Orphan designation for Benitec gene therapy

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The US FDA has granted Australian gene therapy company Benitec Biopharma (ASX:BLT) Orphan Drug Designation for its BB-301 for the treatment of oculopharyngeal muscular dystrophy (OPMD).

The Orphan Drug Designation provides a range of potential benefits, including fast-track process for clinical regulatory approval, a seven-year period of market exclusivity if approved, as well as tax credits for qualifying clinical trials and an exemption from FDA application fees.

The European Medicines Agency has also granted BB-301 orphan designation.

BB-301 is a single vector (gene therapy construct) system that 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.

According to the company, non-clinical safety studies and manufacturing work are progressing and it plans to file an Investigational New Drug Application (IND) in the last quarter of calendar year 2018.

“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," said CEO Greg West.

"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.”

He continued, “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.”