The FDA has denied Eagle Pharmaceuticals’ request for seven years of orphan drug exclusivity in the U.S. for Bendeka, a liquid, low-volume (50 mL), 10-minute infusion formulation of bendamustine hydrochloride. Bendeka was approved in December 2015 for the treatment of patients with chronic lymphocytic leukemia (CLL) and for the treatment of patients with indolent B-cell non-Hodgkin lymphoma that has progressed during or within six months of treatment with rituximab or a rituximab-containing regimen. The efficacy of Bendeka in CLL relative to first-line therapies other than chlorambucil has not been established.
An orphan drug designation is granted by the FDA’s Office of Orphan Products Development to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation typically provides the drug developer with a seven-year period of U.S. marketing exclusivity upon approval, as well as certain financial incentives that can help support its development.
The FDA currently requires sponsors of certain orphan-designated drugs to make a “clinical superiority” demonstration as a condition to obtaining the seven-year exclusivity period upon approval. The FDA applies this requirement whenever it has previously approved another drug of the same active moiety for the same indication.
In September 2014, DepoMed, Inc. prevailed in litigation in federal district court in the District of Columbia, challenging the FDA’s application of this clinical superiority demonstration requirement to its orphan-designated drug product Gralise (gabapentin), according to Eagle Pharma. DepoMed argued that the requirement violates the Orphan Drug Act, which automatically confers seven years of marketing exclusivity on orphan-designated products upon approval. District Judge Ketanji Brown Jackson agreed with DepoMed’s position, and ordered the FDA to recognize orphan drug exclusivity for Gralise without requiring proof of “clinical superiority.” While the FDA granted orphan drug exclusivity for Gralise without a clinical superiority demonstration, it has continued to require the clinical superiority demonstration for other orphan-designated drugs.
Eagle believes that the FDA’s rejection of orphan drug exclusivity for Bendeka closely mirrors the decision that Judge Jackson overturned in the DepoMed litigation, and the company is evaluating options to obtain a reversal of the FDA’s decision.
Source: Eagle Pharmaceuticals; March 28, 2016.