Uplizna Approved in Brazil for Adults With AQP4-positive NMOSD
Move follows similar approvals in the US and Europe for the same indication
The Brazilian Health Regulatory Agency has approved Uplizna (inebilizumab-cdon) for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD) who test positive for antibodies against aquaporin-4 (AQP4-IgG).
The Horizon Therapeutics’ therapy is the first and only man-made antibody against CD19 — a cell surface protein present in immune cells implicated in NMOSD — approved as a single therapy to help protect this group of patients from disease attacks.
“The NMOSD community in Brazil now has an approved medicine that has been proven in clinical studies to provide sustained protection against NMOSD attacks,” Vikram Karnani, Horizon’s executive vice president, international and global medical affairs, said in a company press release.
Uplizna now approved in the US, Europe, and Japan
In addition to Brazil, Uplizna is approved for the same indication in the U.S. and in Europe. The therapy is available in Japan for all NMOSD adult patients, regardless of AQP4-IgG status, and it’s commercialized by Mitsubishi Tanabe Pharma.
NMOSD is characterized by inflammation in the brain and spinal cord, which can result in severe and recurrent attacks and permanent disability, including paralysis and vision loss.
In about 80% of cases, this is caused by the production of abnormal antibodies against AQP4, a protein found at the surface of neuron-supporting cells called astrocytes. These aberrant antibodies, as well as normal antibodies involved in the fight against infections, are produced by plasmablasts and plasma cells — mature immune B-cells.
Uplizna is an antibody-based therapy designed to destroy B-cells by targeting CD19, a receptor protein found at the surface of plasmablasts and plasma cells. As such, it is expected to suppress the production of antibodies, including AQP4-IgG, and ultimately reduce the risk of attacks.
The treatment is administered directly into the bloodstream every six months following an initial loading dose given two weeks apart.
“Uplizna is unique in that it prevents attacks by depleting a broad range of B cells, including plasmablasts and plasma cells, and has a very good safety profile in NMOSD patients,” said Douglas Sato, MD, PhD, a neurologist and professor of medicine at the Pontifical Catholic University of Rio Grande do Sul, in Brazil.
Its approval in Brazil “represents a significant advancement in the treatment of NMOSD and can provide hope for those who have been struggling with this disease,” Sato added.
Regulatory approvals were based mainly on results from the six-month Phase 2/3 N-MOmentum trial (NCT02200770), which involved 230 adults with NMOSD (93% positive for anti-AQP4 antibodies).
Among AQP4-IgG-positive patients, 89% remained attack-free during the six months of treatment. Additionally, more than 83% on treatment remained free of attacks for at least four years.
“This approval is an important part of Horizon’s commitment to helping people worldwide who are living with challenging rare diseases,” Karnani said.