Arrowhead Receives Positive EMA

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Arrowhead Receives Positive EMA Opinion on Orphan Designation for ARO-AAT for Treatment of Congenital Alpha-1 Antitrypsin Deficiency
Jun 27, 2018 at 7:30 AM EDT
PASADENA, Calif. --(BUSINESS WIRE)--Jun. 27, 2018-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on Arrowhead’s application for orphan designation of its RNAi

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PASADENA, Calif.--(BUSINESS WIRE)--Jun. 27, 2018-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on Arrowhead’s application for orphan designation of its RNAi candidate, ARO-AAT, for the treatment of congenital alpha-1 antitrypsin deficiency. ARO-AAT was previously granted orphan drug designation by the United States Food and Drug Administration in February 2018.

Bruce Given, M.D., Arrowhead’s chief operating officer and head of R&D, said, “ARO-AAT has the potential to provide patients and physicians with a much-needed treatment option for liver disease associated with alpha-1 antitrypsin deficiency, a rare genetic disorder. The COMP’s adoption of a positive opinion for ARO-AAT orphan drug designation is another important milestone for this program, which continues to advance rapidly.”

The positive opinion issued by COMP will be sent to the European Commission which is expected to grant the orphan designation within 30 days. Orphan designation in the EU allows Arrowhead to benefit from a number of key incentives, including reduced regulatory fees, protocol assistance, and market exclusivity, to develop a medicine for the treatment of a rare disease affecting not more than five in 10,000 people in the European Union.

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.