Inclination toward Multidisciplinary Approach for Nephropathic Cystinosis Treatment

For several decades, researchers, healthcare providers, drug developers, and other participants of the healthcare systems worldwide continue to develop new treatments and drugs for patients suffering from rare and chronic diseases that are complex in nature. A similar trend can be observed within the nephropathic cystinosis treatment market, as continual research and clinical trials bring forward new potential treatments and medications. At present, improving the overall quality of patient care suffering from nephropathic cystinosis remains the key area of focus for companies involved in the current market for nephropathic cystinosis.

Healthcare providers are increasingly inclined toward adopting the multidisciplinary approach for patients suffering from nephropathic cystinosis, which predominantly includes psychologists, nurses, physicians, social workers, and nutritionists. Over the past three decades, long-term prognosis for patients suffering from cystinosis has improved at a rapid pace due to the availability of cysteine-depleting drug cysteamine and notable advancements in kidney transplantation techniques. The advent of cysteamine for the treatment of nephropathic cystinosis has also played a key role in addressing the complications associated with extra-renal diseases. The treatment of nephropathic cystinosis varies in the developing and developed regions of the world. However, with growing investments and promising outcome from research, healthcare providers around the world are increasingly providing effective and optimum nephropathic cystinosis treatments. At the back of these factors the global nephropathic cystinosis treatment market is expected to attain a market value of ~US$ 263 Mn by the end of 2027.

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Advancements and Innovations in Monitoring of Nephropathic Cystinosis Treatment to Boost Market Growth

Cystinosis is deemed as a rare inheritable multisystem storage disease, and can be characterized by lysosomal cysteine accumulation and other crystal formation in the different tissues within the body. Due to the narrow therapeutic window, cysteamine treatment needs to be monitored closely. Conventional treatment monitoring techniques have proved to be impractical, complex, and require specialized laboratories. Participants operating in the current nephropathic cystinosis treatment market are currently focusing on the development of innovative strategies that are likely to streamline the monitoring of cystinosis. As per recent developments in this regard, macrophages are anticipated to play a key role in pathogenesis of cystinosis. Innovations and advancements in the monitoring of nephropathic cystinosis treatment are expected to come at a consistent pace during the forecast period due to which, the market for nephropathic cystinosis is expected to witness a considerable growth in the coming years.

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Market Participants Continue to Place Trust in Systematic Depletion Therapy

Within the nephropathic cystinosis treatment market, systematic depletion therapy and symptomatic therapy are the two main types of treatment. The adoption of systematic depletion therapy is expected to remain considerably higher than that of symptomatic therapy, as it has emerged as an ideal treatment type in the removal of cysteine from lysosomes, thereby preventing any sort of complications. Several short-term studies have also revealed that systematic depletion therapy have shown promising results in maintaining the growth rate and improving renal function stabilization. Moreover, as systematic depletion therapy leads to the reduction of intralisossomal cysteine and cysteamine stocks, prognosis improvement in these patients can be observed.

Companies Aim to Attain FDA Approval for New Products to Gain Advantage

Receiving the approval from the FDA is one of the most critical phases in the development of new treatment and drugs. Several companies operating in the nephropathic cystinosis treatment market are increasingly focusing on receiving the FDA approval for their products. For instance, in July 2019, Horizon Therapeutics announced that the company has received the green signal from the U.S. Food and Drug Administration for the development of PROCYSBI Delayed-release Oral Granules in Packets. While companies continue to launch new drugs for nephropathic cystinosis treatment and seek FDA approval for the same, several drugs in the pipeline are expected to enter the nephropathic cystinosis market in the coming years. Therapy and renal replacement will continue to provide optimum care for patients suffering from nephropathic cystinosis.

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Analysts’ Viewpoint

The global nephropathic cystinosis treatment market is expected to grow at a modest CAGR of ~2% during the forecast period. The market growth can be largely attributed to a range of factors, including soaring investments for research & development activities, advent of patient assistance, product approval from the FDA, and increasing awareness among individuals pertaining to the effectiveness of nephropathic cystinosis treatments. Companies operating in the current market landscape should ideally focus on the development of new drugs and treatment, and align their growth strategies in-sync with the current trends, regulatory guidelines, and more to gain an edge in the current nephropathic cystinosis treatment market.

Nephropathic Cystinosis Treatment Market: Overview

  • Nephropathic cystinosis is a rare form of disease. It is an inherited (autosomal recessive) lysosomal storage disorder caused by defective transport of the amino acid cystine out of lysosomes. The stored cystine is poorly soluble and crystallizes within the lysosomes of many cell types, leading to widespread tissue and organ damage.
  • Nephropathic cystinosis treatment comprises cysteamine depletion therapy with symptomatic treatment for the disease. At a severe stage, renal transplantation option is adopted.
  • Systemic depletion therapy by cysteamine is the most common and only effective option for direct effect on nephropathic cystinosis treatment
  • Moreover, symptomatic therapy is provided to cure major symptoms such as electrolytes in Fanconi syndrome in children and Indomethacin in water losses in patients with nephropathic cystinosis
  • Renal transplantation is usually required in patients with nephropathic cystinosis who are diagnosed late and do not begin cysteamine treatment at an early age, even patients who start cysteamine at an early age may eventually require transplantation. This may be because patients find it impossible to continue taking Cystagon four times a day at six-hour intervals for their entire lives.
  • Increase in public and private initiatives in funding for research & treatment of cystinosis is the major driving factor responsible for the growth of the global nephropathic cystinosis treatment market
  • For instance, in 2018, CRF awarded 16 new grants totaling US$ 2.8 Mn for cystinosis research and it funded 10 research grants in 2019 which totaled US$ 2.6 Mn
  • Emergence of patient assistance and helping hand programs for nephropathic cystinosis patients and rise in awareness regarding nephropathic cystinosis among people through various programs are some of the major factors responsible for the growth of the global nephropathic cystinosis treatment market
  • Moreover, product approval by regulatory bodies and dosage form modification for better patient compliance are also fueling the growth of the global nephropathic cystinosis treatment market

Market Segmentation: Nephropathic Cystinosis Treatment Market

  • Based on treatment type, the global nephropathic cystinosis treatment market has been bifurcated into therapy and renal transplantation
  • Therapy is further sub-segmented into systemic depletion therapy and symptomatic therapy
  • In terms of sales channel, the global nephropathic cystinosis treatment market has been divided into hospitals, specialty/retail pharmacies, and others
  • Each of the nephropathic cystinosis treatment market segments has been analyzed in detail for market trends, recent trends and developments, drivers, restraints, opportunities, and useful insights
  • The nephropathic cystinosis treatment market report provides current and future revenues (US$ Mn) for each of these segments for the period from 2017 to 2027, considering 2018 as the base year. The compound annual growth rate (% CAGR) has been provided for each segment and market from 2019 to 2027 along with market size estimations.

Regional Overview: Nephropathic Cystinosis Treatment Market

  • In terms of region, the global nephropathic cystinosis treatment market has been divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. The regions have been further divided into major countries and sub-regions.
  • North America is anticipated to be a highly lucrative market for the nephropathic cystinosis during the forecast period and dominated the market in 2018
  • The current and future nephropathic cystinosis treatment market sizes in terms of revenue (US$ Mn) of these regional markets and their major countries have been provided in the report for the period from 2017 to 2027, with their CAGRs for the period from 2019 to 2027
  • The study also offers a list of recommendations, highlights, and useful insights of the market which will help new companies willing to enter the market and existing companies to increase market shares, and help in the decision-making process

Major Players

  • The nephropathic cystinosis treatment market report concludes with the company profiles section that includes key information about the major players in the market
  • Key players analyzed in nephropathic cystinosis treatment market report are
    • Horizon Therapeutics plc
    • Mylan N.V.
    • Chiesi Farmaceutici SpA
    • Recordati Rare Diseases
    • AVROBIO, Inc.
    • Leadiant Biosciences, Inc.
    • Eloxx Pharmaceuticals
    • GlaxoSmithKline plc.
  • Each of these players has been profiled in the global nephropathic cystinosis treatment market report based on parameters such as company overview, financial overview, business strategies, product portfolio, business segments, and recent developments

Nephropathic Cystinosis Treatment Market – Scope of the Report

TMR’S report on the global nephropathic cystinosis treatment market studies past as well as current growth trends and opportunities to gain valuable insights of the same indicators for the said market during the forecast period from 2019 to 2027. The report provides the overall market revenue of the global nephropathic cystinosis treatment market for the period of 2017–2027, with 2018 as the base year and 2027 as the forecast year. The report also provides the compound annual growth rate (CAGR) for the global nephropathic cystinosis treatment market for the forecast period.

The report has been prepared after an extensive primary and secondary research. Primary research involves bulk of research efforts, wherein, analysts carry out interviews with key opinion leaders, industry leaders, and opinion makers. Extensive secondary research involves referring to key players’ product literature, annual reports, press releases, and relevant documents to understand the global nephropathic cystinosis treatment market.

Secondary research also includes Internet sources, statistical data from government agencies, websites, and trade associations. Analysts have employed a combination of top-down and bottom-up approach to study various phenomena in the global nephropathic cystinosis treatment market.

The report includes an elaborate executive summary, along with a snapshot of the growth behavior of various segments and sub-segments included in the scope of the study. Furthermore, the report sheds light on changing competitive dynamics in the global nephropathic cystinosis treatment market. These indices serve as valuable tools for existing market players as well as for entities interested in participating in the global nephropathic cystinosis treatment market.

The report delves into the competitive landscape of the global nephropathic cystinosis treatment market. Key players operating in the global nephropathic cystinosis treatment market have been identified, and each one of them has been profiled for their distinguishing business attributes. Company overview, financial standings, recent developments, and SWOTs are some of the attributes of players in the global nephropathic cystinosis treatment market that have been profiled in this report.

Key Questions Answered in Nephropathic Cystinosis Treatment Market Report

  • What is the sales/revenue generated by each treatment type and sales channel segment of the nephropathic cystinosis treatment market across all regions during the forecast period?
  • What are the opportunities in the global nephropathic cystinosis treatment market?
  • What are the major drivers, restraints, opportunities, and threats in the market?
  • Which region is likely to expand at the fastest CAGR during the forecast period?
  • Which nephropathic cystinosis treatment type and sales channel segments are expected to generate the highest revenue globally in 2027? Which segment is projected to expand at the highest CAGR during the forecast period?
  • What is the market share or position of different companies operating in the global market in 2018?

Nephropathic Cystinosis Treatment Market – Research Objectives and Research Approach

The comprehensive report on the global nephropathic cystinosis treatment market begins with an overview of the said market, followed by the scope and objectives of this study. Following this, the report provides detailed explanation of the objectives behind this study, key industrial developments in terms of merger, acquisition, strategic partnerships, regulatory scenario, pipeline analysis, and disease epidemiology have been provided.

For reading comprehensibility, the report has been compiled in a chapter-wise layout, with each section divided into small sections. The entire report comprises an exhaustive collection of graphs and tables appropriately interspersed in the entire compilation. Pictorial representation of actual and projected values of key segments is visually appealing to readers. This also allows comparison of the market shares of key segments in the past and at the end of the forecast period.

The report analyzes the global nephropathic cystinosis treatment market in terms of treatment type, sales channel, and region. Key segments under each criteria have been studied at length, and the market share for each of them at the end of 2027 has been provided herein. Such valuable insights enable market stakeholders in making informed business decisions for investment in the global nephropathic cystinosis treatment market.

Nephropathic Cystinosis Treatment Market – Segmentation

TMR’s study on the global nephropathic cystinosis treatment market includes information divided into three sections ? treatment type, sales channel, and region. Changing industry trends and other crucial market dynamics associated with these sections of the global nephropathic cystinosis treatment market have been discussed in detail.

Treatment Type

Therapy

  • Systemic Depletion Therapy
  • Symptomatic Therapy

Renal Transplantation

Sales Channel

Hospitals

Specialty/Retail Pharmacies

Others

Region

North America

Europe

Asia Pacific

Latin America

Middle East & Africa

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