UA-discovered drug for skin disease approved for patients in Europe

A synthetic hormone developed by faculty at the University of Arizona Cancer Center and the UA’s Colleges of Medicine and Science more than two decades ago that has been shown to offer relief for a rare skin disease has been approved for patients in Europe.

Clinuvel Pharmaceuticals Ltd. of Melbourne, Australia, received permission Oct. 23 from the European Medicines Agency to market the drug, whose trade name is Scenesse. The company says it plans to seek approval for Scenesse from the U.S. Food and Drug Administration and also for patients in the Asia-Pacific region.

The molecule was developed, patented and initially tested at the UA under the name afamelanotide by Victor Hruby, PhD, Regents Professor Emeritus of chemistry and biochemistry; Robert Dorr, PhD, leader of the UACC’s therapeutic development scientific program and professor of medical pharmacology in the UA’s College of Medicine-Tucson; and the late Mac Hadley, PhD, a UA cell biology and anatomy professor. The UA licensed the molecule to a predecessor company, Melanotan Inc., in 1995.

"The discovery and development of this melanotropin compound for treatment of skin disorders and related medical applications had tremendous significance because it was a first of its kind and involved the very close collaboration of chemists, biologists and medical doctors who brought similar excitement and creativity to the problem but from different perspectives. These perspectives were all critical to the eventual success. Without this enthusiastic collaboration it would not have happened," Dr. Hruby said.

“This is the first pure melanotropin ever approved,” Dr. Dorr said.

Scenesse has been demonstrated to reduce and prevent painful reactions to sunlight experienced by people with erythropoietic protoporphyria (EPP) following trials in Europe, Australia and the United States.

EPP is a rare genetic disease found mainly in people with fair skin. It is characterized by intolerance to light and results in pain, swelling, burning and scarring of sun-exposed areas of skin. People with this condition are often forced to remain indoors during daylight hours. As many as 10,000 people suffer from EPP worldwide, with approximately 45 percent of them in Europe. EPP is considered the most common form of porphyria in children.

Afamelanotide, formerly known as Melanotan I, is a superpotent form of the naturally occurring alpha-Melanocyte Stimulating Hormone (MHS), which stimulates melanin production. Melanin is known for its photoprotective effect on the skin. Initial proof-of-principle clinical trials were conducted at the UA Cancer Center under the Chemoprevention of Skin Cancer program project grant, led by David S. Alberts, MD. Initial study results were published in the Journal of the American Medical Association in 1991.

Afamelanotide originally was developed by UA scientists as a defense against skin cancer, Dr. Dorr said. Stimulating a person’s natural photoprotective skin pigmentation, melanin, would create tanned skin prior to sun exposure, protecting the person from harmful UV rays.

Clinuvel also is testing Scenesse as a treatment for several other skin conditions, including vitiligo and Hailey-Hailey disease.

Read Dr. Dorr's recollections of the development of afamelanotide.

- Nov. 3, 2014