The FDA has granted Breakthrough Therapy Designation to Chugai’s satralizumab, a treatment currently in phase 3 for the rare CNS disorders neuromyelitis optica and neuromyelitis optica spectrum disorders (NMO/NMOSD).
Chugai is part of the Roche group, and the announcement marks the seventh Breakthrough Designation Therapy (BTD) received for four drugs discovered by the Japanese firm, which include haemophilia treatment Hemlibra.
Satralizumab is a recycling antibody created via Chugai’s proprietary antibody engineering technologies, and works by blocking the autoimmune condition underlying NMO/NMOSD.
“We are delighted by the FDA’s designation for this innovative antibody based on the results of a Chugai-initiated global phase 3 study” said Chugai’s executive vice president, co-head of project & lifecycle management unit, Dr. Yasushi Ito.
“Satralizumab is designed to inhibit signalling of the inflammatory cytokine IL-6, which is known to play a role in the pathogenesis of NMO/NMOSD. 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 BTD designation for satralizumab is based on data from the phase 3 study (SAkuraSky study, NCT02028884) evaluating it in addition to baseline therapy.
NMOSD is a rare, lifelong, and debilitating autoimmune disease of the central nervous system (CNS) characterised 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, often including visual impairment, motor disability, and loss of quality of life. In some cases, attacks of NMOSD result in death.
Just yesterday the company announced results from a further phase 3 trial of the drug as a monotherapy in the disease. The therapy achieved its primary endpoint, a significant reduction in the risk of relapse was confirmed in patients who received satralizumab compared to a placebo arm.
Dr. Yasushi Ito confirmed that the company would now work to prepare the drug for filing with regulators.