Printer-friendly format

Milton H. Ellis, RDT President

A child born with homocystinuria usually appears healthy at first, but victims of this inherited genetic disorder may face a variety of complications. A failure of the body to properly process some amino acids, homocystinuria is characterized by a dislocation of the eye lens, an increased risk of blood clots, skeletal abnormalities, and sometimes development and learning problems. Fewer than 2,000 patients worldwide suffer from this rare metabolic syndrome.

Thanks to Franklin-based Rare Disease Therapeutics (RDT), those patients in the United States, South America and Canada have access to the prescription drug Cystadane® (betaine anhydrous for oral solution), effective for treatment of the three most common forms of homocystinuria. In 2007, RDT signed a licensing agreement with a European pharmaceutical company to market Cystadane.

When low profit margins for a drug prompt Big Pharma to sometimes turn its back, small companies like RDT see an opening and step in. As RDT did with Cystadane, these companies license and market low-volume pharmaceuticals developed by other companies. What's more, they may also shepherd development of such pharmaceuticals themselves, contracting with researchers to refine a drug and conduct clinical trials. They also handle the regulatory intricacies involved with federal Food and Drug Administration approval. The drugs they champion treat what are called "orphan" diseases, which are defined in the United States as diseases afflicting fewer than 200,000 Americans.

"We are totally focused on the development of drugs for orphan diseases," explained Milton H. Ellis, RDT president. A veteran of Bristol Myers Squibb, Ellis co-founded RDT in Nashville in 1991 as Orphan Pharmaceuticals, U.S.A. The company changed its name in 2001 to Rare Disease Therapeutics to better describe its mission. In July, the 10-employee company moved from Nashville to the Cool Springs area.

RDT was an early player in the American orphan-drug market, taking advantage of the U.S. Orphan Drug Act, which offered pharmaceutical companies tax incentives and seven years of marketing exclusivity to develop drugs, biologics and other therapeutics for the treatment of orphan diseases. Since the act's passage 25 years ago, more than 325 orphan drugs have been approved by the FDA. Contrast that with the decade before the act, when fewer than 10 orphan drugs hit the U.S. market. Additionally, about 1,800 already existing drugs have received orphan designation, meaning they've been identified as effective treatments for orphan diseases. Today, orphan drug statutes that mirror the American act are in effect in the European Union, Australia, Singapore and Japan, according to the National Organization for Rare Disorders.

Ellis acknowledged that the competition to identify or develop orphan drugs has increased through the years. "The orphan drugs are getting harder and harder to find. That's because a lot of the companies now are taking advantage of the act and developing these products, particularly smaller pharmaceutical companies that want to show their shareholders that they can bring a product to market," he said.

RDT's first product... and its best seller... is Orfadin® (nitisinone), which has been on the market for eight years in the United States, South America and Canada.

"Sales are doing extremely well," Ellis said. He explained that Orfadin treats a rare disorder called Hereditary Tyrosinemia Type 1/HT-1, a genetic metabolic problem that causes progressive liver failure and liver cancer in young children. There are less than 500 known cases in the world and less than 100 children with the diagnosis in the United States. Left untreated, the disorder is fatal in the first year of life.

RDT is also in the pre-clinical stage with a therapy called Recombinant Wolinella Succinogenes Asparaginase to battle childhood leukemia. A similar product already on the market causes allergic reactions after several treatments, so its use must be limited. RDT has licensed the less reactive product from the Children's Hospital of Los Angeles Research Institute and has the product in development through the National Cancer Institute's Rapid Access to Intervention Development program and the NCI's Children's Oncology Group.

RDT is also in the anti-venom business. Ellis said the company is set to file an FDA application by year's end for its product called Anascorp® to treat envenomation by the Centruroides scorpion, which is found in the American Southwest, the Baja Peninsula and parts of Mexico. About 10,000 people annually are stung by the scorpions, including 2,000 children who are usually more drastically affected. Occasional deaths are reported. Anascorp is the first such treatment.

In Phase 3 trials are Anavip®, to treat Crotaline (pit viper) bites, and Analatro®, to treat black widow spider envenomation. Ellis predicted that RDT would file FDA applications for these two therapies within a year.

And where does Ellis see RDT in 10 years? "We should have a good, strong line of anti-venom products on the market, and we still have some that we can develop. So we will continue to work in that area. Being a small company, we currently have quite a big research and development budget with all the products that we've got in clinical trials, and we are working hard to get those products on the market."

Tags:

Milton H. Ellis, Rare Disease Therapeutics, U.S. Orphan Drug Act

Login and voice your opinion!