What OrphanPacific does

OrphanPacific, Inc. (株式会社オーファンパシフィック) is a Tokyo-based specialty pharmaceutical company founded on May 1, 2012 as a joint venture between CMIC Holdings Co., Ltd. (51%) and Medipal Holdings Corporation (49%) with paid-in capital of 100 million yen. The company develops and commercializes orphan drugs for rare diseases in Japan, addressing an estimated gap where approximately half of orphan drugs approved in the US and EU remain unapproved in Japan. Its portfolio spans approximately ten products including Buphenyl and Ravicti for urea cycle disorders, Normosang for acute porphyria, Dantrium for malignant hyperthermia and neuroleptic malignant syndrome, Diazoxide for hyperinsulinemic hypoglycemia, Rasilez for hypertension, Rynspad for severe α1-antitrypsin deficiency, Rabipur for rabies prevention, and the recently approved Joenja (leniolisib) for activated PI3K delta syndrome (APDS) and Ravicti oral liquid.

The company operates under its proprietary "Innovative Pharma Model" (IPM), which leverages CMIC Holdings' pharmaceutical development and manufacturing infrastructure alongside Medipal Holdings' pharmaceutical distribution network—one of Japan's largest—to efficiently bring globally-developed orphan drugs to Japanese patients. OrphanPacific generates revenue through pharmaceutical product sales (one-time licensing model) to healthcare institutions via a direct sales force of medical representatives (MRs), targeting physicians and pharmacists who treat rare disease patients. The company engages in licensing partnerships with international developers such as Pharming Group (Joenja) and Immedica Pharma (Ravicti) to expand its portfolio, distributes products through Medipal's wholesaler network, and supports healthcare professional and patient community engagement via medical conferences, disease awareness websites, and the OPOS online information service. Headcount stands at 51–100 employees, and the company recently relocated its Tokyo head office in July 2025.

Name

OrphanPacific

Legal name

OrphanPacific, Inc.

Website

https://orphanpacific.com

Company type

Private

Founded year

2012

Operating status

Operating

Headcount range

51–100 employees

Short description

OrphanPacific is a Tokyo-based specialty pharmaceutical company that develops and commercializes orphan drugs for rare diseases in Japan, operating as a joint venture between CMIC Holdings and Medipal Holdings with a portfolio of approximately ten specialty products.

Ownership category

akta.pro rank

Product category

Orphan Pharmaceuticals (Rare Disease Drugs)

NAICS

Pharmaceutical and Medicine Manufacturing (3254), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

akta.pro secondary industries

Rare Gastroenterology & Hepatology Disorder Therapies (HLAIAIAL), Pulmonary Hypertension & Rare Pulmonary Disease Therapies (HLAIAIAG), Rare Endocrine & Growth Disorder Therapies (HLAIAIAJ)

Keywords

• Orphan drug development
• Rare disease therapeutics
• Specialty pharmaceuticals
• Pharmaceutical distribution
• Rare disease commercialization

Headquarters

HQ city

Tokyo

HQ country

Japan

HQ region

Asia

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Operations, Marketing or Sales, Technology or R&D, Supply Chain, Others

Revenue model

1. Pharmaceutical product sales: OrphanPacific develops and commercializes orphan drugs (稀少疾病用医薬品) for the Japanese market, generating revenue through sales of specialty pharmaceuticals targeting rare diseases. Products include treatments for urea cycle disorders (Ravicti®, Buphenyl®), acute porphyria (Normosang®), malignant hyperthermia/malignant syndrome (Dantrium®), hyperinsulinemic hypoglycemia (Diazoxide), hypertension (Rasilez®), and alpha-1 antitrypsin deficiency (Rinsoeed™).

Go-to-market motion2 records

Distribution channels1 record

Marketing channels5 records

Core offering

OrphanPacific develops, in-licenses, and commercializes orphan drugs (rare disease pharmaceuticals) for the Japanese market under its proprietary Innovative Pharma Model (IPM). The company handles the full life cycle from obtaining marketing/manufacturing-sales approval to nationwide sales through medical representatives and pharmaceutical wholesalers, addressing rare conditions such as urea cycle disorders, acute porphyria, malignant hyperthermia, alpha-1 antitrypsin deficiency, hyperinsulinemic hypoglycemia, hypertension, and activated PI3K delta syndrome (APDS).

Product overview

OrphanPacific operates as a pharmaceutical company specializing in orphan drugs (稀少疾病用医薬品) for rare diseases in Japan. Their portfolio is not a unified software platform but rather a collection of prescription pharmaceutical products commercialized through licensing agreements. The company focuses on urea cycle disorders, acute porphyria, malignant hyperthermia, α1-antitrypsin deficiency, and other rare conditions. Products include Ravicti and Buphenyl for urea cycle disorders, Normosang for acute porphyria, Dantrium for malignant hyperthermia, and various other specialty pharmaceuticals.

Differentiator

Problem solved

Functional benefit

Products and services

• Ravicti (ラヴィクティ, glycerol phenylbutyrate) Oral liquid formulation (1.1 g/mL) for the chronic management of urea cycle disorders (UCDs) — a group of rare inherited metabolic conditions in which the body cannot properly remove ammonia from the blood. Commercialized in Japan by OrphanPacific under license from Immedica Pharma AB; intended for patients with UCDs.
• Buphenyl (ブフェニル, sodium phenylbutyrate) Pharmaceutical product for urea cycle disorders (UCDs), available as 500 mg tablets and 94% granules, that helps reduce elevated ammonia levels in patients with these rare metabolic conditions. OrphanPacific's first commercial product in Japan.
• Normosang (ノーモサング, hemin) Normosang I.V. Injection 250 mg (hemin) administered via intravenous infusion for the improvement of acute attack symptoms in patients with acute porphyria, a group of rare inherited metabolic disorders.
• Dantrium (ダントリウム, dantrolene sodium hydrate) Dantrium is available as a 20 mg IV formulation and 25 mg capsules containing dantrolene sodium hydrate, used for the treatment of malignant hyperthermia (a life-threatening reaction to general anesthesia) and neuroleptic malignant syndrome.
• Diazoxide (ジアゾキシド) 25 mg capsules A 25 mg diazoxide capsule formulation indicated for the treatment of hyperinsulinemic hypoglycemia, a rare condition of recurrent low blood sugar caused by excessive insulin secretion.
• Rasiles (ラジレス, aliskiren fumarate) 150 mg tablets A 150 mg aliskiren fumarate tablet for the treatment of hypertension, distributed by OrphanPacific in Japan.
• Rynspad / Rinsupad (リンスパッド, dried concentrated human α1-proteinase inhibitor) A 1000 mg IV infusion formulation of dried concentrated human α1-proteinase inhibitor (α1-antitrypsin) for the treatment of severe α1-antitrypsin deficiency, a rare hereditary disorder causing early-onset emphysema and liver disease.
• Astomin (アストミン, dimemorfan phosphate) A non-narcotic antitussive medication containing dimemorfan phosphate, available as 10 mg tablets, 10% powder, and 0.25% syrup formulations for the suppression of cough.
• Rabipur (ラビピュール, inactivated tissue culture rabies vaccine) An inactivated tissue culture rabies vaccine for intramuscular injection (2.5 I.U. or more per dose) used for rabies prevention and post-exposure prophylaxis against rabies onset.
• Somazon (ソマゾン, mecasermin) A human somatomedin C (IGF-1, mecasermin) preparation for injection used for treating conditions related to growth hormone deficiency and severe primary insulin-like growth factor deficiency.
• Orladeo (オラデオカプセル150mg) Orladeo 150 mg capsule (icatibant) approved in Japan for the treatment of acute attacks of hereditary angioedema (HAE), a rare genetic disorder causing recurrent severe swelling.
• Joenja (ジョエンジャ, leniolisib) Joenja (leniolisib) is an oral targeted therapy approved for the treatment of activated PI3K delta syndrome (APDS), a rare primary immunodeficiency, in patients aged 4 years and older. Commercialized by OrphanPacific in Japan under license from Pharming Group N.V.

Quantifiable outcome

• Over 10 years of continuous orphan drug supply in Japan

Named customers1 record

Segments2 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Partnerships

One partnership is on record.

• LTL Pharma Co., Ltd.minorStrategic or Co-development Partner · 2 August 2017OrphanPacific entered into a business partnership agreement with LTL Pharma Co., Ltd. for MR activities and academic operations. OrphanPacific began providing product information for LTL Pharma products (Solar Salt, Nivazil, Logan) through its MR activities. The partnership ended at a later date.

Scale indicators3 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Nippon Shinyaku Co., Ltd. Japanese pharmaceutical company with a meaningful rare disease and specialty pharma franchise alongside its core urology business. Directly comparable as a mid-cap Japanese pharma with an active rare disease commercialization arm and Japan-centric distribution model.
• JCR Pharmaceuticals Co., Ltd. Japanese specialty pharmaceutical company focused on rare diseases, biopharmaceuticals, and regenerative medicine products. Comparable as a Japan-headquartered, mid-sized pharma commercializing rare disease and specialty therapies with a similar sales model targeting physicians and hospitals.
• Kyowa Kirin Co., Ltd. Japanese specialty pharmaceutical company with a global rare disease focus (notably in hematology and oncology). Comparable as a Japan-rooted pharma specializing in orphan and specialty therapies with biologics-based products commercialized through specialty channels.
• BioMarin Pharmaceutical Inc. Global rare disease specialist with a portfolio of orphan drugs for ultra-rare genetic conditions. Comparable as a pure-play rare disease commercializer focused on diseases with very small patient populations, providing a useful benchmark for orphan-drug business model economics.
• AnGes, Inc. Japanese specialty biotech focused on genetic and rare disease therapeutics. Comparable as a Japan-headquartered, smaller-cap company commercializing specialty therapies and leveraging partnerships for in-licensing and development.

Broad incumbents

• Chugai Pharmaceutical Co., Ltd. Major Japanese pharmaceutical company (Roche group) with significant rare disease and specialty product portfolio, particularly in oncology and hematology. Comparable as a large Japan-headquartered pharma operating in overlapping rare disease therapeutic areas with extensive commercialization infrastructure.
• Takeda Pharmaceutical Company Limited: Japan-headquartered global pharmaceutical major with a substantial rare disease and specialty franchise (especially post-Shire acquisition). Comparable as the dominant Japan-based pharma player in rare diseases globally, representing both a benchmark and a key competitive presence in Japan.
• Taiho Pharmaceutical Co., Ltd. Established Japanese specialty pharmaceutical company with focus areas in oncology and rare diseases, including orphan drug development. Comparable as a larger Japanese specialty pharma with rare disease capabilities, representing a potential incumbent competitor for orphan assets and physician relationships.
• Alexion (AstraZeneca Rare Disease): Global leader in rare disease pharmaceuticals, now part of AstraZeneca, with deep experience commercializing ultra-orphan therapies including complement-mediated and metabolic disorders. Comparable as a global rare disease benchmark and as a potential competitor/partner for Japan market entry on overlapping indications.
• Sumitomo Pharma Co., Ltd. Japanese pharmaceutical company with rare disease and specialty therapeutic areas (including regenerative medicine and oncology). Comparable as a Japan-rooted specialty pharma operating in overlapping rare disease indications with broader portfolio scope.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/orphanpacific-inc

Revenue estimate

Valuation estimate

Number of profiles

Profiles1 record

Funding overview

Funding rounds

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about OrphanPacific