June 30, 2022
Anti-cytomegalovirus chemotherapeutic agents "Valixa® Dry Syrup 5000mg"
Notification of application for additional indication of symptomatic congenital
cytomegalovirus infection in Japan
Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation (Head Office: Chuo-ku, Osaka; Representative
Director: Hiroaki Ueno; hereinafter, MTPC), a member of the Mitsubishi Chemical HoldingsGroup, has announced today that MTPC filed an application for an additional indication of theanti-cytomegalovirushydrochloride,agent Valixa® Dry Syrup 5000mg (generic name: valganciclovir

hereinafterValixa®)

forthetreatment

ofsymptomaticcongenital

cytomegalovirus infection.
An investigator-initiated trial evaluating the efficacy and safety of Valixa® in children withsymptomatic congenital cytomegalovirus infection (VGCV-1) showed improvement ofsymptoms, and therefore MTPC, the manufacturer and distributor of Valixa® in Japan, filed anapplication using the results of VGCV-1 trial. VGCV-1 trial has been done an investigatorinitiated clinical trial conducted by a research group at a total of 6 medical institutions including
The University of Tokyo Hospital, Nihon University Itabashi Hospital, Nagoya University
Hospital, Fujita Health University Hospital, Kobe University Hospital, and Nagasaki UniversityHospital.
Valixa® has been granted Orphan Drug Designation for the treatment of symptomaticcongenital cytomegalovirus infection. If approved for symptomatic congenital cytomegalovirus
infection, it would be the first treatment in the world.
Symptomatic congenital cytomegalovirus infection is a rare disease that develops whencytomegalovirus is transmitted to the fetus via the placenta of a pregnant woman.
Approximately 1,700 newborns per year develop the disease in Japan. In neonates withsymptomatic congenital cytomegalovirus infection, symptoms such as central nervous systemdisorders and deafness are often observed at birth, followed by neurological sequelae such as
mental retardation. Thus, it is a problem that Patients with the symptomatic congenital
cytomegalovirus infection have an impaired growth and development. Currently, there is noapproved treatment for neonates/infants with for symptomatic congenital cytomegalovirus
infection in Japan or overseas, and therefore, there is an unmet medical need in this patientpopulation.

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Valixa® Dry Syrup obtained manufacturing and marketing approval in Japan in 2018. It isapproved as a therapeutic drug for cytomegalovirus infection in “acquired immunodeficiency
syndrome,” “organ transplantation (including hematopoietic stem cell transplantation),” and“malignant tumors,” and a suppressant for cytomegalovirus infection associated with “organtransplantation (excluding hematopoietic stem cell transplantation).”
MTPC will continue to advance R & D activities so that we can deliver the new treatment
options for diseases with unmet needs, including rare diseases.

Mitsubishi Chemical Holdings Group
Corporate Communications Div.
Osaka Corporate Communications Dept.
Media contacts: [+81] (0)6-6205-5119<Reference>■Investigator-initiated clinical trial
An investigator-initiated clinical trial is a clinical trial conducted by a doctor who plans/draws
up a clinical trial by himself/herself and submits a clinical trial plan notification. It is a clinical
trial in which the doctor himself/herself has responsibility for not only the “conduct of the clinicaltrial” but also the entire clinical trial such as “preparation for the clinical trial” and “managementof the clinical trial”.

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