March 23, 2021

Manufacturing and Marketing Approval ofUPLIZNAⓇ for I.V. Infusion 100mg for the Treatmentof Neuromyelitis Optica Spectrum Disorder in Japan
Mitsubishi Tanabe Pharma Corporation (Head Office: Osaka; President &Representative Director; Hiroaki Ueno; hereafter, “MTPC”) today announced that it
has obtained manufacturing and marketing approval of UPLIZNAⓇ for I.V. Infusion
100mg (MT-0551, generic name: inebilizumab (genetically modified), (UPLIZNA))
for the prevention of relapses of neuromyelitis optica spectrum disorder (includingneuromyelitis optica) by the Ministry of Health, Labour and Welfare in Japan on
March 23, 2021. UPLIZNA is a humanized anti-CD19 monoclonal antibody of which
MTPC obtained license from Viela Bio, Inc., which was acquired by Horizon
Therapeutics plc on March 15, 2021.
Neuromyelitis Optica Spectrum Disorder (NMOSD) is an autoimmune disease of
the central nervous system characterized by severe optic neuritis and transverse
myelitis and designated intractable disease. Relapse may occur repeatedly, and asingle relapse may lead to vision loss or wheelchair activity.
UPLIZNA binds to a protein called CD19 which is expressed on B cells including
antibody-producing plasmablasts and plasma cells, and rapidly depletes these cells
from the circulation to prevent relapse in NMOSD. This approval is based on thedata of global clinical study conducted by Viela Bio in patients with NMOSD including
Japan. In the U.S., it was approved for the indication of NMOSD on June 11, 2020
under the name of UPLIZNA.
UPLIZNA is a drug for the treatment of NMOSD with a new mechanism, and theconvenience of a dosing interval of once every six months should provide a newtreatment option for NMOSD patients in the relapse prevention period. In addition,
for myasthenia gravis and IgG4-related disease as indications following NMOSD,
global phase 3 clinical studies including Japan are currently underway and will be
conducted in collaboration with Horizon Therapeutics.
MTPC will continue to actively pursue R&D in pharmaceuticals that address unmet
medical needs.

1/2About Horizon Therapeutics plc
Horizon is focused on the discovery, development and commercialization ofmedicines that address critical needs for people impacted by rare, autoimmune andsevere inflammatory diseases. Our pipeline is purposeful: we apply scientificexpertise and courage to bring clinically meaningful therapies to patients. We
believe science and compassion must work together to transform lives. For more
information on how we go to incredible lengths to impact lives, please visit
www.horizontherapeutics.com.
About Neuromyelitis Optica Spectrum Disorder (NMOSD)NMOSD is a rare, severe, relapsing, autoimmune disease of the central nervous
system that can be fatal. In Japan, it has a low prevalence rate of 2 to 4 cases per
100,000 population1. The body's immune system reacts against healthy cells (mostcommonly in the optic nerve, spinal cord and brain), resulting in NMOSD attack andsevere damage. It may cause pain in the eye and vision loss, severe muscle
weakness and paralysis, numbness, loss of bladder and bowel control andrespiratory failure2. The disease is primarily associated with anti-aquaporin 4(AQP4) antibodies, which are detected in approximately 73%-90% of patients with
NMOSD3.
1

Japanese Society of Neurology, 2017 guidelines for the diagnosis and treatment of multiple
sclerosis / neuromyelitis optica

2

National Institute of Neurological Disorders and Stroke, National Institutes of Healthhttps://www.ninds.nih.gov/Disorders/All-Disorders/Neuromyelitis-Optica-Information-Page

3

Chang VTW, Chang HM. Review: Recent advances in the understanding of thepathophysiology of neuromyelitis optica spectrum disorder. NAN. 2020; 46:199-218.

Mitsubishi Tanabe Pharma Corporation
Corporate Communications Department
Media contacts: TEL:+81 6 6205 5119

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