Benitec receives U.S. Orphan Drug Designation for BB-301, its ddRNAi therapeutic for the treatment of oculopharyngeal muscular dystrophy

Sydney, Australia, 15 January 2018: Benitec Biopharma Limited (ASX:BLT; NASDAQ:BNTC; NASDAQ:BNTCW) today announced that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to BB-301 for the treatment of oculopharyngeal muscular dystrophy (OPMD).
The Orphan Drug Designation granted to Benitec may provide a range of valuable benefits, including fast track process for clinical regulatory approval, potential extension of patent life with a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees. In short, a clear and expeditious path for cost-efficient development and commercialisation. The granting of orphan status from the FDA in the US follows on from receiving orphan designation from the European Medicines Agency in early 2017.
Greg West, CEO, Benitec Biopharma, commented on today’s news, “We are very pleased to have received Orphan Drug Designation from the FDA for BB-301, as it is another significant step forward for a key program in our pipeline. We believe BB-301 represents a promising new approach for the treatment of OPMD and has the potential to make a meaningful impact for patients who have this debilitating disease. The Benitec team is focused on executing our plan to advance BB-301 into human clinical trials by the end of 2018.”
BB-301 is a single vector (gene therapy construct) system which uses DNA directed RNA interference (ddRNAi) to silence expression of the mutant gene associated with OPMD, while simultaneously adding back a copy of the normal version of the same gene to restore gene function. Nonclinical safety studies and manufacturing work are progressing and Benitec intends to file an Investigational New Drug Application (IND) in the last quarter of calendar year 2018.
In November last year Benitec advised it had completed pre-investigational new drug application (pre-IND) and scientific advice meetings with the U.S. FDA, Health Canada and several European agencies. The purpose of these meetings was to discuss the regulatory development pathway for BB-301 as a treatment for OPMD and to ensure Benitec’s proposed development program addressed the regulatory expectations of these agencies. In addition to these regulatory meetings, the transfer of production protocols and optimisation of the processes related to manufacturing of BB-301 are well underway at Benitec’s contract manufacturing organisation (CMO).
Mr. West concluded, “We also believe this program, if successful, can act as a proof of concept for using our groundbreaking ‘silence and replace’ technology for other therapeutic targets potentially expanding market opportunities for Benitec and paving the way for the development of other monogenic orphan disease programs in the future. Management is dedicated to generating shareholder value through this and our other promising programs currently in the development pipeline, and I look forward to sharing 2018 updates with the market as they occur.”
The FDA's Office of Orphan Drug Products may grant orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States

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