Announcement of exclusive license agreement fordevelopment, manufacturing and sale ofglycerol phenylbutyrate in Japan
OrphanPacific, Inc. (hereinafter referred to as “OrphanPacific”) and Immedica Pharma AB(hereinafter referred to as “Immedica”) announced that on May 2, 2022, both companies signed
an exclusive license agreement for the following rights in Japan:
 Development, manufacturing and sale of glycerol phenylbutyrate (the brand name in theUnited States and Europe: Ravicti®) for the treatment of Urea Cycle Disorders (UCD)
OrphanPacific has already manufactured and sold Buphenyl® (sodium phenylbutyrate) thathas been supplied by Immedica to Japan. This time, Immedica has further granted
OrphanPacific the rights within Japan to develop, manufacture and sell glycerol phenylbutyratethat has already been approved and sold for the treatment of UCD in the United States andEurope,
In Japan, both Buphenyl® tablets and granules are currently prescribed to control the increasein blood ammonia levels caused by UCD. In the United States and Europe, Ravicti®, has been
widely used instead of Buphenyl® for the management of UCD patients. Ravicti® is an oralliquid containing glycerol phenylbutyrate that is tasteless, odorless and easy to take, and it isa highly convenient formulation, especially for infants and pediatric patients. Accordingly,
medical professionals have been expecting that Ravicti® will become available in Japan aswell.
OrphanPacific is going to start a clinical trial for obtaining approval for glycerol phenylbutyratein Japan.
About Urea Cycle Disorders (UCD)The Urea Cycle is a metabolic pathway that converts toxic ammonia (NH3) generated in thebody into non-toxic urea mainly in the liver. The UCD is a group of diseases that present withhyperammonemia due to congenital abnormalities in enzymes involved in the Urea Cycle.
Patients with UCD may experience vomiting, poor feeding, tachypnea, convulsions, impaired
consciousness, behavioral disorders, developmental disorders, and sometimes lifethreatening conditions. Most of UCD patients develop in infancy, but some of them arediagnosed only in adulthood. The UCD occurs in 1 in 8,000 to 44,000 people and is one of thedesignated intractable diseases in Japan.
[Reference] Intractable Disease Information Center; Urea Cycle Disorders (DesignatedIntractable Disease 251)https://www.nanbyou.or.jp/entry/4732 (Access as of 18 May 2022, in Japanese)About OrphanPacificOrphanPacific is a Japanese pharmaceutical company that brings new therapeutic drugs topatients with rare diseases through the development, manufacturing and sale of orphan drugs.
The company’s mission is to "deliver smiles and happiness to patients with rare diseases andtheir families." With the determination of "Leave No One Behind", OrphanPacific is actively

working on the development and distribution of drugs for the treatment of rare diseases with avery small number of patients. OrphanPacific is a wholly owned subsidiary of CMIC Holdings,
a pioneer and leading CRO (Contract Research Organization) in Japan. Making the best useof the CMIC Group’s experience, and know-how of drug development, manufacturing andsales, the company aims to enable as many patients with rare diseases as possible to accessto therapeutic drugs.

About Immedica Pharma AB

Immedica is a fast-growing private European niche pharma group with excellent proven trackrecord and high performance in international alliances and sales, and has know-how andexperience in selling orphan drugs/specialty care products in Europe and the Middle East.
Having headquartered in Stockholm, Sweden, Immedica has direct trade areas in Europe andthe Middle East, and, in addition, provides some of its products to other parts of the world via
a network of regional partners. The company's management has an excellent track record ininternational alliances and sales of orphan drugs, and Immedica has capabilities to provide
optimal access of specialty care medicines to patients with significant medical needs, including
key areas such as regulatory affairs, pharmacovigilance, medical affairs, pricing &reimbursement, and product distribution.

＜Contact＞
OrphanPacific, Inc., Corporate Planning Department.
info@orphanpacific.com
Tel.03-6779-8151