Workshop Leads to New Orphan Drug Opportunities

Thanks to a workshop co-sponsored by KGI's Center of Rare Disease Therapies, NATCO Pharma Ltd has become the first Indian company to receive approval from the U.S. Food and Drug Administration (FDA) for an Orphan Drug designation application.

NATCO was one of 21 teams from pharmaceutical and biotech companies, as well as from universities that met with FDA representatives earlier this year at KGI to learn step-by-step how to write and file an Orphan Drug designation application.

A team of academics from Oregon Health and Science University in Portland also was among those who submitted Orphan Drug designation applications that received FDA approval.

Dr. Praveen C. Myneni, Coordinator—Drug Development for NATCO, said the company submitted three separate applications for a compound called NRC-AN-019 to treat Chronic Myelogenous Leukemia (CML), pancreatic cancer and Glioma, a form of brain cancer. The compound fit the FDA's parameters for Orphan Drug designation since it targets a rare disease (one with a patient population of 200,000 or less) and it shows promise for treating the rare disease.

Rare diseases affect nearly 25 million Americans. No treatment is available for most of the 7,000 identified rare diseases, and for those that can be treated, the cost to the patient is often prohibitive.

While Orphan Drug designation does not guarantee drug approval, it allows researchers to seek grant funding to conduct further clinical trials and provides incentives to the existing manufacturer to increase production of the drug or for new manufacturers to develop the drug.

Currently, NATCO is conducting a clinical trial for NRC-AN-019 in India. Based on the results, the company plans to start a clinical trial in the United States later this year.

Myneni, who read about the workshop on the FDA website, said, "It was a unique opportunity to interact with FDA officials and receive their guidance. It was so much more than we expected."

Dr. Ed Neuwelt, a professor of neurology and neurosurgery at Oregon Health and Science University in Portland and his team were the only workshop participants who were strictly academics with no commercial funding nor interest in a company.

They received Orphan Drug designation to use ferumoxytol, an iron oxide nanoparticle, as a contrast agent for magnetic resonance imaging of glioblastoma, a malignant brain tumor.

Neuwelt said ferumoxytol is much more accurate than the "gold standard," gadolinium-based contrast agent, for measuring the blood volume of brain tumors and in differentiating between an active tumor and pseudo inflammation in response to radiation and chemotherapy, which occurs in 30 to 50 percent of patients. 

"Pseudo progression is a good indication that you want to maintain the current therapy," Neuwelt said. "If it's real progression, you will want to change the therapy."

Project Manager Lisa Bennett, who helped write the application, said the team arrived at the workshop with a rough draft of the application and met with FDA officials four times over two days to make revisions.

"Our application never would have been in that good a shape if we had not gone to the workshop," Bennett said.

Six, federally-funded clinical trials on ferumoxytol are under way. Neuwelt said the next step is a meeting with the FDA to plan for marketing approval.

Ian Phillips, PhD, Norris Professor of Applied Life Sciences and director of the Center for Rare Disease Therapies, said the FDA workshops started with educational workshops for KGI students. Thirty pharmaceutical companies and patient advocates attended the first commercial workshop in February 2010.

The KGI model was repeated later that year at University of Minnesota and in Washington, DC. This year, the FDA has expanded the workshops to overseas locations including India and the United Kingdom. The next FDA workshop at KGI is planned for Feb 28-29, 2012.

"These workshops are a highly efficient way to get Orphan Drug designation applications done correctly and into the hands of the FDA for approval, which fits perfectly with the center's mission," Phillips said.

By Elaine Regus