Benitec Seeks Regulatory Approval to Advance Its Gene Therapy Candidate for OPMD

Benitec Seeks Regulatory Approval to Advance Its Gene Therapy Candidate for OPMD

Benitec Biopharma has submitted an application for orphan drug designation with the U.S. Food and Drug Administration (FDA) for its lead gene therapy candidate, BB-301, for the treatment of oculopharyngeal muscular dystrophy (OPMD).

If granted, the orphan drug status will guarantee seven-year market exclusivity upon treatment approval, but it also will provide Benitec assistance in regulatory proceedings, tax credits, and exemption of FDA’s product registration fees.

“This submission follows on from receiving earlier this year orphan designation from the European Medicines Agency and is an exciting first step in our effort to secure orphan status for BB-301 in the U.S. where we believe BB-301 has the potential to be a valuable asset in the treatment of OPMD,” Greg West, CEO of Benitec, said in a press release.

The company also has completed pre-investigational new drug application (pre-IND) meetings with the FDA, Health Canada, and several European agencies. This followed several scientific advice meetings with the regulatory agencies to ensure the proposed clinical development program for BB-301 satisfied all the necessary requirements. Benitec expects to submit an IND application with the FDA during the fourth quarter of 2018.

“The Benitec team is executing on the key initiatives required to advance BB-301 into human clinical trials.” West said. “Our meetings with the FDA and other regulatory agencies were very productive and their guidance will be most valuable in assessing the appropriate clinical and regulatory strategies for BB-301.”

“Assuming approval on a normal time-frame, we should be in an initial human clinical study by the end of 2018,” he stated.

OPMD is a genetic disease caused by mutations on the PABPN1 gene, which leads to the production of a faulty protein that can form insoluble aggregates. This disease is characterized by muscle weakness, with patients commonly presenting drooping eyelids, difficulty standing upright, and difficulty swallowing.

Benitec has developed a gene therapy that was designed to correct the PABPN1 gene defect associated to OPMD. BB-301 is intended to destroy the expression of the mutated PABPN1 gene, and at the same time drive the expression of the normal version of the gene.

Preclinical results have showed that BB-301 can effectively reduce the expression of the faulty version of PABPN1 protein and prevent the formation of protein aggregates. This strategy significantly improved the expression of the correct version of the protein in mice.

In addition, administration of BB-301 was shown to significantly improve muscle weight and strength in an OPMD mice model.

These results were presented during the 22nd International Annual Congress of the World Muscle Society, in France, in a presentation titled “Gene therapy for Oculopharyngeal Muscular Dystrophy.”

Average Rating
0 out of 5 stars. 0 votes.
My Rating:


  1. Jacqueline Seaton says:

    So good to see some positive news about OPMD. Iam now 71 and I had a confirmed diagnosis of this about 3 years ago when I complained to my Optician that my eyelids were drooping. Since then I get a bit of blurred vision and my swallowing isn’t all that great. Living on a farm it’s really annoying not to be able to climb over gates and walk the dog very far. I’ve always been fit and active. I now take a daily spoonful of Creatine for muscles and CoQ10 for energy. Also for the last 2 months I have taken Naproxin that helps with my achy hips. So as you can imagine finding a company who seems to be on the road to a treatment really helps with positive thinking! I hope all goes to plan and maybe I might be cured before I reach 80!!

  2. RAKESH KUMAR says:

    Consider my son as trail treatment patient of DMD age 19 year,wt.28 kg Approx, Distrophin exon result exon 46 to 50 found deletion & exon 51 found present. My son is ready to go under Trail treatment as per your programme.

    • Alice Melão says:

      Dear Rakesh Kumar, We at Muscular Dystrophy News are not involved with the development of the research and clinical studies presented. If you are interested in participating in a trial you should contact the investigators that are conducting the study in question. To know more about clinical trials (such as contacts, locations, and status) you should visit the trial registry website

Leave a Comment

Your email address will not be published. Required fields are marked *