MBS Student Helps Pharma Company Gain Orphan Drug Designation

Silviya Meletath, a first-year Master of Bioscience (MBS) student, helped win an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for BPT Pharmaceuticals in Irvine for a drug that shows promise in treating pediatric Multiple Sclerosis.

"Receiving the Orphan Drug Designation from the FDA was a terrific milestone for BPT Pharmaceuticals," said Lynn Foster, BPT Pharmaceuticals' CEO. "It's a validation of our approach and also a big boost for our discussions with investors and potential licensing partners."

The drug, ProEnzy, is a prophylactic treatment that, if successful, protects patients from the effects of Multiple Sclerosis, potentially even before the debilitating effects of the disease are seen. 

Meletath (MBS '11) researched and drafted the orphan drug application after attending a student workshop in October and taking a course in the application process. She and BPT officials met with representatives from the FDA's Office of Orphan Products Development in February during a public workshop hosted by KGI's Center for Rare Disease Therapies.

"Silviya recommended we seek the designation and provided invaluable help in preparing the application prior to the KGI workshop. She also worked with us directly for both days of the workshop and was critical to developing the successful application," Foster said.

Multiple Sclerosis is most commonly diagnosed in young adults. It afflicts about 450,000 people in the U.S. and about 2.5 million worldwide. But pediatric Multiple Sclerosis only afflicts about 20,000 young people under the age of 18, and it is for this small population that ProEnzy will be used under the Orphan Drug Designation.

To receive orphan designation, the proposed drug therapy must target a rare disease (one with a patient population of 200,000 or less) and it must show promise for treating the rare disease.

Rare diseases affect nearly 30 million Americans or approximately 1 out of 10 people. Of the 7,000 identified rare diseases, many have no treatments. For those that can be treated, the cost to the patient is often prohibitive.

After practicing medicine in India for six years, Meletath enrolled in KGI in August because she wants to be involved in clinical trials. She became acquainted with BPT Pharmaceuticals through a marketing class project. Based on her research, Meletath recommended the company seek Orphan Drug Designation and volunteered to help draft the application. She asked for and received assistance from two other MBS students, Sonali Talele and Michelle Pesce.

"I give Silviya enormous credit for her persistence and skill because it was not easy.  Through her, we have achieved one of the original goals of the Center for Rare Disease Therapies-to help companies develop new treatments for rare diseases," said Dr. M. Ian Phillips, the center's director.  

The success of the application also reflects positively on KGI.

"Our students are so well prepared in this area that even someone who has not yet graduated can lead a start-up company though this process and actually get a designation," said Sue Moore Fenske, KGI's director of development for strategic initiatives.