The European Commission (EC) has authorized approval for Horizon Therapeutics’ Uplizna (Inebilizumab) as monotherapy for the treatment of neuromyelitis of the optic spectrum (NMOSD) in adults.
The treatment is intended for patients with NMOSD who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG +).
In November last year, the Committee for Medicinal Products for Human Use of the European Medicines Agency gave a favorable opinion on Uplizna for the treatment of NMOSD.
The latest approval is based on findings from a double-blind, multicenter, randomized, placebo-controlled phase II / III N-MOmemtum clinical trial conducted in 25 countries.
The results showed that Uplizna showed a significant reduction in the risk of NMOSD attack with two infusions per year after pre-loading doses.
Nearly 87.6% of study participants who received Uplizna were without seizures for 28 weeks.
In addition, 89% of patients in the AQP4-IgG + group had no relapse for six months after treatment, while over 83% of patients in treatment remained seizure-free for at least four years.
With approval, Uplizna is valid for use in all EU countries and Iceland, Liechtenstein and Norway.
Uplizna has been approved by the US Food and Drug Administration (FDA) and the Japanese Ministry of Health, Labor and Welfare as a targeted CD19 B-cell-depleting antibody to treat AQP4-IgG + NMOSD in adults to reduce the risk of seizures.
Vikram Carnani, International Executive Vice President and President of Horizon Therapeutics, said: “NMOSD is a devastating disease with unpredictable attacks, cumulative and often irreversible damage, and potential loss of vision and motor function, causing deep uncertainty for patients.
“Uplizna’s approval marks a significant milestone for patients in Europe and for Horizon, providing a new, targeted treatment option for people living with NMOSD.”
A rare neuroinflammatory autoimmune disease, NMOSD attacks the optic nerve, spinal cord, brain and brainstem.
Last June, Horizon and Arrowhead Pharmaceuticals signed a global collaboration and licensing agreement to develop a therapeutic agent for ribonucleic acid interference, ARO-XDH.