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Global Cystinuria Market Size, Share & Trends Analysis Report By End-user, By Region, and Segment Forecasts, 2023 - 2032
  • Published Date: Nov, 2023
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  • Pages: 200
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  • Report Summary
  • Table of Contents
  • Segmentation
  • Methodology
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Cystinuria is a rare inherited disease that leads to the build-up of cystine levels in the urine. This leads to the development of recurring cystine kidney stones. Also known as cystine-lysine-arginine-ornithinuria, cystinuria is caused by defects in the SLC3A1 and SLC7A9 genes. Normally, cystine dissolves and is reabsorbed into the bloodstream after passing through the kidneys. However, individuals with cystinuria possess a genetic anomaly that disrupts this natural process. Consequently, cystine accumulates in the urine, leading to the formation of crystalline or stone-like structures.

Global Cystinuria Market was valued at US $110.5 Mn in 2022 and is expected to reach ~US $200 Mn by 2032, growing at a CAGR of 6.1% during the forecast period 2023-2032.

The global cystinuria market is poised to maintain a steady CAGR due to the increasing prevalence of SLC3A1 and SLC7A9 gene mutations. These mutations have led to a rise in cystinuria cases, resulting in abnormal cystine transport within the kidneys. Cystinuria is the most common hereditary cause of kidney stone disease, with an estimated global incidence rate of 1 in 7,000 individuals. Specific countries, such as Sweden, Japan, Australia, Israel, Great Britain, and Spain, have varying prevalence rates ranging from 1 per 100,000 to 1 per 2,000 individuals.

The market is experiencing growth driven by various factors. These include increasing special designations granted by regulatory authorities and expanding awareness of available treatment options. Moreover, there is a notable increase in healthcare infrastructure development expenditure, a rising prevalence of inherited disorders, and a growing preference for online pharmacies as prescribed medication sources. Additionally, the market benefits from an increased rate of strategic collaborations among industry players. Furthermore, the market is expected to witness lucrative growth opportunities due to the rising personal disposable income, albeit indirectly.

Extensive research conducted over the years has led to the development and investigation of various products by major manufacturers and organizations for their therapeutic potential in cystinuria. This ongoing research and development activity is contributing to the growth of the cystinuria treatment market. One notable clinical study, sponsored through collaboration between the FDA and NIH and jointly led by Drs. Chi and Stoller focus on using lipoid acid to treat individuals with cystinuria and assess its effects on their blood, urine, and kidney stones. The study is designed as a randomized, double-masked, and placebo-controlled trial.

Furthermore, the market's revenue growth is accelerated by the risk of potentially fatal renal disorders if cystinuria is left untreated. Additionally, the high rate of cystinuria recurrence after surgical interventions contributes to market expansion during the forecast period.

Cystinuria is a genetic metabolic condition characterized by elevated levels of undissolved cystine, along with related amino acids like arginine, lysine, and ornithine, in the urine. Excess cystine in the urine can form crystals and stones (calculi) in the kidneys, bladder, and urinary tract. Initial symptoms often include severe discomfort in the side of the abdomen or lower back, known as renal colic. Other potential symptoms include haematuria, urinary tract obstructions, and urinary tract infections. Recurrent episodes of cystinuria can ultimately result in kidney damage.

COVID -19 Impact on Cystinuria Market

In the initial stages of the pandemic, healthcare systems worldwide shifted their resources and focus towards managing COVID-19 cases. Non-urgent medical procedures and routine health check-ups were postponed or cancelled. This disruption in healthcare services might have delayed diagnosis and treatment for individuals with cystinuria. The global disruption in supply chains, including the pharmaceutical sector, posed challenges in manufacturing and distributing medications and medical supplies. Patients with cystinuria who depend on specific medications may have difficulty accessing necessary treatments.

Numerous clinical trials related to cystinuria treatments and research initiatives experienced delays or were temporarily halted due to the pandemic. This interruption could have postponed the development of novel therapies and treatment alternatives for cystinuria patients. The COVID-19 pandemic induced heightened anxiety and prompted changes in health-seeking behaviours among patients. Some individuals with cystinuria may have hesitated to seek medical attention or undergo elective medical procedures due to concerns about the risk of contracting the virus in healthcare settings. Funding allocation for rare diseases like cystinuria may have been redirected towards COVID-19 research and the development of vaccines, which could have impacted the availability of resources for cystinuria-related research and treatment.

Factors Driving the Cystinuria Market


Increasing prevalence of genetic disorders associated with SLC3A1 and SLC7A9 gene mutations

The global cystinuria market is expected to experience expansion throughout the forecast period, primarily due to the growing prevalence of genetic disorders associated with SLC3A1 and SLC7A9 gene mutations. According to the National Library of Medicine, the global and U.S. incidence rate is approximately 1 in 7,000 individuals, with men being twice as likely to be affected as women. Cystinuria is the most common hereditary cause of kidney stone disease, and the overall risk of renal damage or failure is substantial, reaching up to 70%. Additionally, a substantial portion of cystinuria patients, ranging from 20% to 40%, exhibits other urine chemical abnormalities linked to kidney stones, such as hypocitraturia (44%), hypercalciuria (19%), or hyperuricosuria (greater than 40%).

Moreover, the market is poised for revenue growth due to the notable recurrence rate of cystinuria following surgical interventions, with recurrence rates reaching nearly 45% in the absence of preventive medical care. However, the introduction of therapy reduces the typical three-year recurrence rate to 25%, further contributing to revenue growth expectations over the forecast period.

Another driver of market revenue growth is the increasing rate of cystinuria diagnoses, driven by heightened awareness of the potential severity of the condition, especially in younger patients. Timely and accurate diagnosis facilitates early detection, leading to more effective patient care. Consequently, there is a heightened demand for treatment options, with conventional therapies gradually being replaced by innovative drug delivery systems.

Furthermore, continued investments in the research and development of ground-breaking medications addressing the unmet needs of a substantial patient population are expected to propel the market's growth further.


The cystinuria market is expected to face limitations in revenue growth due to several factors. These include the relatively limited focus of pharmaceutical companies on the research and development (R&D) of treatments for rare diseases like cystinuria. Additionally, the market may be constrained by the absence of well-established and effective treatments, the high cost associated with therapy, and potential side effects linked to the medications used.

Furthermore, the scarcity of knowledge and awareness surrounding this rare condition can act as a hindrance, as can the absence of comprehensive reimbursement policies in developing countries. The market's revenue growth may also be impeded by the limited availability of effective treatments and the substantial costs of conducting research and development for cystinuria-related therapies.

Moreover, stringent regulations governing the approval of medications in the market are expected to pose challenges and potentially limit revenue growth in the cystinuria market.


Growing emphasis on innovative drug delivery systems

The global cystinuria market is poised for growth, primarily driven by a growing emphasis on innovative drug delivery systems. Furthermore, several factors are expected to propel the global market in the forecast period. These factors include the increasing prevalence of kidney stones resulting from cystinuria, rising cases of renal failure associated with cystinuria, and the incidence of cystinuria in both adults and newborns.

Moreover, the market's revenue growth is facilitated by advancements in healthcare infrastructure, adopting new technologies, and utilizing innovative technologies for early diagnosis.

Cystinuria Market Segmentation 

By Product Type

In the market for cystinuria, a condition characterized by the formation of cystine stones in the kidneys and urinary tract, segmentation based on product type categorizes the medications used for managing the condition. Medications in solid dosage forms come in various formulations such as tablets, capsules, or powders. These forms are designed for oral administration and include medications that help in reducing cystine stone formation, controlling symptoms, and managing the underlying causes of cystinuria. Solid dosage forms often provide a convenient and stable way to deliver medications, allowing for controlled and sustained release of the active ingredients. Liquid medications for cystinuria may include solutions, suspensions, or syrups. These forms are also for oral administration and may offer advantages such as quicker absorption, easy swallowing (especially for individuals who have difficulty swallowing solid forms), and the ability to tailor dosages for individual needs. Some patients might find it easier to adhere to a treatment regimen with solid dosage forms, while others might prefer or require the flexibility and ease of liquid forms. By offering both options, healthcare providers aim to ensure that patients have access to medications in forms that best suit their individual circumstances, thereby enhancing treatment adherence and effectiveness.

By Therapy Type

Gene therapy form of therapy involves using genetic material to treat or prevent a disease. In the context of cystinuria, gene therapy research aims to correct the underlying genetic mutations responsible for the condition. While still in experimental stages, gene therapy for cystinuria focuses on potentially modifying the genes responsible for cystine transport, with the goal of reducing cystine stone formation or addressing the root cause of the condition. Copper injections are a form of treatment for cystinuria that involves the use of a chelating agent called D-penicillamine, which binds with cystine to form a soluble complex that can be excreted in the urine.

This therapy aims to reduce the concentration of cystine in the urine, thereby lowering the risk of stone formation in the kidneys and urinary tract. These segmented approaches in the cystinuria market represent different strategies for managing the condition. While gene therapy holds promise for potentially addressing the genetic cause of cystinuria, copper injection therapy is a more established treatment that aims to manage the symptoms and decrease the likelihood of stone formation by altering the chemical composition of cystine in the body. Each approach represents a different level of development and application in the treatment landscape for cystinuria.

By Disease Type

The segmentation of cystinuria into these types is based on the specific genetic mutations that underlie the condition. Each type has a distinct genetic basis, although they all lead to increased levels of cystine in the urine, which can result in the formation of cystine stones. Understanding these different types is essential in diagnosing and treating cystinuria as it allows for more precise and tailored approaches to managing the condition based on its genetic origin. Type I cystinuria is characterized by a defect in the SLC3A1 gene, which encodes for a protein involved in the transport of cystine in the kidneys. This defect affects the reabsorption of cystine, leading to increased levels of cystine in the urine, subsequently contributing to stone formation.

Type II cystinuria involves mutations in the SLC7A9 gene. This gene is responsible for encoding a different protein involved in the transport of cystine. Mutations in SLC7A9 also disrupt the reabsorption of cystine in the kidneys, leading to elevated cystine levels in the urine and an increased risk of stone formation. Type III cystinuria is a much rarer form, and it's typically considered a milder variant of the condition. It's associated with mutations in genes other than SLC3A1 or SLC7A9, which still impact the transportation and reabsorption of cystine in the kidneys, albeit through different genetic mechanisms.

By Route of Administration

By route of administration segmentation based on administration routes categorizes the various treatment methods available for managing the condition. Oral treatments for cystinuria involve medications taken by mouth, typically in the form of tablets, capsules, or powders. These medications aim to modify the composition of urine, reduce cystine levels, and prevent stone formation in the kidneys and urinary tract. They often work by altering the way cystine behaves in the body or by enhancing its solubility to decrease the risk of stone development. Parenteral treatments are administered through routes other than the digestive tract, typically via injections or intravenous infusions.

In the context of cystinuria, this could involve therapies like intravenous infusions of chelating agents that bind to cystine, allowing it to be excreted in the urine, thus reducing the risk of stone formation. While less common than oral treatments, parenteral therapies may be utilized in cases where oral medications are not effective or well-tolerated. Others segment may encompass alternative or emerging administration methods not classified under oral or parenteral routes. It could include experimental treatments, advanced drug delivery systems, or non-traditional administration routes being explored for cystinuria management.

By Treatment Type

The segmentation analysis of the treatment type segment is as Captopril, Tiopronin, Penicillamine and Others.

The Penicillamine segment is projected to command a significant market share during the forecast period. As a first-generation chelating agent, Penicillamine combines with cystine to create a soluble disulfide complex. This action helps prevent the formation of stones and may potentially dissolve pre-existing cystine stones. In May 2019, Amerigen Pharmaceuticals Limited obtained approval from the U.S. FDA for Penicillamine Capsules USP 250 mg.  

Due to strong demand, the drugs segment featuring tiopronin accounted for ~50% of the market share in the cystinuria treatment market.

In 2022, the tiopronin category within the drugs segment emerged as the market leader, and it is expected to continue its dominance throughout the forecast period. This sustained leadership is attributed to the global growth in cystinuria treatment. As a prescribed thiol medication, Tiopronin is crucial in treating severe homozygous cystinuria. Patients affected by cystinuria tend to excrete significant amounts of cystine in their urine, making them prone to kidney stone formation.

By End-User

The segment segmentation analysis of the end users is as hospitals, online pharmacies, retail pharmacies and others.

In 2022, the retail pharmacies held the largest portion of the global cystinuria treatment market. Nevertheless, the online pharmacies segment is anticipated to experience rapid growth between 2023 and 2032. This surge can be attributed to the increasing preference among consumers for acquiring prescribed medications through online pharmacies and the consistent adoption of this distribution channel.

By Distribution Channel

Hospital pharmacies are part of healthcare facilities and provide medications and specialized care primarily to inpatients and outpatients receiving treatment for cystinuria within a hospital setting. They dispense medications prescribed by healthcare providers and ensure that patients have access to necessary treatments during their hospital stays or visits. Retail pharmacies are more commonly found in community settings, such as drugstores or standalone pharmacy outlets. These pharmacies provide medications and prescriptions for various health conditions, including cystinuria.

They serve as accessible points for individuals to fill their prescriptions, get refills, and obtain information about managing the condition. With the rise of digital healthcare services, online pharmacies offer a convenient way for individuals to access medications for cystinuria. These platforms allow patients to order medications online and have them delivered to their homes. Online pharmacies provide an accessible option for those who prefer remote access to their prescriptions. Others category might include specialty or compound pharmacies that offer personalized formulations or specific medication preparations for cystinuria. It might also encompass non-profit organizations providing support, resources, and advocacy for individuals affected by cystinuria.

By Regions

By region, the market is divided into North America, Europe, APAC, and Others. Others are further divided into the Middle East and South America.

ria in the United States is estimated at approximately one in every 7,000 individuals, as reported by the National Organization for Rare Disease. Additionally, efforts to raise awareness about the disease within the local population contribute to this dominance. Revenue growth in the global cystinuria disease therapeutics market during the forecast period is expected to be further driven by an increasing number of patients experiencing kidney stones, heightened healthcare expenditures, technological advancements, innovations in product development, and the attainment of FDA approvals for a diverse range of product portfolios.

Several key factors, including an increase in cystinuria cases, a surge in the utilization and awareness of cystinuria treatments and procedures, and ongoing technological advancements, are propelling the growth of the cystinuria treatment market in North America. Cystinuria, affecting roughly 1 in 15,000 individuals in the United States, with about 1 in 170 people being heterozygote carriers, constitutes approximately 1% of all cases of renal stones.

Conversely, the Asia Pacific region is projected to experience the most rapid growth, primarily driven by elevated demand for early diagnosis and technological advancements, along with government initiatives and support funding. Developing nations in this region, such as South Korea, China, and India, prioritize research and development efforts and the creation of novel products. Additionally, market expansion in these countries is anticipated to be propelled by healthcare reforms, including initiatives to enhance healthcare accessibility, affordability, and quality. This is exemplified by ongoing changes to improve healthcare systems in countries like China and India.

Cystinuria Market Competitive Landscape

The competitive landscape of the cystinuria treatment market involves assessing the competitive landscape to understand the strengths, weaknesses, opportunities, and threats. Cystinuria treatment providers are investing in the research development of innovative and more effective treatments. Prominent players on the global stage in the cystinuria treatment market encompass names such as Travere Therapeutics, Inc., Dr Reddy's Laboratories Ltd., Advicenne, Teva Pharmaceutical Industries Ltd., ANI Pharmaceuticals, Inc., Viatris Inc., AdvaCare Pharma, Camber Pharmaceuticals, Inc., Bausch Health Companies Inc., and Panacea Biotec, among other industry leaders.

Recent Developments 

  • November 28, 2022 - Lupin Limited, an established pharmaceutical company, a wholly-owned Brazilian subsidiary, MedQumica Indstria Farmacêutica, has entered into a binding agreement to acquire all rights to nine drugs from BL Indstria tica Ltd., a division of Bausch Health Companies Inc.
  • May 17, 2021 - Teva Pharmaceuticals, a subsidiary of Teva Pharmaceutical Industries Ltd., introduced the first generic version of THIOLA (tiopronin) tablets in the United States. These tablets are intended for use alongside increased fluid intake, alkali, or dietary modifications to prevent the formation of cystine kidney stones in adults and pediatric patients over 9 years old who have severe homozygous cystinuria and do not respond adequately to these treatments alone.
  • March 2, 2021, Lupin Limited, a pharmaceutical industry giant, launched Penicillamine Tablets USP, 250 mg, following FDA (United States Food and Drug Administration) approval. These tablets, the generic equivalent of Depen Tablets, 250 mg by Mylan Specialty, L.P., treat significant, active rheumatoid arthritis when conventional medications have proven ineffective. They are also employed in managing conditions such as Wilson's disease and cystinuria.

Table of Contents

  1. Executive summary
    • MARKET Definitions
  3. research methodology
  4. market outlook
    • Key Trends
    • COVID- 19 IMPACT ON GLOBAL Cystinuria Market
      • Drivers
        • Rising awareness among both patients and healthcare providers about cystinuria
        • Improved diagnostic techniques, such as genetic testing and imaging modalities like CT scans
        • Increasing prevalence of genetic disorders associated with SLC3A1 and SLC7A9 gene mutations
      • Restraints
        • Limited availability of effective treatment options
        • High cost of Cystinuria treatment
      • Opportunities
        • Ongoing research and development efforts in investments
        • Development of Point-of-Care Diagnostics
      • Challenges
        • Cystinuria is classified as a rare disease
        • Cystinuria patients remain at risk of stone recurrence
      • PriCing Analysis
        • Threat of new entrants
        • Threat of substitute
        • Bargaining power of suppliers
        • Bargaining power of buyers
        • Intensity of competitive rivalry
  1. GLOBAL Cystinuria Market, By product, MARKET ESTIMATION AND FORECAST, 2019-2032), value (USD million)
    • Solid Dosage Form
      • Tablets
      • Capsules
    • Liquid Dosage Form
      • Injectable
      • Others
  1. GLOBAL Cystinuria Market, By THERAPY TYpe, MARKET ESTIMATION AND FORECAST, 2019-2032), value (USD million)
    • Gene Therapy
    • copper injection therapy
  2. GLOBAL Cystinuria Market, BY disease Type, MARKET ESTIMATION AND FORECAST, 2019-2032, value (USD million)
    • Type I cystinuria
    • Type II cystinuria
    • Type III cystinuria
  3. GLOBAL Cystinuria Market, BY route of administration, MARKET ESTIMATION AND FORECAST, 2019-2032, value (USD million)
    • oral
    • parenteral
    • OTHERS
  4. GLOBAL Cystinuria Market, BY treatment, MARKET ESTIMATION AND FORECAST, 2019-2032, value (USD million)
    • captopril
    • tiopronin
    • penicillamine
    • other
  5. GLOBAL Cystinuria Market, BY end-users, MARKET ESTIMATION AND FORECAST, 2019-2032, value (USD million)
    • hospitals
    • online pharmacies
    • retail pharmacies
    • others
  6. GLOBAL Cystinuria Market, BY distribution channel, MARKET ESTIMATION AND FORECAST, 2019-2032, value (USD million)
    • Hospital Pharmacy
    • Retail Pharmacy
    • Online Pharmacy
    • Others
  7. GLOBAL Cystinuria Market, REGIONAL ANALYSIS MARKET ESTIMATION AND FORECAST, 2019-2032, value (USD million)
    • NORTH AMERICA Cystinuria Market
      • North America Cystinuria Market, By Country
        • US
        • Canada
        • Mexico
      • North America Cystinuria Market, By Product
      • North America Cystinuria Market, By Therapy Type
      • North America Cystinuria Market, By Disease Type
      • North America Cystinuria Market, By Route of Administration
      • North America Cystinuria Market, By Treatment
      • North America Cystinuria Market, By End-Users
      • North America Cystinuria Market, By Distribution Channel
    • EUROPE Cystinuria Market
      • Europe Cystinuria Market, By Country
        • Germany
        • UK
        • France
        • Italy
        • Spain
        • Russia
        • Rest of Europe
      • Europe Cystinuria Market, By Product
      • Europe Cystinuria Market, By Therapy Type
      • Europe Cystinuria Market, By Disease Type
      • Europe Cystinuria Market, By Route of Administration
      • Europe Cystinuria Market, By Treatment
      • Europe Cystinuria Market, By End-Users
      • Europe Cystinuria Market, By Distribution Channel
    • ASIA PACIFIC Cystinuria Market
      • Asia Pacific Cystinuria Market, By Country
        • China
        • Japan
        • India
        • Australia
        • Singapore
        • South Korea
        • Thailand
        • Indonesia
        • Malaysia
        • Philippines
        • Rest of Asia Pacific
      • Asia Pacific Cystinuria Market, By Product
      • Asia Pacific Cystinuria Market, By Therapy Type
      • Asia Pacific Cystinuria Market, By Disease Type
      • Asia Pacific Cystinuria Market, By Route of Administration
      • Asia Pacific Cystinuria Market, By Treatment
      • Asia Pacific Cystinuria Market, By End-Users
      • Asia Pacific Cystinuria Market, By Distribution Channel
    • MIDDLE EAST & AFRICA Cystinuria Market
      • Middle East & Africa Cystinuria Market, By Country
        • UAE
        • Saudi Arabia
        • South Africa
        • Rest of Middle East & Africa
      • Middle East & Africa Cystinuria Market, By Product
      • Middle East & Africa Cystinuria Market, By Therapy Type
      • Middle East & Africa Cystinuria Market, By Disease Type
      • Middle East & Africa Cystinuria Market, By Route of Administration
      • Middle East & Africa Cystinuria Market, By Treatment
      • Middle East & Africa Cystinuria Market, By End-Users
      • Middle East & Africa Cystinuria Market, By Distribution Channel
    • SOUTH AMERICA Cystinuria Market
      • South America Cystinuria Market, By Country
        • Brazil
        • Argentina
        • Peru
        • Rest of South America
      • South America Cystinuria Market, By Product
      • South America Cystinuria Market, By Therapy Type
      • South America Cystinuria Market, By Disease Type
      • South America Cystinuria Market, By Route of Administration
      • South America Cystinuria Market, By Treatment
      • South America Cystinuria Market, By End-Users
      • South America Cystinuria Market, By Distribution Channel
  1. GLOBAL Cystinuria Market, COMPETITIVE Intelligence
    • competitor benchmarking
    • market share analysis, 2022 (%)
  2. GLOBAL Cystinuria Market, COMPANY ANALYSIS
    • Abbott loaboratories
      • Company Overview
        • Business outlook
        • Headquarter
        • Established/Incorporated
        • Headcounts/ Employee strength
      • Financial Overview
        • Sales/revenue
        • R&D expenditure
        • Business segment sales share
        • Regional sales share
      • Product Offerings
      • SWOT Analysis
      • Key Developments (Expansion/New Product Development/Agreement/Mergers & Acquisitions, Partnership/Investment/Etc.)
    • Advicenne
    • Aktis Pharma India Pvt Ltd.
    • Amerigen Pharmaceuticals Limited
    • Amneal Pharmaceuticals LLC
    • ANI Pharmaceuticals, Inc.
    • Astrazeneca
    • Aurobindo Pharma
    • Bausch Health Companies Inc.
    • Hoffmann-La Roche Ltd.
    • Incepta Pharmaceuticals Ltd.
    • Mission Pharmacal Company
    • Mylan N.V.
    • Pfizer Inc.
    • Revive Therapeutics Ltd.
    • Taro Pharmaceutical Industries Ltd.
    • Teva Pharmaceutical Industries Ltd.
    • Travere Therapeutics, Inc.
    • Zhejiang Huahai Pharmaceutical Co., Ltd.
    • others


*Financials and Details May Not be Included in Case of Privately Held Company

Global Cystinuria Market Segmentation

Cystinuria by Type: Market Size & Forecast 2023-2030

  • Cystinuria Type I
  • Cystinuria Type II
  • Cystinuria Type III

Cystinuria by Therapy Type: Market Size & Forecast 2023-2030

  • Medication
  • Gene Therapy
  • Dietary Management
  • Surgical Intervention

Cystinuria by Application: Market Size & Forecast 2023-2030

  • Stone Prevention
  • Pain Management
  • Management of Kidney Function
  • Management of Urinary Tract Infections
  • Management of Renal Insufficiency
  • Long-Term Management of Cystinuria

Cystinuria by End User: Market Size & Forecast 2023-2030

  • Hospitals
  • Clinics
  • Urologists
  • Nephrologists
  • Patients
  • Payers

Cystinuria by Geography: Market Size & Forecast 2023-2030

  • North America (USA, Canada, Mexico)
  • Europe (Germany, UK, France, Russia, Italy, Rest of Europe)
  • Asia-Pacific (China, Japan, South Korea, India, Southeast Asia, Rest of Asia-Pacific)
  • South America (Brazil, Argentina, Columbia, Rest of South America)
  • Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, South Africa, Rest of MEA)

Major Players:

  • Bausch Health Companies Inc.
  • Retrophin, Inc.
  • Recordati Rare Diseases Inc.
  • Mylan N.V.
  • Avadel Pharmaceuticals plc
  • Chiesi Farmaceutici S.p.A.
  • ANI Pharmaceuticals, Inc.
  • Cumberland Pharmaceuticals Inc.
  • Meda Pharmaceuticals Inc.
  • Roche Holding AG
  • Horizon Therapeutics plc
  • Valeant Pharmaceuticals International, Inc.
  • Endo Pharmaceuticals Inc.
  • GlaxoSmithKline plc
  • Novartis AG
  • Pfizer Inc.
  • Sanofi S.A.
  • Merck & Co., Inc.
  • Johnson & Johnson
  • Takeda Pharmaceutical Company Limited
  • Recordati S.p.A.
  • Kyowa Kirin Co., Ltd.
  • Vifor Pharma Group
  • Cystinosis Research Network

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