RARE Daily

FDA Grants Fast Track Designation to Annexon for Guillain-Barré Therapy

September 24, 2019

The U.S. Food and Drug Administration has granted Fast Track designation to Annexon Biosciences for ANX005, its experimental treatment of Guillain-Barré Syndrome, a rare, acute, autoimmune disease.

Guillain-Barré syndrome (GBS) is an antibody-mediated condition that impacts the peripheral nervous system. There are currently no approved therapies in the United States. The condition is typically triggered by a preceding infection and usually involves rapidly progressive weakness in the limbs that may culminate within four weeks to widespread peripheral nerve damage and paralysis. Despite the use of plasmapheresis and high-dose immunoglobulin therapy, many patients are left with residual neurological disability, accompanied by chronic pain and fatigue.

ANX005 is an experimental monoclonal antibody intended to treat patients suffering from autoimmune and neurodegenerative disorders. It is formulated for intravenous administration and is designed to inhibit C1q and the entire classical complement pathway. Annexon has completed a phase 1b clinical trial of ANX005 and plans to advance ANX005 into later-stage clinical trials in GBS. ANX005 has received both Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration for the treatment of GBS.

Fast Track is a designation granted by the FDA to expedite the development, review, and potential approval of experimental product candidates that are intended to treat serious or life-threatening diseases or conditions with an unmet medical need, and includes more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a New Drug Application or Biologics License Application.

“Fast Track designation is a testament to the urgent need for a new therapy to treat patients with GBS, as there are no FDA-approved therapies for this debilitating neurological disease,” said Doug Love, president and CEO of Annexon. “Our goal is to bring ANX005 to patients with GBS as quickly as possible.”

Photo: Doug Love, CEO of Annexon

Author: Rare Daily Staff

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