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Dicerna's drug for inherited lung, liver disorder snags EU's orphan-drug tag

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Dicerna's drug for inherited lung, liver disorder snags EU's orphan-drug tag

Dicerna Pharmaceuticals Inc.'s experimental treatment for an inherited disorder that causes lung or liver injury has been granted orphan-drug designation from the European Commission.

DCR-A1AT was previously recommended by the European Medicines Agency's Committee for Orphan Medicinal Products for the designation. Cambridge, Mass.-based Dicerna will receive market exclusivity, protocol assistance, fee reductions and government-funded research from the designation.

The drug is intended to treat congenital alpha-1 antitrypsin deficiency, which stems from mutations in the SERPINA1 gene. The gene, responsible for making the protein A1AT, normally protects the body from an infection-fighting enzyme that can attack normal tissues if not regulated. Deficiency of the protein can result in fatigue, jaundice or unintended weight loss. Serious complications include liver cancer, liver failure and destruction of the alveoli in the lungs, causing lung disease.

According to Dicerna's Dec. 17 press release, about 2.4 people out of every 10,000 in the EU are affected by congenital A1AT deficiency. DCR-A1AT targets the mutated SERPINA1 gene and is being tested in clinical trials, including a phase 1/2 study in healthy volunteers.