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European Commission grants orphan drug designation to oculopharyngeal muscular dystrophy treatment
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After a positive recommendation from the European Medicines Agency, the European Commission has granted orphan drug designation to BB-301, an investigational therapy for the treatment of ocularpharyngeal muscular dystrophy, the manufacturer announced.
BB-301 (Benitec Biopharma) is a ddRNAi or “expressed RNAi” treatment derived from the developing company’s gene-silencing technology, which is being investigated for the treatment of ocularpharyngeal muscular dystrophy (OPMD). The treatment entails a single expression construct for the “knockdown and replace strategy” of mutant PABPN1, the main cellular component involved in the development of OPMD, according to a press release.
OPMD is a rare inherited myopathy causing dysphagia, loss of muscle strength and weakness in several parts of the body. Patients typically experience severe dysphagia, ptosis, tongue atrophy, proximal limb weakness, dysphonia, limitation in looking upward and weakness of the facial muscles and proximal upper limbs.
This progressive disease is usually not diagnosed until patients reach their 50s or 60s, and they can become malnourished, lose weight, become dehydrated and experience aspiration pneumonia as dysphagia severity increases. “The latter two ailments often result in death,” according to the press release. “Currently, therapeutic strategies employ repetitive surgical interventions that have limited efficacy.”
BB-301 is in preclinical development and the company plans to begin studies to support an IND later this year. In addition, a phase 1/2 clinical study in OPMD is planned for 2018 depending on toxicity data and regulatory review.
“We are very excited that BB-301 has received orphan drug designation in Europe from the EMA COMP,” David Suhy, PhD, chief scientific officer of Benitec, said in the press release. “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.”
European orphan drug designation incentivizes development and marketing of treatments for life-threatening or chronically debilitating conditions with insufficient treatment options, and which affect less than five in 10,000 people in the EU. It provides 10-year marketing exclusivity upon approval, incentives to companies seeking the EMA’s assistance with drug development protocol, and direct access to the centralized authorization procedure.
Disclosure: Suhy is employed by Benitec.