Akcea Therapeutics (NASDAQ: AKCA) has beaten rival Alnylam Pharmaceuticals (NASDAQ: ALNY) to a first drug approval, getting the nod for its drug inotersen (Tegsedi) in the European Union.
Akcea and Alnylam, both of Cambridge, MA, are developing treatments for the rare genetic disease transthyretin amyloidosis, or ATTR. It stems from a gene mutation that causes the protein transthyretin, which carries vitamin A around the body, to misfold and build up in nerves and other tissues, causing pain, organ failure, and early death. EU regulators approved inotersen to treat neuropathy caused by ATTR.
Akcea is a spinout of Carlsbad, CA-based Ionis Pharmaceuticals (NASDAQ: IONS), which transferred responsibility for inotersen to Akcea in March. Akcea shares rose nearly 5 percent Wednesday. For the EU approval, Ionis receives a $40 million payment in Akcea stock.
As Xconomy reported last week, both inotersen and Alnylam’s patisiran did well in critical Phase 3 studies, but major questions remain as they head toward U.S. approval decisions. Other than patient self-reporting, there will be no way for doctors to judge either drug’s efficacy. “It will be a real challenge to know if a drug is working well or not,” the Mayo Clinic’s Morie Gertz told Xconomy. The lack of clarity could have consequences for pricing and reimbursement.
The FDA is due to rule on patisiran by August 11 and inotersen by October 6.
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