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,… Read more »
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