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Nashville's Milton Ellis is Honoree of National Organization for Rare Disorders
By: Wallace Nicoll
(June 2000)

Nashville, TN: Milton Ellis, President/CEO of Nashville-based Orphan Pharmaceuticals USA, Inc. has been honored for his efforts to help individuals afflicted with rare disorders in this country as well as internationally. This was recently announced by, Abbey Meyers, President of the National Organization For Rare Disorders (NORD) in Washington, DC.

“It’s an honor for me to present Mr. Ellis with this Humanitarian Award,” said Mrs. Meyers. “His efforts to help find relief for those suffering from a variety of rare disorders goes way beyond the norm. Mr. Ellis and his company, Orphan Pharmaceuticals USA, Inc. are continually searching for ways to assist patients and their families.

Ellis was recognized specifically for the development of Orfadin, a treatment for children with tyrosinemia. This is a life-threatening genetic metabolic disease affecting nfants and children. Assisting Ms. Meyers in presenting the award was Danielle Barckett of Little Falls, New Jersey, a patient with the disease, who is now in excellent health, and leading the normal life of a four year old.

Orphan Pharmaceuticals USA, Inc. specializes in pharmaceutical products to treat orphan diseases. Disorders afflicting less than 200,000 Americans are considered to be orphan diseases. There are more than 5,000 orphan diseases affecting some 20 million Americans. One out of every 12 people has a rare disorder. In 1983, Congress enacted the Orphan Drug Act to give companies financial incentives and regulatory assistance in developing products to treat rare diseases. Such products are designated as “orphan products” by the FDA’s Office of Orphan Product Development.

Orphan Pharmaceuticals USA, Inc. founded in 1991, is a profitable, privately held company that is a leader in the orphan pharmaceutical marketplace.


May, 2000 Nashville Business Journal
Pharmaceutical Firm Inks Pact with Harvard

Nashville based Orphan Pharmaceuticals USA, Inc. has reached an agreement with Harvard University to register and market a drug to treat a rare skin disorder called erythropoietic protoporphyria, or EPP. The research and development of the product was performed by Harvard’s Dr. Micheline M. Mathews-Roth and underwritten by grants from the Food and Drug Administration and National Institutes of Health. Orphan plans to file a new drug application with the FDA within a few months. This product will be distributed by Nashville’s Metro Medical Supply.




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