Benitec Biopharma announced that the European Commission, based on a favorable recommendation from the EMA Committee for Orphan Medicinal Products (COMP), has granted Orphan Drug Designation to BB-301 as an orphan medicinal product for the treatment of patients with oculopharyngeal muscular dystrophy (OPMD). 

Orphan Drug Designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.

OPMD is a rare inherited myopathy characterized by dysphagia (difficulty in swallowing), the loss of muscle strength, and weakness in multiple parts of the body. Patients typically suffer from severe dysphagia, ptosis (eye lid drooping), tongue atrophy, proximal lower limb weakness, dysphonia (altered and weak voice), limitation in looking upward, as well as facial muscle and proximal upper limb weakness. Progressing throughout that patient's life, OPMD is not typically diagnosed until the individuals reach their 50s or 60s. As the dysphagia becomes more severe, patients become malnourished, lose significant weight, become dehydrated and suffer from repeated incidents of aspiration pneumonia. The latter two ailments often result in death.

Currently, therapeutic strategies employ repetitive surgical interventions that have limited efficacy.

"We are very excited that BB-301 has received Orphan Drug designation in Europe from the EMA COMP. This is a key program in our pipeline and we are happy to see the EMA recognizing the urgent and unmet medical need for a safe and effective treatment for OPMD patients. We believe that our innovative approach may offer new treatment options for patients who might not otherwise be able to receive benefit in treating their disease. Having European Orphan Drug designation will allow us to optimize steps to further advance BB-301 towards regulatory approval," said David Suhy, chief scientific officer.

BB-301 is a ddRNAi therapeutic for the treatment of OPMD comprised of a single expression construct for the 'knockdown and replace strategy' of mutant PABPN1, the principle cellular component that leads to the diseased condition in humans. BB-301 is currently in preclinical development and Benitec plans to initiate IND-enabling studies later this year. Entry into the clinic with a phase I/II study in OPMD patients is anticipated in 2018, subject to toxicity results and future regulatory review.