FDA Grants Chugai Breakthrough Therapy Designation for Rare Eye Disease

Rare Daily Staff

The U.S. Food and Drug Administration granted Chugai Pharmaceutical Breakthrough Therapy designation for Chugai’s anti-interleukin-6 (IL-6) receptor humanized recycling antibody satralizumab, an experimental therapy for neuromyelitis optica and neuromyelitis optica spectrum disorders (NMO/MNOSD).

Neuromyelitis optica spectrum disorder is a rare, lifelong, and debilitating autoimmune disease of the central nervous system characterized by inflammatory lesions in the optic nerves and spinal cord. Patients with NMOSD frequently experience a relapsing disease course with repeated attacks leading to accumulating neurological damage and disability. Symptoms may include visual impairment, motor disability, and loss of quality of life. In some cases, attacks of NMOSD result in death.

NMOSD pathogenesis is thought to involve AQP4-IgG autoantibody entry into the CNS, however approximately one-third of patients with NMOSD are AQP4-IgG seronegative1)-4). The inflammatory cytokine IL-6 is now emerging as an important factor in NMOSD pathogenesis.

“Satralizumab is designed to inhibit signaling of the inflammatory cytokine IL-6, which is known to play a role in the pathogenesis of NMO/NMOSD,” said Yasushi Ito, Chugai’s executive vice president, co-head of Project & Lifecycle Management Unit. “We continue our efforts to hopefully bring satralizumab as a new treatment option as soon as possible to people living with this devastating disease with no approved drugs.”

The designation for satralizumab is based on data from the phase 3 evaluating satralizumab added to baseline therapy.

Breakthrough Therapy designation is intended to expedite the development and review of drugs for the treatment of severe or life-threatening diseases or symptoms. In order to grant Breakthrough Therapy designation, preliminary clinical evidence is required demonstrating that the drug may have substantial improvement on at least one clinically significant endpoint over existing therapies.

Breakthrough Therapy Designation includes the features of a Fast Track designation, with the addition of intensive guidance on efficient drug development as well as organizational commitment from FDA.

December 20, 2018