FDA grants orphan drug status for UNMC therapy


Designation will help Juvenile Batten Disease therapy move into clinical trials before end the year, officials say

NEW YORK and CLEVELAND (July 5, 2017)—The FDA announced recently that it has granted Abeona Therapeutics Inc. the Orphan Drug Designation for its work on a gene-therapy approach to Juvenile Batten Disease, according to an Abeona press release issued last week.

“This designation helps advance the [Juvenile Batten Disease gene therapy] program and we look forward to initiating human clinical trials later this year,” Abeona President and CEO, Timothy J. Miller, Ph.D., said in the release.

UNMC Professor of Pathology Tammy Kielian, Ph.D., (left)—seen here with doctoral student Megan Bosch—is UNeMed’s 2015 Innovator of the Year for work against Juvenile Batten Disease and biofilm infections.

Abeona is a biopharmaceutical company focused on gene therapy approaches for rare deadly diseases. Abeona’s work with Juvenile Batten Disease is based on the research of Tammy Kielian, Ph.D., one of the University of Nebraska Medical Center’s most celebrated and talented researchers.

Dr. Kielian was the 2016 Scientist Laureate, UNMC’s highest honor bestowed on researchers, and was named the 2015 Innovator of the Year by UNMC’s technology transfer and commercialization office, UNeMed.

Dr. Kielian directed her research toward Juvenile Batten Disease several years ago when her young niece was diagnosed with the fatal neurodegenerative disorder.

According to Abeona’s press release, the “Orphan drug designation is granted by the FDA to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.”

Dr. Kielian was also named a UNMC Distinguished Scientist in 2009 and won UNeMed’s Emerging Inventor Award in 2012.

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