Pharmaceutical Business review

Viela Bio receives US FDA breakthrough therapy designation for Inebilizumab to treat Neuromyelitis Optica Spectrum Disorder

Image: The US FDA’s Center for Drug Evaluation and Research. Photo: courtesy of The U.S. Food and Drug Administration.

NMOSD is a rare, life-threatening autoimmune disease affecting the central nervous system.

“The Breakthrough Therapy Designation for inebilizumab is based on results from the largest monotherapy study ever conducted in NMOSD,” said Jorn Drappa, M.D., Ph.D., Chief Medical Officer and Head of Research & Development at Viela Bio.

“Inebilizumab is a humanized monoclonal antibody designed to bind with high affinity to CD19 and deplete a broad range of B cells, including autoantibody-secreting plasmablasts and CD19-expressing plasma cells. We continue our efforts to bring inebilizumab to patients suffering from this devastating disease for which there are currently no approved medicines.”

Breakthrough Therapy Designation is designed to expedite the development and regulatory review of medicines intended to treat a serious condition that have shown encouraging early clinical results which may demonstrate substantial improvement on a clinically significant endpoint over available medicines.

The designation for inebilizumab is based on data from the pivotal N-MOmentum study evaluating inebilizumab as monotherapy.

The FDA and the European Medicines Agency granted Orphan Drug Designation for inebilizumab for the treatment of patients with NMOSD in March 2016 and March 2017, respectively.

NMOSD is a rare, life-threatening autoimmune disease of the central nervous system in which the body’s immune system attacks healthy cells, most commonly in the optic nerves and spinal cord, resulting in severe damage. NMOSD may cause severe muscle weakness and paralysis, loss of vision, respiratory failure, problems with bowel and bladder function and neuropathic pain.

There is currently no cure or approved treatment for NMOSD.

Breakthrough Therapy Designation (BTD) is a U.S. FDA program designed to expedite the development and review of drugs intended for serious or life-threatening conditions. In order to receive a BTD, preliminary clinical evidence must demonstrate that the drug may provide substantial improvement over currently available therapy on at least one clinically significant endpoint.

The benefits of BTD include more intensive guidance from FDA on an efficient drug development program, access to a scientific liaison to help accelerate review time and organizational commitment from FDA.

Source: Company Press Release