Clinigen also supplies hard-to-find and unlicensed medicines through its Clinigen Direct platform, drawing on more than 30 years of experience in that work.FARGO, ND
SymbioSymbio is a specialty contract research organization (CRO) running global clinical trials for dermatology, aesthetics, gastroenterology, and inflammatory diseases. With more than 25 years of therapeutic expertise, the company combines a 5,000-plus-site network across 50 countries with stable teams—45% of its staff have worked together for more than 15 years. Through subsidiary Dow Development Laboratories, Symbio also connects clinical research with topical drug formulation and manufacturing in a 23,000-square-foot FDA-registered, cGMP-compliant facility.PORT JEFFERSON, NY
Leadiant Biosciences, IncFrom its Rockville base, Leadiant Biosciences develops and distributes prescription therapies for people living with rare diseases. The private U.S. pharmaceutical company draws on a longstanding heritage in orphan-drug development, becoming the fourth company in the world to receive a U.S. Orphan Drug Designation in 1984. Its research teams work with physicians, researchers, academic institutions and biotechnology organizations, while patient-support programs provide education and resources beyond the medicine itself.ROCKVILLE, MD