水疱性角膜症市場 - 市場の洞察、疫学、市場予測：2032年

水疱性角膜症の病因の増加により、米国では水疱性角膜症の発生が増加しています。主な病因は、フックス内皮角膜ジストロフィー、白内障手術、緑内障手術などです。角膜内皮機能と上皮細胞再生の理解における進歩により、DSAEKやDMEKのような薬理学的で侵襲の少ない手術技術が開発されました。さらに、細隙灯検査や超音波生体顕微鏡検査などの高度な診断技術により、水疱性角膜症の診断が向上しています。現在の治療法は対症療法です。潤滑点眼薬、高浸透圧薬、抗緑内障薬、ステロイド薬、軟膏、包帯コンタクトレンズなどの薬剤が症状の緩和に使用されます。重度の場合、特に視力が著しく損なわれ、生活の質に影響が出る場合は、角膜移植（角膜形成術）が検討されます。水疱性角膜症の市場を理解する上で大きな懸念は、最近の疫学研究の欠如と、水疱性角膜症を日常的に管理する際に使用される介入を検証するエビデンスが乏しいことです。米国、欧州4ヶ国、英国、そして日本では、水疱性角膜症の診断ガイドラインや臨床ガイドラインは存在しないです。

2022年の水疱性角膜症の市場規模は、主要7ヶ国諸国の中で米国が最も大きく、約770万米ドルを占めました。2032年までに増加すると予想されています。角膜移植手術はしばしば行われていますが、拒絶反応、感染、移植片の失敗など固有のリスクがあり、高額な費用がかかります。さらに、角膜ドナーが不足しています。新たな治療法であるVYZNOVA（ネルテペンドセル）は、米国、EU、日本の水疱性角膜症市場にポジティブな変化をもたらすと思われます。この同種細胞療法はヒト角膜内皮細胞療法で、腔内投与用の溶液として製剤化され、2023年3月に日本で発売されます。

その他、Trefoil TherapeuticsのTTHX1114（NM141）、EmmecellsのEO2002、CellusionsのCLS001などが開発初期段階にあります。TTHX1114（NM141）は、FGF-1分子の半減期を増加させ、偽水疱性角膜症患者の角膜内皮細胞の増殖と移動を刺激するように設計された、線維芽細胞増殖因子-1タンパク質（FGF-1）の人工型です。

主要7ヶ国における水疱性角膜症の総市場規模は、2022年に約1,360万米ドルであり、予測期間中（2023年～2032年）に拡大すると予測されています。米国における水疱性角膜症の市場規模は、病因の増加や新たな治療法の発売により、CAGR 13.3%で増加します。2022年、米国における水疱性角膜症の標準治療別収益は約770万米ドルとなりましたが、新興治療薬の発売により予測期間中に減少すると予測されます。欧州4ヶ国および英国諸国の中で、2022年の水疱性角膜症の市場規模はドイツが最大で、スペインは最下位でした。ドイツは主要7ヶ国の中で2番目に大きな水疱性角膜症の市場規模を占め、2022年には欧州4ヶ国および英国市場の約27%のシェアを占めます。日本は主要7ヶ国の中で水疱性角膜症の第3位の市場を占め、2022年の収益は約110万米ドルで、予測期間中に増加する見込みです。

当レポートでは、主要7ヶ国における水疱性角膜症市場について調査し、市場の概要とともに、疫学、患者動向、新たな治療法、2032年までの市場規模予測、および医療のアンメットニーズなどを提供しています。

目次

第1章 重要な洞察

第2章 レポートのイントロダクション

第3章 水疱性角膜症市場概要

• 2019年の水疱性角膜症の市場シェア（%）分布
• 2032年の水疱性角膜症の市場シェア（%）分布

第4章 水疱性角膜症の疫学と市場の調査手法

第5章 水疱性角膜症のエグゼクティブサマリー

第6章 主要な出来事

第7章 疾患の背景と概要

• イントロダクション
• 角膜の状態
• 分類
  • 角膜症
  • 水疱性角膜症
• 病因
• 兆候と症状
• 危険因子
• 病態生理学
• 診断
• 治療
  • 治療的アプローチ
  • 角膜移植

第8章 患者動向

第9章 疫学と患者数

• 主な調査結果
• 仮定と根拠：主要7ヶ国
• 主要7ヶ国における水疱性角膜症の総症例数
• 米国
• 欧州4ヶ国と英国
• 日本

第10章 上市済薬剤

第11章 新興薬剤

第12章 水疱性角膜症：市場分析

• 主な調査結果
• 主要な市場予測の前提条件
• 市場の見通し
• 主要7ヶ国における水疱性角膜症の総市場規模
• 主要7ヶ国における治療法別の水疱性角膜症の総市場規模
• 米国における水疱性角膜症の市場規模
• 欧州4ヶ国および英国における水疱性角膜症の市場規模
• 日本における水疱性角膜症の市場規模

第13章 主要なオピニオンリーダーの見解

第14章 SWOT

第15章 アンメットニーズ

第16章 市場アクセスと償還

• 米国
• 欧州4ヶ国と英国
• 日本

第17章 付録

第18章 DelveInsightのサービス内容

第19章 免責事項

第20章 DelveInsightについて

List of Tables

• Table 1: Summary of Market and Epidemiology (2019-2032)
• Table 2: Key Events for Bullous Keratopathy
• Table 3: Total Cases of Bullous Keratopathy in the 7MM (2019-2032)
• Table 4: Cases of Major Etiologies of Bullous Keratopathy in the US (2019-2032)
• Table 5: Bullous Keratopathy Cases in Major Etiologies in the US (2019-2032)
• Table 6: Total Bullous Keratopathy Cases in the US (2019-2032)
• Table 7: Gender-specific Cases of Bullous Keratopathy in the US (2019-2032)
• Table 8: Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty in the US (2019-2032)
• Table 9: Cases of Major Etiologies of Bullous Keratopathy in EU4 and the UK (2019-2032)
• Table 10: Bullous Keratopathy Cases in Major Etiologies in EU4 and the UK (2019-2032)
• Table 11: Total Bullous Keratopathy Cases in EU4 and the UK (2019-2032)
• Table 12: Gender-specific Cases of Bullous Keratopathy in EU4 and the UK (2019-2032)
• Table 13: Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty in EU4 and the UK (2019-2032)
• Table 14: Cases of Major Etiologies of Bullous Keratopathy in Japan (2019-2032)
• Table 15: Bullous Keratopathy Cases in Major Etiologies in Japan (2019-2032)
• Table 16: Total Bullous Keratopathy Cases in Japan (2019-2032)
• Table 17: Gender-specific Cases of Bullous Keratopathy in Japan (2019-2032)
• Table 18: Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty in Japan (2019-2032)
• Table 19: Comparison of Emerging Drugs for Treatment
• Table 20: TTHX1114 (NM141), Clinical Trial Description, 2023
• Table 21: EO2002, Clinical Trial Description, 2023
• Table 22: CLS001, Clinical Trial Description, 2023
• Table 23: Key Market Forecast Assumptions for VYZNOVA (neltependocel)
• Table 24: Total Market Size of Bullous Keratopathy in the 7MM, in USD million (2019-2032)
• Table 25: Total Market Size of Bullous Keratopathy by Therapies in the 7MM, in USD million (2019-2032)
• Table 26: Total Market Size of Bullous Keratopathy in the US, in USD million (2019-2032)
• Table 27: The Market Size of Bullous Keratopathy by Therapies in the US, in USD million (2019-2032)
• Table 28: Total Market Size of Bullous Keratopathy in EU4 and the UK, in USD million (2019-2032)
• Table 29: The Market Size of Bullous Keratopathy by Therapies in EU4 and the UK, in USD million (2019-2032)
• Table 30: Total Market Size of Bullous Keratopathy in Japan, in USD million (2019-2032)
• Table 31: The Market Size of Bullous Keratopathy by Therapies in Japan, in USD million (2019-2032)

List of Figures

• Figure 1: Layers of Cornea
• Figure 2: Common Corneal Conditions That Require a Transplant
• Figure 3: Types of Keratopathy
• Figure 4: Etiology of PBK
• Figure 5: Symptoms of Bullous Keratopathy
• Figure 6: Difference Between a Healthy Cornea and a Bullous Keratopathic Cornea
• Figure 7: Overview of Bullous Keratopathy
• Figure 8: Patient Journey of Bullous Keratopathy
• Figure 9: Total Cases of Bullous Keratopathy in the 7MM (2019-2032)
• Figure 10: Cases of Major Etiologies of Bullous Keratopathy in the US (2019-2032)
• Figure 11: Bullous Keratopathy Cases in Major Etiologies in the US (2019-2032)
• Figure 12: Total Bullous Keratopathy Cases in the US (2019-2032)
• Figure 13: Gender-specific Cases of Bullous Keratopathy in the US (2019-2032)
• Figure 14: Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty in the US (2019-2032)
• Figure 15: Cases of Major Etiologies of Bullous Keratopathy in EU4 and the UK (2019-2032)
• Figure 16: Bullous Keratopathy Cases in Major Etiologies in EU4 and the UK (2019-2032)
• Figure 17: Total Bullous Keratopathy Cases in EU4 and the UK (2019-2032)
• Figure 18: Gender-specific Cases of Bullous Keratopathy in EU4 and the UK (2019-2032)
• Figure 19: Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty in EU4 and the UK (2019-2032)
• Figure 20: Cases of Major Etiologies of Bullous Keratopathy in Japan (2019-2032)
• Figure 21: Bullous Keratopathy Cases in Major Etiologies in Japan (2019-2032)
• Figure 22: Total Bullous Keratopathy Cases in Japan (2019-2032)
• Figure 23: Gender-specific Cases of Bullous Keratopathy in Japan (2019-2032)
• Figure 24: Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty in Japan (2019-2032)
• Figure 25: Total Market Size of Bullous Keratopathy in the 7MM, in USD million (2019-2032)
• Figure 26: Total Market Size of Bullous Keratopathy by Therapies in the 7MM, in USD million (2019-2032)
• Figure 27: Total Market Size of Bullous Keratopathy in the US, in USD million (2019-2032)
• Figure 28: The Market Size of Bullous Keratopathy by Therapies in the US, in USD million (2019-2032)
• Figure 29: Total Market Size of Bullous Keratopathy in EU4 and the UK, in USD million (2019-2032)
• Figure 30: The Market Size of Bullous Keratopathy by Therapies in EU4 and the UK, in USD million (2019-2032)
• Figure 31: Total Market Size of Bullous Keratopathy in Japan, in USD million (2019-2032)
• Figure 32: The Market Size of Bullous Keratopathy by Therapies in Japan, in USD million (2019-2032)
• Figure 33: SWOT Analysis of Bullous Keratopathy
• Figure 34: Unmet Needs
• Figure 35: Health Technology Assessment
• Figure 36: Reimbursement Process in Germany
• Figure 37: Reimbursement Process in France
• Figure 38: Reimbursement Process in Italy
• Figure 39: Reimbursement Process in Spain
• Figure 40: Reimbursement Process in the United Kingdom
• Figure 41: Reimbursement Process in Japan

Key Highlights:

• The occurrence of bullous keratopathy has been increasing in the US due to the increase in etiologies of bullous keratopathy. The major etiologies include Fuchs endothelial corneal dystrophy, cataract surgery, and glaucoma surgery.
• Advancements in understanding corneal endothelium function and epithelial cell regeneration have developed pharmacological and less invasive surgical techniques like DSAEK and DMEK. Further, advanced diagnostic techniques like slit-lamp examination and ultrasound biomicroscopy have improved bullous keratopathy diagnosis.
• The current treatment regime is symptomatic. Medications such as lubricating eye drops, hyperosmotic medicines, antiglaucoma, steroids, ointments, or bandage contact lenses are used to alleviate symptoms.
• In severe cases, especially where vision is significantly impaired and quality of life is affected, corneal transplantation (keratoplasty) is considered.
• The major concern in understanding the market for bullous keratopathy is a lack of recent epidemiology studies and a paucity of evidence to validate interventions used in daily managing bullous keratopathy. No diagnostic or clinical guidelines for bullous keratopathy are available in the US, EU4 and the UK, and Japan.
• Fusing various technologies like magnetic nanoparticles and iPS cells to develop novel treatments will facilitate the effective delivery and retention of therapeutic agents at the target site.
• In 2022, the market size of bullous keratopathy was highest in the US among the 7MM countries, accounting for approximately USD 7.7 million. It is expected to increase by 2032.
• Although corneal transplant surgeries are often used, they carry inherent risks, including rejection, infection, and graft failure, and are associated with high costs. Moreover, there is a lack of cornea donors.
• Emerging therapy VYZNOVA (neltependocel) will create a positive shift in the US, EU, and Japan markets of bullous keratopathy. This allogeneic cell therapy is a human corneal endothelial cell therapy formulated as a solution for the intracameral route of administration and launched in Japan in March 2023.
• Other assets, including Trefoil Therapeutics' TTHX1114 (NM141), Emmecells' EO2002, and Cellusions' CLS001, are in their early development phase.
• TTHX1114 (NM141) is an engineered form of fibroblast growth factor-1 protein (FGF-1) designed to increase the half-life of the FGF-1 molecule and to stimulate the proliferation and migration of corneal endothelial cells in pseudophakic bullous keratopathy patients.
• With no approved treatment in the US and EU, limited application of corneal transplant, and significant risk of rejections, there is a window of opportunity for pharma companies to develop pharmacological therapies.

DelveInsight's "Bullous Keratopathy - Market Insights, Epidemiology, and Market Forecast - 2032" report delivers an in-depth understanding of the bullous keratopathy historical and forecasted epidemiology as well as the market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The bullous keratopathy market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM bullous keratopathy market size from 2019 to 2032. The report also covers current bullous keratopathy treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2019-2032

Bullous Keratopathy Understanding and Treatment Algorithm

Bullous Keratopathy Overview

The term keratopathy comes from the root words Kera, meaning cornea, and pathy, meaning disease; therefore, keratopathy is the disease of the cornea with a vast array of underlying causes and mechanisms. Keratopathy can occur due to an eye condition or systemic conditions. There are several types of keratopathy, including bullous keratopathy.

Bullous keratopathy is a pathological condition in which small vesicles, or bullae, formation occurs in the cornea due to endothelial dysfunction. Initially, there is endothelial trauma, followed by progressive stromal and epithelial edema. The epithelial edema results in the formation of bullae, hence the name bullous keratopathy.

Bullous keratopathy is also called pseudophakic bullous keratopathy (PBK) or pseudophakic corneal edema (PCE) in certain cases because it specifically occurs in individuals who have undergone cataract surgery and have had an intraocular lens (IOL) implanted in their eye. The main characterization is the formation of fluid-filled blisters or bullae on the cornea, which can cause pain, blurred vision, and other visual disturbances. This condition typically occurs due to damage to the corneal endothelium, the innermost layer of cells that maintains the cornea's clarity and controls its fluid balance.

There are several causes of bullous keratopathy, such as Fuchs' endothelial dystrophy, corneal surgery, and certain types of eye surgeries, eye trauma, cataract, glaucoma surgery, or corneal transplantation. Studies suggest prolonged contact lens wear can also damage the corneal endothelium and result in bullous keratopathy.

Bullous Keratopathy Diagnosis

Diagnosis of bullous keratopathy typically involves a comprehensive eye examination, including visual acuity testing, slit-lamp examination, and evaluation of corneal thickness and clarity. In some cases, additional tests such as corneal topography or specular microscopy are required to assess the severity and extent of corneal damage.

Further details related to country-based variations are provided in the report…

Bullous Keratopathy Treatment

The treatment of bullous keratopathy aims to alleviate symptoms, improve visual function, and manage corneal edema. The treatment choice depends on the severity of the condition and the patient's circumstances. In the early stages, conservative therapies such as lubricating eye drops, hyperosmotic medicines, anti-glaucoma, steroids, ointments, or bandage contact lenses are used to reduce pain and protect the cornea. Furthermore, these also help draw out excess fluid from the cornea, reducing edema.

In severe cases, especially in cases where vision is significantly impaired, and quality of life is affected, corneal transplantation (keratoplasty) is considered. Different types of corneal transplantation, including full-thickness penetrating keratoplasty, anterior lamellar keratoplasty, descemet's stripping automated endothelial keratoplasty (DSAEK), or descemet's membrane endothelial keratoplasty (DMEK) are available. Aurion Biotech's VYZNOVA is the first approved allogeneic cell therapy for bullous keratopathy in Japan.

Bullous Keratopathy Epidemiology

As the market is derived using a patient-based model, the bullous keratopathy epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by cases of major etiologies of bullous keratopathy, bullous keratopathy cases in major etiologies, total bullous keratopathy cases, gender-specific cases of bullous keratopathy, and bullous keratopathy cases in corneal transplant/keratoplasty in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2019 to 2032.

• In 2022, the total bullous keratopathy cases in FECD were approximately 79,250 in the 7MM, which is expected to increase by 2032.
• Among the 7MM, the US accounted for the highest number of bullous keratopathy cases in the 7MM. Among total cases of bullous keratopathy, cataract surgery patients contributed to nearly 49.4% in the US, while Spain accounted for the least, making up 7.1% of the total bullous keratopathy cases in 2022.
• In EU4 and the UK, out of total glaucoma surgery patients, 5,389 were affected by bullous keratopathy in 2022. The bullous keratopathy cases in glaucoma surgery patients are expected to increase by 2032.
• In the US, there were around 77,896 cases of bullous keratopathy in 2022, and the cases are expected to increase by 2032.
• Among EU4 and the UK, Germany contributed the highest number of bullous keratopathy cases (nearly 25,692), followed by France, Italy, the UK, and Spain in 2022.
• In 2022, among the 7MM, Japan ranked fifth and contributed nearly 9% to the total bullous keratopathy cases.
• Of the total cases of bullous keratopathy in Japan, around 87% of the cases were contributed by cataract surgery, while nearly 10% and 2% of the cases were contributed by glaucoma surgery and Fuchs endothelial corneal dystrophy, respectively, in 2022.
• According to estimates based on DelveInsight's epidemiology model, bullous keratopathy affects more females than males. In EU4 and the UK, around 39% of males and 61% of females were affected by bullous keratopathy in 2022, and the cases are expected to increase during the forecast period.
• In Japan, approximately 7,371 males and 10,306 females were affected by bullous keratopathy in 2022; the cases are expected to decrease by 2032.
• Among the 7MM, the US accounted for the highest number of bullous keratopathy cases in corneal transplants. Of the total corneal transplant cases, around 28% were affected by bullous keratopathy in 2022.
• In Japan, there were 2,322 corneal transplant cases in 2022, of which approximately 55% were affected by bullous keratopathy.

Bullous Keratopathy Drug Chapters

The drug chapter segment of the bullous keratopathy report encloses a detailed analysis of bullous keratopathy, currently used drugs, and mid-stage (Phase II and Phase I) pipeline drugs. It also helps understand the bullous keratopathy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Emerging Drugs

TTHX1114 (NM141): Trefoil Therapeutics

TTHX1114 (NM141) is an engineered form of fibroblast growth factor-1 protein (FGF-1). The native FGF-1 is a potent cell proliferation and migration stimulator and has cell protective properties. The compound uniquely activates all seven forms of the FGF receptor, contributing to its potency; however, the naturally occurring FGF-1 molecule has an extremely short half-life. The engineered FGF-1, TTHX1114, is designed to increase the half-life of the FGF-1 molecule and to stimulate the proliferation and migration of corneal endothelial cells.

It is being developed as an intracameral injection that involves injecting a small amount of TTHX1114 into the anterior chamber of the eye (directly behind the cornea) using a very small needle. Further, a topical formulation of the product is also under development to treat multiple epithelial indications where corneal ulcerations are present.

The drug has completed a Phase I/II trial in patients with PBK. Phase II trials are ongoing to treat endothelial dystrophy and ulcerative conditions that affect the epithelial cell.

EO2002: Emmecell

EO2002 is a first-in-class, non-surgical, magnetic cell-based therapy that can modify disease, developed through exclusive magnetic cell delivery (MCD) nanoparticle platform. The MCD platform facilitates cell therapies' delivery, retention, and integration by leveraging magnetic nanoparticles to effectively localize and integrate cell therapies to the appropriate target tissue.

The drug is undergoing two Phase I trials to treat corneal edema in patients with PBK. A Phase I trial is ongoing in patients with post-cataract surgery.

Note: Detailed emerging therapies assessment will be provided in the final report.

Drug Class Insights

Bullous keratopathy is used to describe a specific clinical presentation of corneal pathology characterized by the presence of fluid-filled blisters because of corneal endothelial cell dysfunction. The corneal endothelium is a single layer of cells that maintains corneal transparency by regulating fluid balance. The common symptoms of bullous keratopathy include corneal edema, blurred or distorted vision, eye pain or discomfort, redness, light sensitivity, and the sensation of a foreign body in the eye. Diagnosis typically involves a comprehensive eye examination, including visual acuity testing, ultrasound biomicroscopy, slit-lamp examination, and corneal thickness and clarity evaluation. At present, therapeutic options for bullous keratopathy are symptomatic and insufficient.

Several drug classes are being used off-label to manage various symptoms experienced by bullous keratopathy patients. These include hyperosmotic agents, antiglaucoma, steroids, anti-inflammatories, and others. Further, ointments or bandage contact lenses are used to relieve pain and protect the cornea.

Hyperosmotic agents like topical sodium chloride 2% drops and 5% ointment. These agents help form a hypertonic tear film by imbibing water from the cornea. The drops are taken four times per day, and ointment is at bedtime to reduce early morning corneal edema due to the accumulation of fluid overnight.

Steroids reduce acute inflammation or postoperative uveitis immediately after cataract surgery. Before starting steroids, the cornea stains with fluorescein to rule out any epithelial defect or infectious keratitis.

Antiglaucoma drugs commonly employed are beta-blockers and alpha agonists; they reduce intraocular pressure, reducing corneal edema and thickness in the postoperative period. Miotics and prostaglandins usually aggravate the inflammation, thus not taken. Carbonic anhydrase inhibitors act as epitheliotoxic are used.

Bullous Keratopathy Market Outlook

The current treatment landscape of bullous keratopathy involves both pharmacological and surgical therapies. Medicinal interventions such as cell therapy, hypertonic saline drops and ointment (sodium chloride 5%), antibiotic, anti-inflammatories, antiglaucoma, lubricating drops, and other medications are used for symptomatic relief. However, medical management is only favorable in the early stages of the disease, and when it fails, surgery is considered.

Cornea transplant remains the gold standard of treatment for bullous keratopathy, which requires the replacement of damaged endothelium with a healthy endothelium from a donor to restore endothelial cells' normal structure and function; however, visual recovery takes some time. The graft size is usually 7-7.5 mm to avoid complications of small and large grafts, such as astigmatism and secondary glaucoma.

The approval of the first allogenic cell therapy globally marked a significant advancement in the treatment of corneal endothelial disease. In March 2023, PMDA approved VYZNOVA for treating bullous keratopathy of the cornea. The therapy addresses the need for non-surgical intervention and overcomes the donor cornea shortage by treating more than 100 eyes with fully differentiated CECs from a single donor. Healthy cells from a donor cornea are cultured in a novel, multi-step, proprietary, and patented process that produces off-the-shelf, allogeneic, fully differentiated CEC. Further, the endothelial cells are administrated intracamerally, where a repopulation of cells into a healthy monolayer occurs. Removing fluid from the cornea starts leading to the reduction of corneal edema.

Steroids are recommended to reduce acute inflammation or postoperative uveitis immediately after cataract surgery. However, before starting steroids, the cornea must always be stained with fluorescein to rule out any epithelial defect or infectious keratitis. Further, systemic L-cysteine facilitated corneal edema remission in cataract surgery postoperative period, thus advocating its concurrent use in patients developing bullous keratopathy. A 2015 study demonstrated an increased expression of several pro-inflammatory mediators at the protein level in the corneal epithelium in patients with pseudophakic corneal edema when treated with systemic L-cysteine.

Further, a new promising treatment, Rho-kinase inhibitors, has emerged for early managing endothelial decompensation. Ripasudil (0.4%) and netarsudil (0.02%) four times a day inhibit apoptosis and promote endothelial cell proliferation. A molecular study reveals that ROCK inhibitor increases cyclin D levels and suppresses phosphorylation of p27 by activating phosphatidylinositol 3-kinase signaling as these two factors are regulators of the G1/S progression. Ki67-positive proliferating cells are also increased, which suggests that ROCK inhibition promotes endothelial proliferation. ROCK inhibitor eye drops can promote the proliferation of the residual endothelium following corneal endothelial damage and increase the number of endothelial cells available for coverage, thereby reducing the risk of corneal decompensation.

Full-thickness cornea transplantation, also known as penetrating keratoplasty, was first performed in 1905 by Dr. Eduard Zirm. A few years later, in 1998, Dr. Gerrit Melles described a posterior lamellar keratoplasty (PLK), where only a select portion of the cornea was transplanted. The transplant paradigm was revolutionized when less invasive techniques, DMEK and DSAEK, were introduced in 2006.

While treatment options are available, shortcomings remain. The symptomatic treatment is limited to the early stages of the disease and does not address the underlying cause of bullous keratopathy or provide a long-term solution. Although corneal transplantation is considered standard for treatment, risks are associated with any transplant surgery.

The methods for generating corneal endothelial cells into numbers that could address the current tissue shortage and the possible strategies used to deliver them have now become therapeutic with the approval of VYZNOVA. Several other approaches for corneal endothelial regeneration have been identified in studies and are under development, including cell therapies, acellular graft substitutes, and pharmacological and genetic modulation of the corneal endothelium.

The current emerging pipeline is limited and in the early phase of development; VYZNOVA is the only drug expected to launch during the forecast period in the US and EU. With no approved drugs, the US and EU markets quench for effective non-surgical therapies.

The current market has been covered by the symptomatic treatment that includes different pharmacological agents used across the 7MM, which presents minor variations in the overall prescription pattern. Hyperosmotic sodium chloride and other agents are considered the standard of care in the forecast model.

Key players Trefoil Therapeutics (TTHX1114 [NM141]), Emmecell (EO20020, Cellusion (CLS001), and others are evaluating their lead candidates in the early stages of clinical development. They aim to investigate their products for the treatment of bullous keratopathy. Further, Aurion Biotechnologies' VYZNOVA (neltependocel) is approved in Japan, and the company plans to develop the drug in the US and EU as well.

• The total market size of bullous keratopathy in the 7MM was approximately USD 13.6 million in 2022 and is projected to increase during the forecast period (2023-2032).
• The market size of bullous keratopathy in the US will increase at a CAGR of 13.3% due to increasing etiologies of the disease and the launch of the emerging therapy.
• In 2022, the standard of care generated nearly USD 7.7 million in revenue in the US for bullous keratopathy, which is anticipated to decrease during the forecast period owing to the launch of emerging therapies.
• Among EU4 and the UK countries, Germany accounted for the maximum market size of bullous keratopathy in 2022, while Spain occupied the bottom of the ladder.
• Germany accounted for the second largest market of bullous keratopathy among the 7MM, taking around 27% share of EU4 and the UK market in 2022.
• Japan accounted for the third largest market of bullous keratopathy among the 7MM, with a revenue of approximately USD 1.1 million in 2022, expected to increase during the forecast period.
• In the US, VYZNOVA (neltependocel) is expected to enter by 2029, while in EU4 and the UK, it is estimated to launch by 2030.
• In Japan, VYZNOVA (neltependocel) was launched in 2023 and is expected to attain a peak in its seventh year.

Bullous Keratopathy Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2019-2032. For example, Aurion Biotechnologies' VYZNOVA (neltependocel), a cell therapy to treat corneal endothelial disease, with an anticipated entry by 2029 in the US, is predicted to have a medium uptake during the forecast period.

Further detailed analysis of emerging therapies drug uptake in the report…

Bullous Keratopathy Pipeline Development Activities

The report provides insights into therapeutic candidates in Phase II and Phase I. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for emerging therapies for bullous keratopathy.

KOL Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on the bullous keratopathy evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Medical/scientific writers, Medical Professionals, Professors, Directors, and Others.

DelveInsight's analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers like the University of Texas Health Science Center, the University of Connecticut Health Center, the University Eye Hospital in Tubingen, Metz-Thionville Regional Hospital Center, the University of Manchester and the Manchester Royal Eye Hospital, and the University of Tokyo Hospital were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or bullous keratopathy market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Physician's View

According to our primary research analysis, despite a lack of treatment guidelines, sodium chloride 5%, antibiotics, and anti-inflammatories are used to reduce stromal edema, infection, and pain, respectively. Pressure-lowering agents like beta-blockers and alpha agonists are considered a first-line treatment to decrease corneal edema and thickness in the postoperative setting, even if IOP is normal or only mildly elevated. There is a need for further advancements in treatment to bring pharmacological options. Nevertheless, the Japan PMDA has recently approved an allogeneic cell therapy, VYZNOVA. Further, researchers and clinicians are actively exploring alternative therapies, such as tissue engineering techniques and regenerative medicine approaches, to improve the outcomes for individuals with bullous keratopathy.

Qualitative Analysis

We perform Qualitative and Market Intelligence analysis using various approaches, such as SWOT Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Market Access and Reimbursement

Reimbursement of rare disease therapies can be limited due to lack of supporting policies and funding, challenges of high prices, lack of specific approaches to evaluating rare disease drugs given limited evidence, and payers' concerns about budget impact. The high cost of rare disease drugs usually has a limited effect on the budget due to the small number of eligible patients being prescribed the drug. The US FDA has approved several rare disease therapies in recent years. From a patient perspective, health insurance and payer coverage guidelines surrounding rare disease treatments restrict broad access to these treatments, leaving only a small number of patients who can bypass insurance and pay for products independently.

The report provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenarios, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report:

• The report covers a segment of key events, an executive summary, descriptive overview of bullous keratopathy, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment have been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of early-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the bullous keratopathy market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis, expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM bullous keratopathy market.

Bullous Keratopathy Report Insights

• Patient Population
• Therapeutic Approaches
• Bullous Keratopathy Pipeline Analysis
• Bullous Keratopathy Market Size and Trends
• Existing and Future Market Opportunity

Bullous Keratopathy Report Key Strengths

• 10 years Forecast
• The 7MM Coverage
• Bullous Keratopathy Epidemiology Segmentation
• Key Cross Competition
• Conjoint Analysis
• Drugs Uptake and Key Market Forecast Assumptions

Bullous Keratopathy Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT Analysis)

Key Questions

Market Insights

• What was the total market size of bullous keratopathy, the market size of bullous keratopathy by therapies, and market share (%) distribution in 2019, and what would it look like by 2032? What are the contributing factors for this growth?
• How will VYZNOVA (neltependocel) affect the treatment paradigm of bullous keratopathy?
• How will VYZNOVA (neltependocel) compete with other off-label symptomatic treatments?
• Which drug is going to be the largest contributor by 2032?
• What are the pricing variations among different geographies for off-label therapies?
• How would future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights

• What are the disease risk, burdens, and unmet needs of bullous keratopathy? What will be the growth opportunities across the 7MM concerning the patient population about bullous keratopathy?
• What is the historical and forecasted patient pool of bullous keratopathy in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan?
• Out of the countries mentioned above, which country would have the highest diagnosed bullous keratopathy diagnosed prevalent population during the forecast period (2023-2032)?
• What factors are contributing to the growth of bullous keratopathy cases?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies

• What are the current options to treat bullous keratopathy?
• How many companies are developing therapies for the treatment of bullous keratopathy?
• How many emerging therapies are in the early stage of development for treating bullous keratopathy?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What is the cost burden of current treatment on the patient?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the accessibility issues of approved therapy in the US?
• What is the 7MM historical and forecasted market of bullous keratopathy?

Reasons to Buy:

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the bullous keratopathy market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunity in varying geographies and the growth potential over the coming years.
• The distribution of historical and current patient share is based on real-world prescription data in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet need of the existing market so that the upcoming players can strengthen their development and launch strategy

Table of Contents

1. Key Insights

2. Report Introduction

3. Bullous Keratopathy Market Overview at a Glance

• 3.1. Market Share (%) Distribution of Bullous Keratopathy in 2019
• 3.2. Market Share (%) Distribution of Bullous Keratopathy in 2032

4. Methodology of Bullous Keratopathy Epidemiology and Market

5. Executive Summary of Bullous Keratopathy

6. Key Events

7. Disease Background and Overview

• 7.1. Introduction
• 7.2. Corneal Conditions
• 7.3. Classification
  • 7.3.1. Keratopathy
  • 7.3.2. Bullous Keratopathy
• 7.4. Etiology
• 7.5. Signs and Symptoms
• 7.6. Risk Factors
• 7.7. Pathophysiology
• 7.8. Diagnosis
• 7.9. Treatment
  • 7.9.1. Therapeutic Approaches
  • 7.9.2. Corneal Transplantation

8. Patient Journey

9. Epidemiology and Patient Population

• 9.1. Key Findings
• 9.2. Assumptions and Rationale: The 7MM
  • 9.2.1. Cases of Major Etiologies of Bullous Keratopathy
  • 9.2.2. Bullous Keratopathy Cases in Major Etiologies
  • 9.2.3. Gender-specific Cases of Bullous Keratopathy
  • 9.2.4. Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty
• 9.3. Total Cases of Bullous Keratopathy in the 7MM
• 9.4. The US
  • 9.4.1. Cases of Major Etiologies of Bullous Keratopathy in the US
  • 9.4.2. Bullous Keratopathy Cases in Major Etiologies in the US
  • 9.4.3. Total Bullous Keratopathy Cases in the US
  • 9.4.4. Gender-specific Cases of Bullous Keratopathy in the US
  • 9.4.5. Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty in the US
• 9.5. EU4 and the UK
  • 9.5.1. Cases of Major Etiologies of Bullous Keratopathy in EU4 and the UK
  • 9.5.2. Bullous Keratopathy Cases in Major Etiologies in EU4 and the UK
  • 9.5.3. Total Bullous Keratopathy Cases in EU4 and the UK
  • 9.5.4. Gender-specific Cases of Bullous Keratopathy in EU4 and the UK
  • 9.5.5. Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty in EU4 and the UK
• 9.6. Japan
  • 9.6.1. Cases of Major Etiologies of Bullous Keratopathy in Japan
  • 9.6.2. Bullous Keratopathy Cases in Major Etiologies in Japan
  • 9.6.3. Total Bullous Keratopathy Cases in Japan
  • 9.6.4. Gender-specific Cases of Bullous Keratopathy in Japan
  • 9.6.5. Bullous Keratopathy Cases in Corneal Transplant/Keratoplasty in Japan

10. Marketed Drugs

• 10.1. VYZNOVA (neltependocel): Aurion Biotechnologies
  • 10.1.1. Drug Description
  • 10.1.2. Regulatory Milestones
  • 10.1.3. Other Development Activities
  • 10.1.4. Clinical Development
  • 10.1.5. Safety and Efficacy
  • 10.1.6. Product Profile

11. Emerging Drugs

• 11.1. Key Cross Competition
• 11.2. TTHX1114 (NM141): Trefoil Therapeutics
  • 11.2.1. Drug Description
  • 11.2.2. Other Developmental Activity
  • 11.2.3. Clinical Development
  • 11.2.4. Clinical Trials Information
  • 11.2.5. Safety and Efficacy
  • 11.2.6. Product Profile
  • 11.2.7. Analysts' Views
• 11.3. EO2002: Emmecell
  • 11.3.1. Drug Description
  • 11.3.2. Other Developmental Activity
  • 11.3.3. Clinical Development
  • 11.3.4. Clinical Trials Information
  • 11.3.5. Safety and Efficacy
  • 11.3.6. Product Profile
  • 11.3.7. Analysts' Views
• 11.4. CLS001: Cellusion
  • 11.4.1. Drug Description
  • 11.4.2. Other Developmental Activity
  • 11.4.3. Clinical Development
  • 11.4.4. Clinical Trials Information
  • 11.4.5. Safety and Efficacy
  • 11.4.6. Product Profile
  • 11.4.7. Analysts' Views

12. Bullous keratopathy: Market Analysis

• 12.1. Key Findings
• 12.2. Key Market Forecast Assumptions
• 12.3. Market Outlook
• 12.4. Total Market Size of Bullous Keratopathy in the 7MM
• 12.5. Total Market Size of Bullous Keratopathy by Therapies in the 7MM
• 12.6. Market Size of Bullous Keratopathy in the US
  • 12.6.1. Total Market Size of Bullous Keratopathy
  • 12.6.2. The Market Size of Bullous Keratopathy by Therapies
• 12.7. Market Size of Bullous Keratopathy in EU4 and the UK
  • 12.7.1. Total Market Size of Bullous Keratopathy
  • 12.7.2. The Market Size of Bullous Keratopathy by Therapies
• 12.8. Market Size of Bullous Keratopathy in Japan
  • 12.8.1. Total Market Size of Bullous Keratopathy
  • 12.8.2. The Market Size of Bullous Keratopathy by Therapies

13. Key Opinion Leaders' Views

14. SWOT

15. Unmet Needs

16. Market Access and Reimbursement

• 16.1. The United States
  • 16.1.1. Centre for Medicare & Medicaid Services (CMS)
• 16.2. In EU4 and the UK
  • 16.2.1. Germany
  • 16.2.2. France
  • 16.2.3. Italy
  • 16.2.4. Spain
  • 16.2.5. The United Kingdom
• 16.3. Japan
  • 16.3.1. MHLW

17. Appendix

• 17.1. Bibliography
• 17.2. Acronyms and Abbreviations
• 17.3. Report Methodology

18. DelveInsight Capabilities

19. Disclaimer

20. About DelveInsight