Therapy Areas: Central Nervous System
Horizon Therapeutics Submits Brazil Regulatory Filing for Uplinza
15 June 2022 - - Dublin-based biotechnology company Horizon Therapeutics plc (NASDAQ: HZNP) has submitted a regulatory filing to the Brazil National Health Surveillance Agency (ANVISA) for Uplinza for the treatment of adult patients with anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD), the company said.

In the N-MOmentum Phase 3 clinical trial, the largest NMOSD trial to date, Uplinza demonstrated a significant reduction in the risk of an NMOSD attack with only two infusions per year, following the initial two loading doses.

Additionally, 89% of patients in the AQP4-IgG+ group remained attack-free during the six-month period post-treatment and 83% of patients on treatment remained attack-free for at least four years.

Uplinza was approved by the US Food and Drug Administration in June 2020, by the Japanese Ministry of Health, Labor and Welfare in March 2021 and by the European Commission in April 2022.

Mitsubishi Tanabe Pharma Corp. has the rights to develop and commercialize Uplinza in Japan, Thailand, South Korea, Indonesia, Vietnam, Malaysia, the Philippines, Singapore and Taiwan.

Hansoh Pharmaceutical Group company Ltd., another strategic partner to Horizon, has also recently received manufacturing and marketing approval from the National Medical Products Administration of the People's Republic of China for Uplinza.

About Neuromyelitis Optica Spectrum Disorder (NMOSD) is a unifying term for neuromyelitis optica and related syndromes. NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord, brain and brain stem.

Approximately 80% of all patients with NMOSD test positive for anti-AQP4 antibodies.

AQP4-IgG binds primarily to astrocytes in the central nervous system and triggers an escalating immune response that results in lesion formation and astrocyte death.

Anti-AQP4 autoantibodies are produced by plasmablasts and plasma cells.

These B-cell populations are central to NMOSD disease pathogenesis, and a large proportion of these cells express CD19.

Depletion of these CD19 B cells is thought to remove an important contributor to inflammation, lesion formation and astrocyte damage.

Clinically, this damage presents as an NMOSD attack, which can involve the optic nerve, spinal cord and brain.

Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure can all be manifestations of the disease.

Each NMOSD attack can lead to further cumulative damage and disability.

NMOSD occurs more commonly in women and may be more common in individuals of African and Asian descent.

Horizon is a global biotechnology company focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases.


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