Nephropathic Cystinosis Treatment Market: Introduction

According to the report, the global nephropathic cystinosis treatment market was valued at ~US$ 215 Mn in 2018. It is expected to expand at a CAGR of ~2% during the forecast period from 2019 to 2027. Nephropathic cystinosis is an inherited condition in which the body accumulates the amino acid cystine (a building block of proteins) within the cells. Excess cystine forms crystals that can build up and damage cells. These crystals can negatively affect many systems in the body, especially the kidneys and eyes. Treatment options for nephropathic cystinosis include systemic depletion therapy, symptomatic therapy, and renal transplantation. Cysteamine is the only effective treatment drug used for cystine depletion and is available in immediate-release and delayed-release capsules. North America dominated the global nephropathic cystinosis treatment market in 2018 and it is expected to continue dominating by the end of 2027 in terms of revenue.

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Emergence of Patient Assistance and Helping Hand Programs for Nephropathic Cystinosis Patients Drive Nephropathic Cystinosis Treatment Market

The increase in public and private initiatives for funding for research & treatment of cystinosis and emergence of patient assistance and helping hand programs for nephropathic cystinosis patients are some of the major factors responsible for the growth of global nephropathic cystinosis treatment market. For instance, Horizon Therapeutics plc has run Horizon Patient Services program, which is a patient-support program that provides support for patients to manage cystinosis. Product approvals by regulatory bodies and dosage form modification for better patient compliance are also fuelling the growth of the global nephropathic cystinosis treatment market. Moreover, increase in awareness among people through various programs by different organizations is expected to boost the growth of the global nephropathic cystinosis treatment market.

High Demand for Therapy for Nephropathic Cystinosis Treatment

Based on treatment type, the global nephropathic cystinosis treatment market has been bifurcated into therapy and renal transplantation. The therapy segment dominated the global nephropathic cystinosis treatment market in 2018 and its share in global market in increasing. Better efficacy to cure symptoms, delayed renal transplantation, and novel therapy approaches being developed in recent times are some of the major factors responsible for the dominance of the therapy segment. The therapy segment is further sub-segmented into systemic depletion therapy and symptomatic therapy. Systemic depletion therapy is a more popular therapy due to being the only effective treatment option for nephropathic cystinosis.

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Specialty/Retail Pharmacies to be Promising Sales Channel

In terms of sales channel, the global nephropathic cystinosis treatment market has been segmented into hospitals, specialty/retail pharmacies, and others. The hospitals segment dominated the global market in 2018, due to higher patient admissions to hospitals for treatment & renal transplantation, assistance in reimbursement for treatment, and well-structured supply chain management. The specialty/retail pharmacies segment is expected to be a highly lucrative segment and grow at a high CAGR during the forecast period. This can be attributed to easy accessibility of symptomatic as well as cysteamine therapies at retail pharmacies and patient assistance programs by specialty pharmacies.

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North America to Dominate Global Market

On the basis of region, the global nephropathic cystinosis treatment market has been divided into five major regions: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America accounted for major share of the global nephropathic cystinosis treatment market in 2018, owing to comparatively higher diagnosed patient population for nephropathic cystinosis in the region, availability of U.S. FDA approved products, and research funding for new treatment approaches. For instance, as per Cystinosis Research Network (CRN), cystinosis affects an estimated 2,000 people worldwide with just over 600 of those diagnosed living in the U.S. Europe follows North America in terms of market share in 2018, due to higher reported cases of cystinosis, presence of major companies, higher funding for treatment & research of cystinosis, and increase in demand for treatment of nephropathic cystinosis in Europe.

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Competition Landscape

Horizon Therapeutics plc., Mylan N.V., Chiesi Farmaceutici SpA, and Recordati Rare Diseases are some of the leading players in the global nephropathic cystinosis treatment market. The global nephropathic cystinosis treatment market is fragmented in terms of number of players with entry of several players particularly in symptomatic therapy. Key players in the global market include Horizon Therapeutics plc, Mylan N.V., Chiesi Farmaceutici SpA, Recordati Rare Diseases, AVROBIO, Inc., Leadiant Biosciences, Inc., Eloxx Pharmaceuticals, and GlaxoSmithKline plc. New product launch with dosage form modification, merger & acquisition, and patient assistance programs provided by companies for financial & other supports are some of the major strategies adopted by major companies in the nephropathic cystinosis treatment market.

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