Benitec Biopharma Ltd (ADR)(NASDAQ:BNTC) reported that the European Commission, depending on a favorable recommendation from the EMA Committee for Orphan Medicinal Products has given Orphan Drug Designation to “BB-301” as an orphan medicinal offering for the treatment of people with oculopharyngeal muscular dystrophy.
Orphan Drug status by the European Commission offers financial and regulatory incentives for firms to advance and market treatments that treat a chronically debilitating or life-threatening condition affecting almost five in 10,000 persons in the EU, and where no satisfactory cure is available. In addition to a ten-year period of marketing exclusivity in the European Union after product acceptance, orphan drug designation offers incentives for firms seeking protocol help from the EMA in the product development stage, and direct access to the centralized authorization process.
OPMD can be defined as a rare inherited myopathy characterized by dysphagia, the weakness in different parts of the body and the loss of muscle strength. Patients typically face severe dysphagia, ptosis, proximal lower limb weakness, tongue atrophy, dysphonia, limitation in looking upward, and proximal upper limb and facial muscle weakness.
Advancing throughout that person’s life, OPMD is not typically identified until the people reach their 50’s or 60’s. At a stage, when the dysphagia becomes extremely grave, patients become malnourished, become dehydrated, lose significant weigh and suffer from repeated occurrences of aspiration pneumonia. Currently, therapeutic plans use repetitive surgical interventions.
David Suhy, the Chief Scientific Officer of Benitec, reported that they are very thrilled that BB-301 has obtained Orphan Drug status in Europe. It is a key plan in their pipeline and they are thrilled to see the EMA recognizing the unmet and urgent medical need for an effective and safe treatment for OPMD subjects. They consider that their unique approach may provide new treatment alternatives for people who might not otherwise can get benefit in curing their disease.