United Therapeutics

United Therapeutics was founded in 1996 by Martine Rothblatt, an American lawyer, author, and entrepreneur, who created [6] Sirius XM. In 1994, Rothblatt's young daughter was diagnosed with a fatal orphan disease, pulmonary arterial hypertension (PAH).[7][8] Rothblatt sold her telecom stock and started the $3 million PPH Cure Foundation to fund PAH research.[2][8]

By 2002, the U.S. Food and Drug Administration (FDA) had approved United Therapeutics' new drug Remodulin,[9] a prostacyclin vasodilator used to treat PAH. Remodulin provided PAH patients an alternative to GlaxoSmithKline's Flolan.[7] Within a year of being approved, Remodulin annual sales had reached $50 million.[8] By 2010, annual sales were $300 million and United Therapeutics' share price had increased 800 percent from the 1999 initial public offering price.[8]

Over the years, United Therapeutics has completed several strategic acquisitions. In 2011, it acquired Revivicor, a company focused on developing genetic biotechnology platforms to provide alternative tissue sources for treatment.[10] In 2018, it acquired SteadyMed, a medical device company developing injectable therapeutic drugs for pulmonary hypertension.[11] Other acquisitions include SynQuest in 1999 and Cooke Pharma in 2000. United Therapeutics has entered numerous licensing agreements and collaborations with companies such as Eli Lilly and Company, Medtronic, DEKA Research & Development, and with the National Cancer Institute. In October 2020, United Therapeutics’ market capitalization was $4.7 billion.[12]

United Therapeutics products are sold in the U.S., Canada, Central and South America, Europe, the Middle East, and Asia. In the U.S., the company has received multiple approvals from the FDA.[13] These cover various strengths and routes of administration for Orenitram, Remodulin, and Tyvaso, each indicated for the treatment of pulmonary arterial hypertension, and Unituxin used in the treatment of pediatric patients with high-risk neuroblastoma. United Therapeutics also markets Adcirca (tadalafil) for the treatment of pulmonary arterial hypertension under a licensing agreement with Eli Lilly and Company.

In collaboration with Medtronic, United Therapeutics has developed and expects to commercialize an implantable system for Remodulin.[14] The system will provide continuous delivery of Remodulin from a pump implanted in the patient's abdomen through a surgical procedure.

In February 2020, the U.S. Food and Drug Administration granted an extra clearance to the Unity Subcutaneous Delivery System for Remodulin Injection, also known as the Remunity pump, allowing the system to be used with drug cassettes that have been prefilled by specialty pharmacies.[15] The Remunity system allows for constant, subcutaneous (under-the-skin) delivery of Remodulin, which mimics the effects of prostacyclin, a natural compound that works as a vasodilator keeping blood vessels open. Remunity includes a small, lightweight, ambulatory pump with an intended service life of three years and was developed by United Therapeutics and DEKA. The Remunity system is indicated for the treatment of pulmonary arterial hypertension in patients older than 22. Currently, United Therapeutics and DEKA are working on a new version of the system that will contain disposable components that are prefilled during the manufacturing process.[16]

In March 2015, the U.S. FDA approved United Therapeutics’ Biologics License Application (BLA) for Unituxin.[17] Unituxin is a monoclonal antibody used as a second-line treatment for children with high-risk neuroblastoma, a rare form of cancer.

United Therapeutics is advancing a pipeline of research and development projects that includes new indications, formulations, and delivery devices for its existing products, such as the Remunity and Trevyent delivery systems for Remodulin. The company's R&D pipeline also includes more than a dozen new products to treat pulmonary arterial hypertension, end-stage organ disease, and other conditions.

In February 2020, United Therapeutics reported that it had successfully completed the INCREASE study of Tyvaso in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) and that the study met its primary endpoint of demonstrating improvement in six-minute walk distance (6MWD).[18] The company submitted the INCREASE study results to the U.S. Food and Drug Administration in support of an efficacy supplement to the Tyvaso New Drug Application, which is expected to result in revised labeling reflecting the outcome of the INCREASE study.

Ongoing clinical trials of new medicines include PERFECT (Phase 3) for COPD; ADVANCE CAPACITY and ADVANCE OUTCOMES (Phase 3) for ralinepag in pulmonary hypertension; and SAPPHIRE (Phase 2) for gene therapy.[19] Other medicines being developed include formulations of treprostinil for interstitial lung disease without pulmonary hypertension and dry powder inhaler preparations for pulmonary arterial hypertension.

United Therapeutics is developing a unique type of biopharmaceutical medicinal product known as a manufactured organ. This type of biologic would be transplanted into the patient as a substitute for a failing organ. The company has regenerative medicine products such as these being developed via xenotransplantation, allogenic recellularization, and autologous bioprinting technologies.[20]

United Therapeutics owns a 415,000-square-foot combination laboratory and office building complex in Silver Spring, Maryland. The facility serves as the company's co-headquarters and is used for commercial manufacturing of active pharmaceutical ingredients and finished dosage form products. United Therapeutics also owns a 380,000-square-foot combination manufacturing plant and office building in Research Triangle Park, North Carolina. The facility serves as the company's co-headquarters and is occupied by clinical research and development, commercialization, logistics, manufacturing, and packaging personnel. Adjacent to its Research Triangle Park complex, United Therapeutics owns a 132-acre site containing approximately 330,000 square feet of building space that is used for research, development and manufacturing related to the company's lung regeneration program. This site is available for future expansion.[21]

Through its wholly owned subsidiary Lung Biotechnology PBC, United Therapeutics focuses on addressing the acute national shortage of transplantable lungs and other organs with a variety of technologies that either delay the need for such organs or expand the supply. Lung Biotechnology is the first public-benefit corporation subsidiary of a public biotechnology or pharmaceutical company.[22]

Other United Therapeutics subsidiaries include Lung Bioengineering Inc.; SteadyMed Ltd., Israel; United Therapeutics Europe, Ltd.; Unither Biotech Inc., Canada; Unither Pharma, LLC; Unither Telmed, Ltd.; Unither Therapeutik GmbH, Germany; Unither Bioélectronique, Quebec; and Revivicor, Inc.[23]

Martine Aliana Rothblatt, founder and chairwoman of the board of United Therapeutics, graduated from University of California, Los Angeles, with J.D. and M.B.A. degrees in 1981. In June 2001, she earned a Ph.D. in medical ethics at the Barts and The London School of Medicine and Dentistry, Queen Mary University of London. Rothblatt worked in Washington, D.C., first in the field of communications satellite law, and eventually in life sciences projects like the Human Genome Project. She also served as the CEO of GeoStar and the creator of SiriusXM Satellite Radio.[6][8] Michael Benkowitz serves as president and chief operating officer; James Edgemond is chief financial officer and treasurer; Paul Mahon is general counsel.