表皮水疱症市場 - 市場の洞察、疫学、市場予測：2034年

主なハイライト

• 2023年、表皮水疱症の市場規模は主要7ヶ国の中で米国が最も大きく、約13億米ドルを占め、2034年までにさらに拡大する見込みです。
• 単純性表皮水疱症（EBS）は、表皮水疱症の中で最も重症度の低いタイプであり、EBS患者の多くは年齢を重ねるにつれて改善がみられます。一方、劣性ジストロフィー型表皮水疱症は最も重篤と考えられています。
• 型別症例のうち、2023年の有病率は30％以上がジストロフィー性表皮水疱症に認められ、接合型表皮水疱症はわずか5％でした。
• 現在、表皮水疱症患者に有効な治療法は限られています。治療と管理の選択肢は、創傷ケア、疼痛と痒みの管理、感染管理、栄養サポート、関連合併症の予防と治療などのサポートを提供することに主眼が置かれています。
• 幹細胞を用いた治療法は、特に以前は不治の病と考えられていた疾患に対して重要性を増しています。ABCB5+間葉系間質細胞（ABCB5+MSCs）は、その特別な免疫調節作用と抗炎症作用で注目されており、表皮水疱症を含む様々な慢性炎症性疾患に対する有望な治療選択肢となっています。
• 全体的な細胞治療開発という点では、日本は主要7ヶ国の中で他国よりやや先行しており、2018年12月には、表皮水疱症に対する最初の細胞治療、すなわちJapan Tissue Engineeringの製品であるJACE（ヒト表皮細胞シート）が承認された最初の国となっています。
• 表皮水疱症に対する承認された治療法の欠如は、表皮水疱症患者に対する重大なアンメット・ニーズを浮き彫りにしています。
• Krystalは、表皮水疱症の劣性型と優性型の両方に有効なVYJUVEKのFDA承認を獲得することにより、表皮水疱症の遺伝子治療競争においてリードを取っています。
• Abeonaもまた、COL7A1を体内に導入することでVYJUVEKと同様の効果を発揮する遺伝子治療EB-101から説得力のあるデータを得ています。クリスタルの開発プロセスはアベオナより1年先行しているが、2つの治療法の間には差し迫った競合があります。
• 承認された治療法がいくつかあるにもかかわらず、表皮水疱症患者のすべてのニーズを満たすにはまだ長い道のりがあり、治療選択肢を改善し、最終的に世界中の表皮水疱症患者の生活の質を高めるためには、さらなる研究開発が不可欠です。

当レポートは、米国、EU4ヶ国（ドイツ、スペイン、イタリア、フランス）、英国、日本における表皮水疱症、歴史的および予測疫学、表皮水疱症市場動向の詳細な理解を提供します。

現在の治療法、新薬、個々の治療法の市場シェア、2020年から2034年までの主要7ヶ国の表皮水疱症市場規模の現状と予測を提供しています。また、現在の表皮水疱症治療法/アルゴリズムやアンメットメディカルニーズも網羅しており、最良の機会を発掘し、市場の潜在力を評価します。

表皮水疱症は、極端な皮膚の脆弱性とわずかな摩擦や外傷に対する水疱形成を特徴とする遺伝性皮膚疾患です。この疾患は、皮膚の構造的完全性を維持する役割を担うタンパク質に影響を及ぼす遺伝子変異によって引き起こされます。表皮水疱症の重症度は様々で、軽症から生命を脅かすものまで、特定の亜型によって異なります。

表皮水疱症患者の症状は、頻繁な皮膚の水疱形成および創傷治癒の遅れのような症状の発現から始まります。皮膚の脆弱性および水疱の分布を評価するためにヘルスケア専門家による臨床検査が行われます。診断を確定し、特定の表皮水疱症サブタイプを分類するために、皮膚生検と遺伝子検査が行われます。診断後は、皮膚科医、創傷ケアの専門家、栄養士、理学療法士が参加する集学的管理計画が立案されます。表皮水疱症とともに生きる課題に対処するために、精神的サポートとカウンセリングが提供されます。皮膚の健康状態を観察し、合併症を予防するためには、長期的なケアと定期的な経過観察が必要です。医療資源や専門知識へのアクセスは、経過の期間や結果に影響を与えます。早期診断はタイムリーな介入と患者のQOL向上のために極めて重要です。

表皮水疱症の治療は、主に症状の管理と合併症の予防に重点を置きます。これには創傷ケアの実施、感染管理、疼痛管理、および適切な栄養補給が含まれます。重度の表皮水疱症に対しては、皮膚外傷を最小限に抑えるために、特殊なドレッシング材、包帯および保護衣が必要となります。また、手の変形などの合併症に対処するために外科的介入が必要となることもあります。

表皮水疱症は、かゆみを効果的に抑えるコルチコステロイドで治療することができます。しかし、局所または経口コルチコステロイドの長期使用は視床下部-下垂体軸を抑制する可能性があり、これは小児に多くみられます。

現在のところ、3つの治療法しか承認されていないです。したがって、現状では、効果的で的を絞った治療が、この疾患の治療における重要なアンメット・ニーズです。

• 米国は表皮水疱症の最大の有病者数に寄与し、2023年には主要7ヶ国の65％を占めました。一方、EU4ヶ国と英国、日本は2023年にそれぞれ約30％と5％を占めています。
• EU4ヶ国諸国および英国の中で、2023年の表皮水疱症症例数が最も多いのは英国、次いでドイツ、一方、症例数が最も少ないのはスペインです。
• 米国では2023年に単純性表皮水疱症が約18,000例、接合型表皮水疱症が約1,450例、ジストロフィー型表皮水疱症が約8,500例でした。
• 男女比には若干の差があり、男性の患者シェアが若干高いです。

上市済み薬剤

VYJUVEK（beremagene geperpavec）：Krystal Biotech

B-VECは、DEB創傷に直接塗布することで、COL7A1遺伝子を2コピー導入するようにデザインされた、非侵襲的、局所的、再利用可能な遺伝子治療薬です。2023年5月、VYJUVEK（beremagene geperpavec-svdt）が生後6ヵ月以上のDEB患者の治療薬として承認されました。VYJUVEKは史上初の再利用可能な遺伝子治療薬であり、劣性・優性を問わずDEBの治療薬としてFDAに承認された最初で唯一の医薬品です。

2023年11月、Krystal Biotechは、欧州医薬品庁（EMA）のヒト用医薬品委員会（CHMP）に提出した、ジストロフィー性表皮水疱症（DEB）治療薬VYJUVEKの販売承認申請（MAA）が有効であり、現在CHMPの審査中であることを発表しました。CHMPの見解は2024年後半に予想されています。一方、日本での承認は2025年初頭に見込まれています。

FILSUVEZ（オレオゲル-S10）：Chiesi Farmaceutici

FILSUVEZ（オレオゲル-S10／白樺トリテルペン／旧名AP101）は、白樺樹皮由来の白樺トリテルペンを含有する生薬製剤です。FILSUVEZは2022年6月21日にEU全域で有効な販売承認を取得しました。FILSUVEZゲルは、接合型表皮水疱症および異栄養性表皮水疱症に伴う表在性創傷の治療を適応とし、6カ月以上の患者を対象としています。2022年2月、Amryt社は、ジストロフィー型および接合型表皮水疱症の皮膚症状の治療を適応とするオレオゲル-S10の新薬承認申請（NDA）について、米国FDAから審査完了報告書（Complete Response Letter：CRL）を受領したと発表しました。FDAはAmrytに対し、表皮水疱症におけるオレオゲル-S10の有効性に関する追加的な確認証拠の提出を求めました。2023年4月、Chiesi FarmaceuticiがAmryt Pharmaの買収完了を発表しました。

2023年12月、米国FDAは接合型表皮水疱症（JEB）およびジストロフィー型表皮水疱症（DEB）の6カ月以上の患者における部分的な厚さの創傷の治療薬としてFILSUVEZ局所ゲルを承認。FILSUVEZは、JEBに伴う創傷の治療薬として初めて承認されました。

新薬

EB-101：Abeona Therapeutics

EB-101は、劣性遺伝性表皮水疱症の治療薬として開発中の自己細胞治療薬です。EB-101による治療は、遺伝子導入によりCOL7A1遺伝子を患者の皮膚細胞（ケラチノサイトおよびその前駆細胞）に導入し、その細胞を患者に移植します。EB-101は、正常なVII型コラーゲンの発現を可能にし、創傷治癒を促進する能力について研究されています。EB-101は、RDEBの治療薬として初めて承認される可能性があり、最も痛みを伴い衰弱させる大きな慢性創傷に対応する唯一の耐久性のある治療薬となります。

2022年11月、主要な第III相試験であるVIITAL試験において、創傷治癒と疼痛軽減の2つの主要評価項目が達成され、大規模な慢性劣性ジストロフィー性表皮水疱症において、統計学的に有意かつ臨床的に意義のある改善が認められました。2023年9月、当社はEB-101の承認を求めて米国FDAに生物製剤承認申請（BLA）を提出しました。この申請において、Abeonaは優先審査を申請しており、この優先審査が認められれば、FDAの審査期間は通常の審査では10ヶ月であるところ、BLAの申請受理から6ヶ月に短縮されます。

D-Fi（ダボセマジェン・オートフィセル）：Castle Creek Biosciences

D-FiはFCX-007（dabocemagene autoficel）としても知られ、進行性で壊滅的な痛みを伴う衰弱性表皮水疱症（DEB）を治療する自己遺伝子治療薬候補です。D-Fiは現在、劣性遺伝性表皮水疱症患者の慢性創傷の局所治療を目的とした第III相臨床開発段階にあります。臨床試験計画には、D-Fiの多施設共同、患者内無作為化、対照、非盲検試験が含まれ、第I／II相臨床試験で報告されたデータを基礎としています。

表皮水疱症に対する遺伝子療法は、FDAが承認した最初の局所遺伝子療法であるVYJUVEKの承認によって現実のものとなりつつあります。他の2つの遺伝子治療候補はアベオナとCastle Creek Biosciencesで、COL7A1を体内に導入することでVYJUVEKと同様の効果を発揮します。

VYJUVEKは、単純ヘルペスウイルス1型（HSV-1）を遺伝子治療ベクターとして使用した最初の製品です。遺伝子治療に使われる他の多くのウイルスとは異なり、単純ヘルペスウイルスは細胞に感染しても宿主のゲノムに組み込まれることはないです。これは、統合によって正常な遺伝子発現が阻害され、がんを引き起こす危険性がわずかにあるためです。

JACEは2018年に表皮水疱症に対する薬事承認を取得した最初の細胞療法です。幹細胞を用いた治療法は、特に以前は不治の病であった疾患に対して、ますます重要になってきています。ABCB5+間葉系間質細胞（ABCB5+MSC）は、その特別な免疫調節作用と抗炎症作用により、表皮水疱症を含む様々な慢性炎症性疾患に対する新たな有望な治療アプローチとなります。この細胞治療剤は、慢性静脈性創傷（CVU）において、幹細胞が体内の免疫系と局所的に相互作用し、その結果慢性創傷を閉鎖できることをすでに示しています。他の2つの細胞治療薬には、イシン・ファーマのフェーズIII製品であるISN001と、Holostem Terapie Avanzate社が開発したALLO-RV-LAMB3導入表皮幹細胞があります。

様々な変異に起因する表皮水疱症のサブタイプのいずれにも治療法はなく、現在の治療法は創傷と疼痛の管理に焦点を当てるのみです。したがって、新しい効果的な治療アプローチが早急に求められています。表皮水疱症の治療は、支持療法と創傷管理、水疱形成と感染の予防、疼痛と痒みの対症療法、合併症の予防、モニタリング、治療に重点を置いています。創傷ケアは依然として治療の基礎であり、疼痛を軽減し、水疱形成、瘢痕形成および感染を軽減および予防するために、患部への半包帯による保護包帯の使用が含まれます。ドレッシング材の交換や創傷ケアには時間と費用がかかり、患者や介護者にとって負担となります。劣性ジストロフィー性表皮水疱症の多くの患者にとって、創傷ケアは1日4時間以上必要です。包括的かつ生涯にわたるケアが必要なため、患者やヘルスケアシステムにとって臨床的・経済的負担は大きいです。

当レポートでは、主要7ヶ国における表皮水疱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、2034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 報告書のイントロダクション

第3章 表皮水疱症（EB）のエグゼクティブサマリー

第4章 主要な出来事

第5章 疫学と市場予測の調査手法

第6章 表皮水疱症市場概要

• 2020年の治療法別市場シェア（％）分布
• 2034年の治療法別市場シェア（％）分布

第7章 表皮水疱症（EB）：疾患の背景と概要

• イントロダクション
• 表皮水疱症の原因
• 表皮水疱症の兆候と症状
• 表皮水疱症の発症機序
• 表皮水疱症皮膚におけるかゆみの病態生理学
• 表皮水疱症の分類
• 表皮水疱症の遺伝的基盤
• 表皮水疱症の診断

第8章 表皮水疱症の治療と管理

• 水ぶくれの管理
• 皮膚と創傷管理

第9章 ガイドライン

• 診断ガイドライン

第10章 主要7ヶ国の疫学と患者人口

• 主な調査結果
• 仮定と根拠
• 主要7ヶ国における表皮水疱症の有病者数の合計
• 主要7ヶ国で診断された表皮水疱症の流行症例
• 米国
• EU4ヶ国と英国
• 日本

第11章 患者動向

第12章 上市済み薬剤

• 主な競合
• VYJUVEK（bermagene geperpavec）：Krystal Biotech
• FILSUVEZ（オレオゲル-S10）：Chiesi Farmaceutici（Amryt Pharma）
• JACE（ヒト表皮細胞シート）：Japan Tissue Engineering

第13章 新興薬剤

• 主な競合
• EB-101：Abeona Therapeutics
• D-Fi（dabocemagene autoficel）：Castle Creek Biosciences
• ABCB5+間葉系幹細胞（ABCB5+MSC）：RHEACELL
• ISN001：Ishin Pharma
• RV-LAMB3導入表皮幹細胞：Holostem Terapie Avanzate
• PTR-01：BridgeBio（Phoenix Tissue Repair）
• INM-755：InMed Pharmaceuticals
• レダセムチド：Shionogi
• ALLO-ASC-SHEET：Anterogen

第14章 表皮水疱症：主要7ヶ国分析

• 主な調査結果
• 表皮水疱症市場の展望
• 主要な市場予測の前提条件
• コンジョイント分析
• 主要7ヶ国における表皮水疱症の総市場規模
• 米国の市場規模
• EU4ヶ国と英国の市場規模
• 日本市場規模

第15章 アンメットニーズ

第16章 SWOT分析

第17章 KOLの見解

第18章 市場アクセスと償還

• 米国
• EU4ヶ国と英国
• 日本
• 表皮水疱症市場へのアクセスと償還

第19章 付録

第20章 DelveInsightのサービス内容

第21章 免責事項

List of Tables

• Table 1: Summary of Epidermolysis Bullosa Market and Epidemiology (2020-2034)
• Table 2: EBS Subtypes and Their Features
• Table 3: DEB Subtypes and Their Features
• Table 4: JEB Subtypes and Their Features
• Table 5: Recommendations for Laboratory Diagnosis of Epidermolysis Bullosa (EB)
• Table 6: Level of Evidence
• Table 7: Grades of Recommendation Made by the Guideline Panel
• Table 8: Total Prevalent Cases of Epidermolysis Bullosa in the 7MM (2020-2034)
• Table 9: Diagnosed Prevalent Cases of Epidermolysis Bullosa in the 7MM (2020-2034)
• Table 10: Total Prevalent Cases of Epidermolysis Bullosa in the US (2020-2034)
• Table 11: Diagnosed Prevalent Cases of Epidermolysis Bullosa in the US (2020-2034)
• Table 12: Gender-specific Cases of Epidermolysis Bullosa in the US (2020-2034)
• Table 13: Age-specific Cases of Epidermolysis Bullosa in the US (2020-2034)
• Table 14: Type-specific Cases of Epidermolysis Bullosa in the US (2020-2034)
• Table 15: Total Prevalent Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Table 16: Total Diagnosed Prevalent Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Table 17: Gender-specific Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Table 18: Age-specific Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Table 19: Type-specific Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Table 20: Total Prevalent Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Table 21: Diagnosed Prevalent Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Table 22: Gender-specific Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Table 23: Age-specific Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Table 24: Type-specific Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Table 25: Comparison of Marketed Drugs
• Table 26: VYJUVEK (beremagene geperpavec), Clinical Trial Description, 2024
• Table 27: Comparison of Emerging Drugs
• Table 28: EB-101, Clinical Trial Description, 2024
• Table 29: D-Fi (dabocemagene autoficel), Clinical Trial Description, 2024
• Table 30: Allogenic ABCB5-positive Stem Cells, Clinical Trial Description, 2024
• Table 31: ISN001, Clinical Trial Description, 2024
• Table 32: RV-LAMB3-transduced Epidermal Stem Cells, Clinical Trial Description, 2024
• Table 33: PTR-01, Clinical Trial Description, 2024
• Table 34: INM-755, Clinical Trial Description, 2024
• Table 35: Redasemtide, Clinical Trial Description, 2024
• Table 36: ALLO-ASC-SHEET, Clinical Trial Description, 2024
• Table 37: Key Market Forecast Assumption of Epidermolysis Bullosa in the US
• Table 38: Key Market Forecast Assumption of Epidermolysis Bullosa in EU4 and the UK
• Table 39: Key Market Forecast Assumption of Epidermolysis Bullosa in Japan
• Table 40: Market Size of Epidermolysis Bullosa in the 7MM, in USD million (2020-2034)
• Table 41: Market Size of Epidermolysis Bullosa in the US, in USD million (2020-2034)
• Table 42: Market Size of Epidermolysis Bullosa by Current and Emerging in the US, in USD million (2020-2034)
• Table 43: Market Size of Epidermolysis Bullosa in EU4 and the UK, in USD million (2020-2034)
• Table 44: Market Size of Epidermolysis Bullosa by Current and Emerging Therapies in EU4 and the UK, in USD million (2020-2034)
• Table 45: Market Size of Epidermolysis Bullosa in Japan, in USD million (2020-2034)
• Table 46: Market Size of Epidermolysis Bullosa by Current and Emerging in Japan, in USD million (2020-2034)

List of Figures

• Figure 1: Symptoms and Complications of Epidermolysis Bullosa Simplex
• Figure 2: Symptoms and Complications of Junctional Epidermolysis Bullosa
• Figure 3: Symptoms and Complications of Kindler Syndrome
• Figure 4: The Histological Section of Skin Showing the Different Layers of Skin and the Cell Types in These Layers
• Figure 5: The Mechanism of Epidermolysis Bullosa
• Figure 6: Method of Blister Lancing
• Figure 7: Total Prevalent Cases of Epidermolysis Bullosa in the 7MM (2020-2034)
• Figure 8: Diagnosed Prevalent Cases of Epidermolysis Bullosa in the 7MM (2020-2034)
• Figure 9: Total Prevalent Cases of Epidermolysis Bullosa in the US (2020-2034)
• Figure 10: Diagnosed Prevalent Cases of Epidermolysis Bullosa in the US (2020-2034)
• Figure 11: Gender-specific Cases of Epidermolysis Bullosa in the US (2020-2034)
• Figure 12: Age-specific Cases of Epidermolysis Bullosa in the US (2020-2034)
• Figure 13: Type-specific Cases of Epidermolysis Bullosa in the US (2020-2034)
• Figure 14: Total Prevalent Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Figure 15: Total Diagnosed Prevalent Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Figure 16: Gender-specific Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Figure 17: Age-specific Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Figure 18: Type-specific Cases of Epidermolysis Bullosa in EU4 and the UK (2020-2034)
• Figure 19: Total Prevalent Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Figure 20: Diagnosed Prevalent Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Figure 21: Gender-specific Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Figure 22: Age-specific Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Figure 23: Type-specific Cases of Epidermolysis Bullosa in Japan (2020-2034)
• Figure 24: Market Size of Epidermolysis Bullosa in the 7MM, in USD million (2020-2034)
• Figure 25: Market Size of Epidermolysis Bullosa in the US, in USD million (2020-2034)
• Figure 26: Market Size of Epidermolysis Bullosa in the US by Current and Emerging Therapies, in USD million (2020-2034)
• Figure 27: Market Size of Epidermolysis Bullosa in EU4 and the UK, in USD million (2020-2034)
• Figure 28: Market Size of Epidermolysis Bullosa by Current and Emerging Therapies in EU4 and the UK, in USD million (2020-2034)
• Figure 29: Market Size of Epidermolysis Bullosa in Japan, in USD million (2020-2034)
• Figure 30: Market Size of Epidermolysis Bullosa by Current and Emerging Therapies in Japan, in USD million (2020-2034)
• Figure 31: Unmet Needs
• Figure 32: Health Technology Assessment
• Figure 33: Reimbursement Process in Germany
• Figure 34: Reimbursement Process in France
• Figure 35: Reimbursement Process in Italy
• Figure 36: Reimbursement Process in Spain
• Figure 37: Reimbursement Process in the United Kingdom
• Figure 38: Reimbursement Process in Japan

Key Highlights:

• In 2023, the market size of epidermolysis bullosa was highest in the US among the 7MM, accounting for approximately USD 1,300 million, which is further expected to increase by 2034.
• Epidermolysis bullosa simplex (EBS) is the least severe type of epidermolysis bullosa, and many individuals with EBS may experience improvements as they age. On the other hand, recessive dystrophic epidermolysis bullosa is considered the most critical.
• Among the type-specific cases, more than 30% of prevalent cases were observed in dystrophic epidermolysis bullosa, and only 5% were found in junctional epidermolysis bullosa in 2023.
• There are limited effective therapies currently available for epidermolysis bullosa patients. Treatment and management options primarily focus on providing support, such as wound care, pain and itch management, infection control, nutritional support, and prevention and treatment of associated complications.
• Stem cell-based therapies are gaining significance, particularly for diseases that were previously considered incurable. ABCB5+ mesenchymal stromal cells (ABCB5+ MSCs) are noteworthy for their special immunomodulatory and anti-inflammatory properties, making them a promising therapeutic option for various chronic inflammatory diseases, including epidermolysis bullosa.
• In terms of overall cell therapy development, Japan is a bit ahead of other countries among the 7MM, and in December 2018, Japan became the first country where the first cell therapy for epidermolysis bullosa got approved i.e., JACE (human epidermal cell sheet) which is a product of Japan Tissue Engineering.
• Lack of approved therapies for epidermolysis bullosa, highlights the significant unmet need for epidermolysis bullosa patients.
• Krystal has taken the lead in the race for gene therapy treatments for epidermolysis bullosa by securing FDA approval for VYJUVEK, which is effective for both the recessive and dominant forms of the condition.
• Abeona has also generated compelling data from its gene therapy, EB-101, which operates similarly to VYJUVEK by introducing COL7A1 into the body. Although Krystal is 1-year ahead of Abeona in its development process, there is impending competition between the two therapies.
• Despite the several approved therapies, there is still a long way to go in meeting all the needs of epidermolysis bullosa patients, and further research and development are essential to improve treatment options and ultimately enhance the quality of life for individuals living with epidermolysis bullosa worldwide.

DelveInsight's "Epidermolysis Bullosa - Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of the Epidermolysis Bullosa, historical and forecasted epidemiology as well as the Epidermolysis Bullosa market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

The Epidermolysis Bullosa market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM Epidermolysis Bullosa market size from 2020 to 2034. The report also covers current Epidermolysis Bullosa treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's underlying potential.

Geography Covered:

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

Study Period: 2020-2034

Epidermolysis Bullosa Disease Understanding and Treatment Algorithm

Epidermolysis Bullosa Overview

Epidermolysis Bullosa is a genetic skin disorder characterized by extreme skin fragility and blistering in response to minimal friction or trauma. The condition is caused by genetic mutations that affect the proteins responsible for maintaining the structural integrity of the skin. The severity of Epidermolysis Bullosa can vary widely, ranging from mild to life-threatening, depending on the specific subtype.

Epidermolysis Bullosa Diagnosis

The patient journey of Epidermolysis Bullosa begins with the onset of symptoms, such as frequent skin blistering and slow wound healing. Concerned caregivers seek medical attention, leading to a clinical examination by a healthcare professional to assess skin fragility and blister distribution. Skin biopsy and genetic testing are performed to confirm the diagnosis and classify the specific Epidermolysis Bullosa subtype. Upon diagnosis, a multidisciplinary management plan is developed involving dermatologists, wound care specialists, nutritionists, and physiotherapists. Emotional support and counseling are provided to cope with the challenges of living with Epidermolysis Bullosa. Long-term care and regular follow-ups are necessary to monitor skin health and prevent complications. Access to medical resources and expertise can influence the journey's duration and outcomes. Early diagnosis is crucial for timely intervention and improving the patient's quality of life.

Epidermolysis Bullosa Treatment

Treatment for Epidermolysis Bullosa primarily focuses on symptom management and complication prevention. This includes implementing wound care, infection control, pain management, and providing appropriate nutritional support. For individuals with severe Epidermolysis Bullosa, specialized dressings, bandages, and protective clothing may be necessary to minimize skin trauma. Surgical intervention might also be required to address complications like hand deformities.

Epidermolysis bullosa can be treated with corticosteroids, which effectively reduce itching. However, extended use of topical or oral corticosteroids may suppress the hypothalamic-pituitary axis, which can be more common in children.

Currently, only three therapies have been approved; therefore, as per the current situation, effective and targeted treatment remains a significant unmet need to treat this disease.

Epidermolysis Bullosa Epidemiology

As the market is derived using a patient-based model, the Epidermolysis Bullosa epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of Epidermolysis Bullosa, total diagnosed prevalent cases of Epidermolysis Bullosa, gender-specific cases of Epidermolysis Bullosa, age-specific cases of Epidermolysis Bullosa, and type-specific cases of Epidermolysis Bullosa in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2020 to 2034. The total prevalent cases of Epidermolysis Bullosa in the 7MM comprised approximately 46,500 cases in 2023 and are projected to increase during the forecasted period.

• The United States contributed to the largest prevalent cases of Epidermolysis Bullosa, accounting for ~65% of the 7MM in 2023. Whereas EU4 and the UK, and Japan accounted for around 30% and ~5% of the total population share, respectively, in 2023.
• Among the EU4 countries and the UK, the United Kingdom accounted for the largest number of Epidermolysis Bullosa cases, followed by Germany, whereas Spain accounted for the lowest number of cases in 2023.
• According to DelveInsight estimates, in the United States, there were around 18,000, 1,450, and 8,500 cases of epidermolysis bullosa simplex, junctional epidermolysis bullosa, and dystrophic epidermolysis bullosa, respectively, in 2023.
• There is a slight difference in the ratio of males vs. females, with a patient share of males being slightly higher.

Epidermolysis Bullosa Drug Chapters

The drug chapter segment of the Epidermolysis Bullosa report encloses a detailed analysis of Epidermolysis Bullosa marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also helps understand the Epidermolysis Bullosa pivotal clinical trial details, recent and expected market approvals, patent details, advantages and disadvantages of each included drug, the latest news, and recent deals and collaborations.

Marketed Drug

VYJUVEK (beremagene geperpavec): Krystal Biotech

B-VEC is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. In May 2023, VYJUVEK (beremagene geperpavec-svdt) was approved for treating patients 6 months of age or older with DEB. VYJUVEK is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for treating DEB, both recessive and dominant; a healthcare professional can administer that in either a healthcare professional setting or in the home.

In November 2023, Krystal Biotech announced that the Company's Marketing Authorization Application (MAA) to the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) for VYJUVEK for the treatment of dystrophic epidermolysis bullosa (DEB) has been validated and is now under CHMP review. A CHMP opinion is anticipated in the second half of 2024. Whereas approval in Japan is expected in early 2025.

FILSUVEZ (oleogel-S10): Chiesi Farmaceutici

FILSUVEZ (oleogel-S10/birch triterpenes/formerly known as AP101) is an herbal medicinal product that contains birch triterpenes from birch bark. FILSUVEZ received a marketing authorization valid throughout the EU on 21 June 2022. FILSUVEZ gel is indicated for patients 6 months and older to treat superficial wounds associated with Junctional Epidermolysis Bullosa and Dystrophic Epidermolysis Bullosa. In February 2022, Amryt announced it received a Complete Response Letter (CRL) from the US FDA regarding its New Drug Application (NDA) for Oleogel-S10 for the treatment of the cutaneous manifestations of dystrophic and Junctional Epidermolysis Bullosa. The FDA asked Amryt to submit additional confirmatory evidence of effectiveness for Oleogel-S10 in epidermolysis bullosa. In April 2023, Chiesi Farmaceutici announced the completion of the acquisition of Amryt Pharma.

In December 2023, the US FDA approved FILSUVEZ topical gel for the treatment of partial thickness wounds in patients 6 months and older with Junctional Epidermolysis Bullosa (JEB) and Dystrophic Epidermolysis Bullosa (DEB). FILSUVEZ is the first approved treatment for wounds associated with JEB.

Emerging Drug

EB-101: Abeona Therapeutics

EB-101 is an autologous, engineered cell therapy currently being developed to treat Recessive Dystrophic Epidermolysis Bullosa. Treatment with EB-101 involves using gene transfer to deliver the COL7A1 gene into a patient's skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient. EB-101 is being investigated for its ability to enable normal Type VII collagen expression and to facilitate wound healing. EB-101 has the potential to be the first approved therapy for RDEB and the only durable treatment to address large chronic wounds, which are the most painful and debilitating.

In November 2022, the pivotal Phase III VIITAL study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic Recessive Dystrophic Epidermolysis Bullosa. In September 2023, the Company submitted a Biologics License Application (BLA) to the US FDA seeking approval of EB-101. As part of the submission, Abeona requested a Priority Review, which, if granted, would shorten the FDA's review period to six months from the filing acceptance of the BLA, instead of 10 months under standard review.

D-Fi (dabocemagene autoficel): Castle Creek Biosciences

D-Fi, also known as FCX-007 (dabocemagene autoficel), is an autologous gene therapy candidate to treat dystrophic epidermolysis bullosa (DEB), a progressive, devastatingly painful, and debilitating, rare genetic skin disorder. D-Fi is currently in Phase III clinical development for the localized treatment of chronic wounds in individuals with Recessive Dystrophic Epidermolysis Bullosa. The clinical study plan includes a multi-center, within-patient randomized, controlled, open-label study of D-Fi and builds on data reported from the Phase I/II clinical trial.

Drug Class Insights

Preclinical or clinical testing of gene therapies (gene replacement, gene editing, RNA-based therapy, natural gene therapy), cell-based therapies (fibroblasts, bone marrow transplantation, mesenchymal stromal cells, induced pluripotential stem cells), recombinant protein therapies, and small molecule and drug repurposing approaches are all being currently explored for epidermolysis bullosa.

Gene therapies for epidermolysis bullosa are becoming a reality with the approval of VYJUVEK, the first FDA-approved topical gene therapy. Two other gene therapy contenders are Abeona and Castle Creek Biosciences, which operate similarly to VYJUVEK by introducing COL7A1 into the body.

VYJUVEK is the first to use the herpes simplex virus type 1 (HSV-1) as a gene therapy vector. Unlike many other viruses used for gene therapy, the herpes simplex virus does not integrate into its host's genome when it infects a cell. This is an advantage because there is a slight risk that integration can disrupt normal gene expression and cause cancer.

JACE was the first cell therapy to get the regulatory nod for Epidermolysis Bullosa in 2018. Stem cell-based therapies are becoming increasingly important, especially for previously incurable diseases. Due to the special immunomodulatory and anti-inflammatory properties, ABCB5+ mesenchymal stromal cells (ABCB5+ MSCs) represent a new, promising therapeutic approach for various chronic inflammatory diseases, including epidermolysis bullosa. The cell therapy agent has already shown in chronic venous wounds (CVU) that the stem cells interact locally with the immune system in the body, and therefore chronic wounds can be closed. The two other cell therapies include ISN001, a Phase III product by Ishin Pharma, and ALLO- RV-LAMB3-transduced epidermal stem cells, developed by Holostem Terapie Avanzate.

Epidermolysis Bullosa Market Outlook

There is no cure for any of the subtypes of epidermolysis bullosa resulting from different mutations, and current therapy only focuses on managing wounds and pain. Novel effective therapeutic approaches are therefore urgently required. Management of epidermolysis bullosa focuses on supportive and wound care, prevention of blistering and infection, symptomatic relief of pain and itch, and prevention, monitoring, and treatment of complications. Wound care remains the cornerstone of treatment and includes the use of semi-occlusive, protective bandages to the affected area to decrease pain and reduce and prevent blistering, scarring, and infection. Dressing changes and wound care can be time-consuming and expensive, making them burdensome to patients and caregivers. For many patients with Recessive Dystrophic Epidermolysis Bullosa, wound care requires more than 4 h per day. Due to the comprehensive and lifelong care needed, the clinical and economic burden can be high for patients and healthcare systems.

In the race for gene therapy treatments for epidermolysis bullosa, Krystal has gained an advantage as it has already received FDA approval for VYJUVEK in both recessive and dominant forms of the condition. On the other hand, Abeona and Castle Creek Biosciences have pursued more conventional gene therapy approaches with their candidates, EB-101 and D-Fi, respectively, targeting Recessive Dystrophic Epidermolysis Bullosa.

Shionogi and Ishin Pharma are exclusively conducting their development efforts in Japan. Shionogi's Redasemtide is currently undergoing Phase II trials for DEB. The company is in discussions with the Pharmaceuticals and Medical Devices Agency (PMDA) to seek drug approval based on the results of the Phase II trial and the follow-up study.

Despite the challenges, the focus remains strong among epidermolysis bullosa specialists and researchers who are deeply engaged in researching new treatment modalities. Further investigation is crucial for this patient population, given the severe impact of epidermolysis bullosa on their quality of life and life expectancy, particularly when complicated by chronic wound infections or sepsis. The commitment to finding effective treatments for epidermolysis bullosa persists, emphasizing the urgency of continued research efforts.

As per DelveInsight's estimates, the potential drugs that can mark a significant change in the forecast period includes JACE, FILSUVEZ, VYJUVEK, PTR-01, RV-LAMB3-transduced epidermal stem cells, and others.

• The total market size of Epidermolysis Bullosa in the 7MM is approximately USD ~1,700 million in 2023 and is projected to increase during the forecast period (2024-2034).
• Among EU4 countries, Germany accounts for the maximum market size in 2023, while Spain occupies the bottom of the ladder.
• In Japan, the highest revenue among current therapies included supportive treatment, followed by JACE.
• Among the emerging therapies, VYJUVEK is expected generate the highest revenue in the 7MM.
• The pipeline of epidermolysis bullosa treatments includes several robust candidates that are poised to transform the market dynamics and address the unmet needs of patients with this condition.
• The long-awaited period for the United States was finally over with the recent approval of the first-ever topical gene therapy, VYJUVEK, in May 2023.

Epidermolysis Bullosa Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024-2034. An expected fast uptake of VYJUVEK in the US as Cheisi's product launched recently in the US after several hurdles, which might gave VYJUVEK some alone time in the US market. Even though we expect a Medium-fast uptake of Abeona's EB-101 and its launch in 2024, the label will only be restricted to RDEB patients, which is approximately 15% of the total EB patient pool comprising of severe patients. Two of the most potential upcoming therapies by Abeona and Castle Creek Biosciences are targeting the same patient pool and are expected to have stiff competition due to launch during the same period. Whereas, this remains quite different in Europe, where we expect FILSUVEZ to have an upper hand in terms of faster adoption and uptake due to first mover advantage and inclusion of a broader patient pool in its EMA label, even though FILSUVEZ has not been launched anywhere except in Germany as of July 2023.

Epidermolysis Bullosa Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III and Phase II. 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 Epidermolysis Bullosa emerging therapy.

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 Epidermolysis Bullosa evolving treatment landscape, patient reliance on conventional therapies, patient's therapy switching acceptability, and drug uptake, along with challenges related to accessibility, include Medical/scientific writers; Dystrophic Epidermolysis Bullosa Research Association of America; Dermatologist and Professors; National Epidermolysis Bullosa Registry, and Center for Blistering Diseases, St John's Institute of Dermatology, NHS Foundation Trust, Departments of Dermatology, and others.

Delveinsight's analysts connected with 30+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the Center for Blistering Diseases, Department of Allergology and Dermatology, Department of Dermatology, Medical Center, etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or Epidermolysis Bullosa market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. 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.

The analyst views analyze multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry.

In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in Epidermolysis Bullosa trials, wound healing/wound closure is one of the most important primary outcome measures.

Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials.

Market Access and Reimbursement

Reimbursement is a crucial factor that affects the drug's access to the market. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, many payment models are being considered by payers and other industry insiders. The payment models are based on clinical outcomes, annuity payments, and expanded risk pools. The Institute for Clinical and Economic Review estimates that the cumulative budget impact for gene and cell therapies alone could rise to USD 3 trillion in the US when only about 10% of eligible patients are treated with these therapies. There are various disease advocacy groups, such as DEBRA International, with nearly 50 national DEBRA and epidermolysis bullosa patient support groups that help patients with epidermolysis bullosa for insurance and reimbursement.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, 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 Epidermolysis Bullosa, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight into the epidemiology segments and forecasts, disease progression, and treatment guidelines has been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the Epidermolysis Bullosa 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 and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM Epidermolysis Bullosa market.

Epidermolysis Bullosa Report Insights

• Patient Population
• Therapeutic Approaches
• Epidermolysis Bullosa Pipeline Analysis
• Epidermolysis Bullosa Market Size and Trends
• Existing and future Market Opportunity

Epidermolysis Bullosa Report Key Strengths

• Eleven Years Forecast
• The 7MM Coverage
• Epidermolysis Bullosa Epidemiology Segmentation
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

Epidermolysis Bullosa Report Assessment

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

FAQs:

• What was the Epidermolysis Bullosa total market size, the market size by therapies, market share (%) distribution in 2020, and what would it look like in 2034? What are the contributing factors for this growth?
• What will be the impact of EB-101's expected approval in 2024?
• How will EB-101 and D-Fi compete with VYJUVEK once they get approved in 2024?
• Which class is going to be the largest contributor in 2034?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
• What are the disease risk, burdens, and unmet needs of Epidermolysis Bullosa? What will be the growth opportunities across the 7MM concerning the patient population of Epidermolysis Bullosa?
• What is the historical and forecasted Epidermolysis Bullosa patient pool in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
• Which type of Epidermolysis Bullosa is the largest contributor?
• Which gender has a higher prevalence of Epidermolysis bullosa?
• Which age group of Epidermolysis bullosa has a high patient share?
• What are the current options for the treatment of Epidermolysis Bullosa? What are the current guidelines for treating Epidermolysis Bullosa in the US and Europe?
• How many emerging therapies are in the mid-stage and late stage of development for the treatment of Epidermolysis Bullosa?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitation of existing therapies?
• What key designations have been granted for the emerging therapies for Epidermolysis Bullosa?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, current therapies? Focusing on the reimbursement policies.

Reasons to Buy:

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the Epidermolysis Bullosa market.
• Insights on patient burden/disease incidence, 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.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products 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 Analyst view section to provide visibility around leading classes.
• Highlights of Access and Reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• 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. Executive Summary of Epidermolysis Bullosa (EB)

4. Key Events

5. Epidemiology and Market Forecast Methodology

6. Epidermolysis Bullosa Market Overview at a Glance

• 6.1. Market Share (%) Distribution by Therapies in 2020
• 6.2. Market Share (%) Distribution by Therapies in 2034

7. Epidermolysis Bullosa (EB): Disease Background and Overview

• 7.1. Introduction
• 7.2. Causes of Epidermolysis Bullosa
• 7.3. Signs and Symptoms of Epidermolysis Bullosa
• 7.4. Pathogenesis of Epidermolysis Bullosa
• 7.5. Pathophysiology of Itch in Epidermolysis Bullosa Skin
• 7.6. Classification of Epidermolysis Bullosa
• 7.7. Genetic Bases of Epidermolysis Bullosa
• 7.8. Diagnosis of Epidermolysis Bullosa
  • 7.8.1. Types of Laboratory Referral
    • 7.8.1.1. Neonate With Skin Fragility
    • 7.8.1.2. Pediatric and Adult Patients With Skin Fragility
    • 7.8.1.3. Carrier Testing
    • 7.8.1.4. Prenatal Diagnosis
  • 7.8.2. Further Testing
    • 7.8.2.1. Skin Biopsy
    • 7.8.2.2. Molecular Testing
    • 7.8.2.3. Genetic Testing for Epidermolysis Bullosa
    • 7.8.2.3.1. Next-generation Sequencing (NGS) Targeted Gene Panel and Whole-exome Sequencing in Epidermolysis Bullosa
    • 7.8.2.3.2. Sanger Sequencing (SS)

8. Treatment and Management of Epidermolysis Bullosa

• 8.1. Management of Blisters
• 8.2. Skin and Wound Management
  • 8.2.1. Management of Epidermolysis Bullosa Simplex (EBS)
  • 8.2.2. Management of Junctional Epidermolysis Bullosa (JEB)
  • 8.2.3. Management of Dystrophic Epidermolysis Bullosa (DEB)
  • 8.2.4. Management of Kindler Syndrome

9. Guidelines

• 9.1. Diagnostic Guidelines
  • 9.1.1. Clinical Practice Guidelines for Epidermolysis Bullosa Laboratory Diagnosis
  • 9.1.2. Japanese Guidelines for Diagnosis and Treatment of Junctional and Dystrophic Epidermolysis Bullosa

10. Epidemiology and Patient Population of the 7MM

• 10.1. Key Findings
• 10.2. Assumption and Rationale
• 10.3. Total Prevalent Cases of Epidermolysis Bullosa in the 7MM
• 10.4. Diagnosed Prevalent Cases of Epidermolysis Bullosa in the 7MM
• 10.5. The United States
  • 10.5.1. Total Prevalent Cases of Epidermolysis Bullosa in the United States
  • 10.5.2. Diagnosed Prevalent Cases of Epidermolysis Bullosa in the United States
  • 10.5.3. Gender-specific Cases of Epidermolysis Bullosa in the United States
  • 10.5.4. Age-specific Cases of Epidermolysis Bullosa in the United States
  • 10.5.5. Type-specific Cases of Epidermolysis Bullosa in the United States
• 10.6. EU4 and the UK
  • 10.6.1. Total Prevalent Cases of Epidermolysis Bullosa in EU4 and the UK
  • 10.6.2. Diagnosed Prevalent Cases of Epidermolysis Bullosa in EU4 and the UK
  • 10.6.3. Gender-specific Cases of Epidermolysis Bullosa in EU4 and the UK
  • 10.6.4. Age-specific Cases of Epidermolysis Bullosa in EU4 and the UK
  • 10.6.5. Type-specific Cases of Epidermolysis Bullosa in EU4 and the UK
• 10.7. Japan
  • 10.7.1. Total Prevalent Cases of Epidermolysis Bullosa in Japan
  • 10.7.2. Diagnosed Prevalent Cases of Epidermolysis Bullosa in Japan
  • 10.7.3. Gender-specific Cases of Epidermolysis Bullosa in Japan
  • 10.7.4. Age-specific Cases of Epidermolysis Bullosa in Japan
  • 10.7.5. Type-specific Cases of Epidermolysis Bullosa in Japan

11. Patient Journey

12. Marketed Drugs

• 12.1. Key Competitors
• 12.2. VYJUVEK (beremagene geperpavec): Krystal Biotech
  • 12.2.1. Product Description
  • 12.2.2. Regulatory Milestones
  • 12.2.3. Other Developmental Activities
  • 12.2.4. Clinical Developmental Activities
    • 12.2.4.1. Clinical Trial Information
  • 12.2.5. Safety and Efficacy
  • 12.2.6. Product Profile
• 12.3. FILSUVEZ (oleogel-S10): Chiesi Farmaceutici (Amryt Pharma)
  • 12.3.1. Product Description
  • 12.3.2. Regulatory Milestone
  • 12.3.3. Other Development Activities
  • 12.3.4. Safety and Efficacy
  • 12.3.5. Product Profile
• 12.4. JACE (human epidermal cell sheet): Japan Tissue Engineering
  • 12.4.1. Product Description
  • 12.4.2. Regulatory Milestones
  • 12.4.3. Safety and Efficacy
  • 12.4.4. Product Profile

13. Emerging Drugs

• 13.1. Key Competitors
• 13.2. EB-101: Abeona Therapeutics
  • 13.2.1. Product Description
  • 13.2.2. Other Developmental Activities
  • 13.2.3. Clinical Developmental Activities
    • 13.2.3.1. Clinical Trial Information
  • 13.2.4. Safety and Efficacy
• 13.3. D-Fi (dabocemagene autoficel): Castle Creek Biosciences
  • 13.3.1. Product Description
  • 13.3.2. Other Developmental Activities
  • 13.3.3. Clinical Developmental Activities
    • 13.3.3.1. Clinical Trial Information
  • 13.3.4. Safety and Efficacy
• 13.4. ABCB5+ mesenchymal stem cells (ABCB5+ MSCs): RHEACELL
  • 13.4.1. Product Description
  • 13.4.2. Other Developmental Activities
  • 13.4.3. Clinical Developmental Activities
    • 13.4.3.1. Clinical Trial Information
  • 13.4.4. Safety and Efficacy
• 13.5. ISN001: Ishin Pharma
  • 13.5.1. Product Description
  • 13.5.2. Other Developmental Activities
  • 13.5.3. Clinical Developmental Activities
    • 13.5.3.1. Clinical Trial Information
• 13.6. RV-LAMB3-transduced epidermal stem cells: Holostem Terapie Avanzate
  • 13.6.1. Product Description
  • 13.6.2. Other Developmental Activities
  • 13.6.3. Clinical Developmental Activities
    • 13.6.3.1. Clinical Trial Information
• 13.7. PTR-01: BridgeBio (Phoenix Tissue Repair)
  • 13.7.1. Product Description
  • 13.7.2. Other Development Activities
  • 13.7.3. Clinical Development
    • 13.7.3.1. Clinical Trials Information
  • 13.7.4. Safety and Efficacy
• 13.8. INM-755: InMed Pharmaceuticals
  • 13.8.1. Product Description
  • 13.8.2. Clinical Development
    • 13.8.2.1. Clinical Trials Information
  • 13.8.3. Safety and Efficacy
• 13.9. Redasemtide: Shionogi
  • 13.9.1. Product Description
  • 13.9.2. Other Development Activities
  • 13.9.3. Clinical Development
    • 13.9.3.1. Clinical Trials Information
  • 13.9.4. Safety and Efficacy
• 13.10. ALLO-ASC-SHEET: Anterogen
  • 13.10.1. Product Description
  • 13.10.2. Other Developmental Activities
  • 13.10.3. Clinical Developmental Activities
    • 13.10.3.1. Clinical Trial Information

14. Epidermolysis Bullosa: The 7MM Analysis

• 14.1. Key Findings
• 14.2. Epidermolysis Bullosa Market Outlook
• 14.3. Key Market Forecast Assumptions
• 14.4. Conjoint Analysis
• 14.5. Total Market Size of Epidermolysis Bullosa in the 7MM
• 14.6. The United States Market Size
  • 14.6.1. Total Market Size of Epidermolysis Bullosa in the United States
  • 14.6.2. Market Size of Epidermolysis Bullosa by Current and Emerging Therapies in the United States
• 14.7. EU4 and the UK Market Size
  • 14.7.1. Total Market Size of Epidermolysis Bullosa in EU4 and the UK
  • 14.7.2. Market Size of Epidermolysis Bullosa by Current and Emerging Therapies in EU4 and the UK
• 14.8. Japan Market Size
  • 14.8.1. Total Market Size of Epidermolysis Bullosa in Japan
  • 14.8.2. Market Size of Epidermolysis Bullosa by Current and Emerging Therapies in Japan

15. Unmet needs

16. SWOT Analysis

17. KOL Views

18. Market Access and Reimbursement

• 18.1. The United States
  • 18.1.1. Centre for Medicare & Medicaid Services (CMS)
• 18.2. EU4 and the UK
  • 18.2.1. Germany
  • 18.2.2. France
  • 18.2.3. Italy
  • 18.2.4. Spain
  • 18.2.5. The United Kingdom
• 18.3. Japan
  • 18.3.1. MHLW
• 18.4. Epidermolysis Bullosa Market Access and Reimbursement

19. Appendix

• 19.1. Bibliography
• 19.2. Report Methodology

20. DelveInsight Capabilities

21. Disclaimer