Oculopharyngeal Muscular Dystrophy Drug Granted Orphan Designation by FDA


Therapy silences the expression of the mutant gene associated with OPMD, while also restoring normal gene function.

The FDA has granted Orphan Drug Designation to an oculopharyngeal muscular dystrophy (OPMD) therapy.

Benitec Biopharma’s BB-301 therapy, a single vector (gene therapy construct) system, was granted the designation almost exactly a year after receiving the same designation from a European Medicines Agency (EMA) committee.

The therapy silences the expression of the mutant gene associated with OPMD with DNA-directed RNA interference (ddRNAi), while also restoring normal gene function. The therapy’s unique distinctions may lay the groundwork for its use in other conditions, Benitec chief executive officer Greg West said in a statement.

“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,” West said.

Benitec is currently undergoing nonclinical safety studies and manufacturing work for the drug, and intend to file an Investigational New Drug Application (IND) in the last quarter of 2018.

BB-301’s pre-investigational new drug application (pre-IND) was completed in November. At that time, Benitec had arranged regulatory advisory meetings with the FDA, Health Canada, and other Europe-based agencies.

The therapy’s designation comes at a time of mixed regulatory reception for novel muscular dystrophy therapies. PTC Therapeutic’s ataluren (Translarna), an investigative oral treatment for Duchenne muscular dystrophy, was rebuffed by an FDA advisory committee in September. The drug’s original New Drug Application was rejected by the administration in 2016.

But West held hope for BB-301, calling its newest designation “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,” West. “The Benitec team is focused on executing our plan to advance BB-301 into human clinical trials by the end of 2018.”

This article was originally published by MD Magazine.

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