WHIM症候群市場 - 市場の洞察、疫学、市場予測：2034年

主なハイライト

• WHIM症候群は、CXCR4変異別引き起こされるまれな遺伝性疾患で、免疫不全、イボ、低ガンマグロブリン血症、骨髄異形成を引き起こします。2023年、主要7ヶ国における診断された有病者数は約179人と推定されました。
• WHIMは米国における重大な健康問題であり、その有病率は過去数十年間増加し続けています。DelveInsightの推計別と、主要7ヶ国の中でWHIMの症例数が最も多いのは米国だけで68％を占め、2023年にはフランスがこれに続きます。
• DelveInsightのアナリスト別評価によると、EU4ヶ国と英国では、男女ともにWHIM症候群と診断された有病者数は、2023年には男性19人、女性29人となり、今後数年間で増加すると予測されています。
• WHIM症候群の市場規模は、疾患の有病率、新たな治療法の利用可能性と採用、規制当局の承認、治療費、疾患に対する全体的な認識など、さまざまな要因の影響を受ける。さらに、診断や個別化医療の進歩も市場力学に影響を与える可能性があります。
• 2023年、WHIMの市場規模は主要7ヶ国の中で米国が最も大きく、約480万米ドルを占め、2034年までにさらに拡大すると予想されます。
• 日本のWHIM市場規模は2023年にはほぼ30万米ドルとなり、主要7ヶ国の総市場のわずか5%に過ぎません。主要7ヶ国の総市場規模は、2034年までにCAGR16.4％で上昇すると予想されます。
• WHIM症候群のパイプラインは著しく限られており、新しい治療法の研究開発が進んでいないことを示しています。この希少性は、この稀な病態に対処するための投資と技術革新の必要性を強調しています。WHIMの総市場規模は、新たな治療法の参入が期待されることから、予測期間中に急増すると予想されます。
• 2024年4月、米国FDAはX4 PharmaceuticalsのXOLREMDI（mavorixafor）カプセルをWHIM症候群の治療薬として承認し、この疾患に対する初の正式承認治療薬としました。この画期的な治療薬は、WHIM症候群の根本原因であるCXCR4経路の機能障害に対処するもので、第III相試験データの優先審査により承認されました。また、X4は希少小児疾患優先審査品目にも指定されました。この承認は、12歳以上の患者に、循環成熟好中球とリンパ球を増加させることを目的とした新たな治療選択肢を提供する、重要な進歩です。

当レポートは、米国、EU4ヶ国および英国（ドイツ、フランス、イタリア、スペイン）、日本のWHIM、歴史的および予測疫学ならびにWHIM市場動向の詳細な理解を提供します。

また、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WHIM市場規模の現在と予測を提供します。また、現在のWHIM治療実践、市場促進要因、市場障壁、SWOT分析、償還と市場アクセス、アンメットメディカルニーズなどを網羅し、最良の機会を選別し、市場の潜在力を評価します。

対象地域

• 米国
• EU4ヶ国（ドイツ、フランス、イタリア、スペイン）および英国
• 日本

調査期間：2020年～2034年

WHIM症候群治療市場

WHIM症候群は、CXCR4遺伝子の変異によって引き起こされるまれな遺伝病であり、免疫不全、イボ、低ガンマグロブリン血症という3つの症状を引き起こし、しばしば骨髄異栄養症（好中球が骨髄に捕捉される状態）を伴う。この症候群は免疫機能を著しく低下させ、感染症にかかりやすくなります。WHIMとは、この症候群の主な徴候である、いぼ、低ガンマグロブリン血症、感染症、骨髄異形成の頭文字をとったものです。

WHIM症候群はいくつかの特徴的な症状を示します。WHIM症候群の患者は免疫不全に陥り、免疫機能の低下により感染症にかかりやすくなります。もう一つの顕著な症状は、皮膚や粘膜に持続性および再発性の疣贅が開発されることです。低ガンマグロブリン血症は、血液中の免疫グロブリン（抗体）レベルの低下を特徴とし、免疫系の機能障害をさらに悪化させる。さらに、骨髄に好中球が蓄積するが血流では減少する骨髄異形成は、WHIM症候群の重要な特徴です。これらの症状はCXCR4遺伝子の変異から生じ、感染症に効果的に対応し、健康全般を維持する身体の能力に影響を及ぼします。

WHIM症候群の診断には、再発性感染症、イボ、低ガンマグロブリン血症、骨髄異形成などの症状の臨床評価と、CXCR4遺伝子の変異を確認するための遺伝子検査が必要です。遺伝子検査は確定診断のために極めて重要であるが、このプロセスにはアンメットニーズがあります。症状が他の疾患と重複している可能性があるため、多くの患者は認識不足や過小診断のために診断の遅れに直面しています。

さらに、専門的な検査や専門知識の利用が制限されることもあり、タイムリーな診断をさらに複雑にしています。WHIM症候群の早期介入と効果的な管理には、認知度の向上、遺伝子検査への幅広いアクセス、診断ツールの強化を通じてこれらのギャップに対処することが不可欠です。

WHIM症候群の治療は、症状の管理と根本的な遺伝子異常への対処に重点を置いています。現在の治療法としては、呼吸器疾患に対する持続陽圧呼吸療法（CPAP）、感染症や免疫不全に対する薬物療法などがあります。XOLREMDI（mavorixafor）のようなCXCR4拮抗薬は、CXCR4受容体を阻害することによって根本的な原因を狙い、免疫機能を改善し症状を軽減する可能性があります。さらに、遺伝子変異を修正する長期的な解決策として遺伝子治療が検討されています。

しかし、アンメットニーズは依然として大きいです。CPAPや感染管理など、現在の治療法のアドヒアランスは患者にとって困難なものです。さらに、利用可能な治療法の多くは、この症候群の複雑な症状に部分的にしか対応しておらず、個別化された治療法の選択肢が不足しています。WHIM症候群患者の予後とQOLを改善するためには、効果的な個別化治療に関するさらなる研究と、新たな治療法へのアクセスの改善が不可欠です。

本レポートのWHIM疫学の章では、2020年から2034年までの米国、EU4ヶ国カ国（ドイツ、フランス、イタリア、スペイン）、英国、日本を対象とした主要7ヶ国におけるWHIM症候群の診断有病者数、性別によるWHIM症候群の診断有病者数、年齢によるWHIM症候群の診断有病者数をセグメント化し、過去の疫学と予測を示しています。

• 米国で診断されたWHIM有病者総数は2023年に約121人となり、予測期間中に増加すると予測されています。
• WHIMと診断された有病者数は米国が主要7ヶ国で最も多く、次いでEU4ヶ国、英国、日本がそれぞれWHIM全体の27％、6％を占めています。
• 予測評価では、WHIM症候群と診断される有病率は増加しており、米国では2023年までに男性約48人、女性約73人に増加すると予測されています。この動向は、予測期間中に予想される両性の増加を反映しています。
• EU4ヶ国と英国では、2023年に診断されたWHIMの有病者数は約48例でした。これらの症例のうち、フランスが最も多いです。

XOLREMDI（mavorixafor）：X4 Pharmaceuticals

XOLREMDI（mavorixafor）は、選択的CXCR4受容体拮抗薬として機能するファースト・イン・クラスの経口低分子薬剤であり、WHIM症候群患者を適応症とする初めてのFDA承認カプセルです。この薬剤は、骨髄からの白血球の動員および輸送を促進します。WHIM症候群以外にも、マボリキサフォールは、重症先天性好中球減少症（SCN）、ワルデンシュトレーム・マクログロブリン血症、乳がん、悪性黒色腫、明細胞腎細胞がん（ccRCC）の適応で検討されています。

XOLREMDIは、規制当局のマイルストーンとして大きな注目を集めています。米国FDAは、成人のWHIM症候群の治療薬として、2020年10月にFast Track Designation（FTD）を、2019年11月にBreakthrough Therapy Designation（BTD）を承認しました。さらに、2018年10月にはFDAから希少疾病用医薬品指定（ODD）を、2020年12月には希少小児疾患指定を受けた。欧州委員会も2019年7月、WHIM症候群に対してODDを付与しました。

XOLREMDIは2024年にピークシェア60%を獲得し、ピークまで6年かかると予想され、中速の取り込みが見込まれます。

米国FDAにより承認されたプレリキサホル（MOZOBIL、AMD3100）は、非ホジキンリンパ腫および多発性骨髄腫患者の自家移植のためにCD34+造血幹細胞を動員します。また、CXCR4を部分的に阻害することでWHIM症候群にも対応し、幹細胞動員よりも低用量で使用されます。米国国立アレルギー感染症研究所は、WHIM症候群に対するplerixaforの開発を進めており、第III相臨床試験を終了しました。そのメカニズムは、CXCR4レセプターを特異的かつ可逆的に阻害し、間質細胞由来因子-1（SDF-1）の結合を阻害し、細胞接着に影響を与えるというものです。

しかし、2021年に第II／III相試験が終了して以来、この薬剤に関する最新情報やさらなる進展は報告されていないです。この停滞は、開発パイプラインや規制状況において、一時停止や進展がない可能性を示唆しています。

2024年にWHIM症候群に対する最初の治療法が承認され、WHIMの市場展望は拡大すると予想されます。

WHIM症候群の治療には、薬理学的アプローチと非薬理学的アプローチの両方が含まれます。薬理学的治療には主に、CXCR4シグナル伝達を部分的に遮断することで好中球減少症の管理に役立つCXCR4受容体拮抗薬であるプレリキサホル（MOZOBIL）が含まれます。これは白血球の動員を促進するために顆粒球コロニー刺激因子（G-CSF）と併用されることが多いです。さらに、WHIM症候群に特徴的な免疫不全に起因する感染症を管理するために、抗生物質や抗真菌薬が処方されます。

薬物療法以外の面では、感染症の管理と予防のための定期的なモニタリングを含む支持療法が重要です。衛生管理を徹底し、感染リスクを最小限に抑えるなどの生活習慣の調整も推奨されます。さらに、慢性疾患とともに生きることに伴う感情的・心理的課題に患者が対処できるよう、心理社会的サポートも重要です。これらの戦略を組み合わせることで、WHIM症候群患者の全体的な健康転帰と生活の質を改善することを目指しています。

• プレリキサフォー（モゾビル）などの新興治療薬の上市は、市場にプラスの影響を与えると予想されます。これらの治療薬が承認されれば、市場力学に大きな影響を与える可能性があるが、その成功率はまだ不透明です。
• 主要7ヶ国におけるWHIMの市場規模は、2023年にはほぼ630万米ドルであり、予測期間中にさらに拡大すると予想されます。
• EU4ヶ国と英国は、2023年のWHIM市場規模約130万米ドルを占めました。
• EU諸国の中では、フランスが2023年にほぼ40万米ドルで最も市場規模が大きく、スペインは2023年に10万米ドルでWHIMの市場規模が最も小さくなっています。
• プレリキサフォー（モゾビル）などの今後の治療薬の上市が期待されることから、WHIMの市場規模は今後数年で変化を見せると予想されます。

当レポートでは、主要7ヶ国におけるWHIM症候群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 WHIM症候群市場概要

• WHIM症候群の市場シェア（％）分布（2020年）
• WHIM症候群の市場シェア（％）分布（2034年）

第4章 疫学と市場予測の調査手法

第5章 主要な出来事

第6章 WHIM症候群のエグゼクティブサマリー

第7章 疾患の背景と概要：WHIM症候群

• イントロダクション
• 臨床的特徴
• 原因
• 免疫病態
• 組織病理学的所見
• 診断
• 診断アルゴリズム
• 治療

第8章 患者動向

第9章 WHIM症候群の疫学と患者人口

• 主な調査結果
• 前提と根拠：主要7ヶ国
  • 米国
  • EU4ヶ国と英国
  • 日本
• 主要7ヶ国におけるWHIM症候群の有病率の高い診断例
• 米国
  • 米国におけるWHIM症候群の有病率
  • 米国におけるWHIM症候群の有病率、性別
  • 米国におけるWHIM症候群の有病率、年齢別
• EU4ヶ国か国と英国
  • ドイツ
  • フランス
  • イタリア
  • スペイン
  • 英国
• 日本
  • 日本におけるWHIM症候群の有病率
  • 日本におけるWHIM症候群の有病率、性別
  • 日本におけるWHIM症候群の有病率、年齢別

第10章 WHIM症候群の市販治療

• ゾレムディ（マボリキサフォル）：X4 Pharmaceuticals
  • 製品プロファイル
  • その他の発達活動
  • 臨床開発
  • 臨床試験情報
  • 安全性と有効性

第11章 WHIM症候群の新たな治療法

第12章 WHIM症候群：主要7ヶ国市場分析

• 主な調査結果
• 市場見通し
• 主要な市場予測の前提条件
• 主要7ヶ国におけるWHIM症候群の市場規模
• 主要7ヶ国におけるWHIM症候群の市場規模（治療法別）
• 米国におけるWHIM症候群の市場規模
• EU4ヶ国と英国におけるWHIM症候群の市場規模
• 日本におけるWHIM症候群の市場規模

第13章 主要オピニオンリーダーの見解

第14章 SWOT分析

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
  • メディケア・メディケイドサービスセンター（CMS）
• EU4ヶ国と英国
  • ドイツ
  • フランス
  • イタリア
  • スペイン
  • 英国
• 日本
  • 厚生労働省

第18章 付録

第19章 DelveInsightのサービス内容

第20章 免責事項

List of Tables

• Table 1 Key Events
• Table 2 Summary of WHIM Syndrome Market and Epidemiology (2020-2034)
• Table 3 Various Sources of Donor Stem Cell and Their Features in HSCT
• Table 4 Diagnosed Prevalent Cases of WHIM Syndrome in the 7MM (2020-2034)
• Table 5 Diagnosed Prevalent Cases of WHIM Syndrome in the United States (2020-2034)
• Table 6 Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome in the United States (2020-2034)
• Table 7 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in the United States (2020-2034)
• Table 8 Diagnosed Prevalent Cases of WHIM Syndrome in EU4 and the UK (2020-2034)
• Table 9 Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome in EU4 and the UK (2020-2034)
• Table 10 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in EU4 and the UK (2020-2034)
• Table 11 Diagnosed Prevalent Cases of WHIM Syndrome in Germany (2020-2034)
• Table 12 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Germany (2020-2034)
• Table 13 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Germany (2020-2034)
• Table 14 Diagnosed Prevalent Cases of WHIM Syndrome in France (2020-2034)
• Table 15 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in France (2020-2034)
• Table 16 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in France (2020-2034)
• Table 17 Diagnosed Prevalent Cases of WHIM Syndrome in Italy (2020-2034)
• Table 18 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Italy (2020-2034)
• Table 19 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Italy (2020-2034)
• Table 20 Diagnosed Prevalent Cases of WHIM Syndrome in Spain (2020-2034)
• Table 21 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Spain (2020-2034)
• Table 22 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Spain (2020-2034)
• Table 23 Diagnosed Prevalent Cases of WHIM Syndrome in the United Kingdom (2020-2034)
• Table 24 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in the United Kingdom (2020-2034)
• Table 25 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in the United Kingdom (2020-2034)
• Table 26 Diagnosed Prevalent Cases of WHIM syndrome in Japan (2020-2034)
• Table 27 Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome in Japan (2020-2034)
• Table 28 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Japan (2020-2034)
• Table 29 XOLREMDI, Clinical Trial Description, 2024
• Table 30 Plerixafor, Clinical Trial Description, 2024
• Table 31 Total Market Size of WHIM Syndrome in the 7MM, in USD million (2020-2034)
• Table 32 Market Size of WHIM Syndrome by Therapies in the 7MM, in USD million (2020-2034)
• Table 33 Total Market Size of WHIM syndrome in the US, in USD million (2020-2034)
• Table 34 Market Size of WHIM Syndrome by Therapies in the US, in USD million (2020-2034)
• Table 35 Market Size by Therapies of WHIM Syndrome in EU4 and the UK, in USD million (2020-2034)
• Table 36 Total Market Size of WHIM Syndrome in Germany, in USD million (2020-2034)
• Table 37 Market Size by Therapies of WHIM Syndrome in Germany, in USD million (2020-2034)
• Table 38 Total Market Size of WHIM Syndrome in France in USD million (2020-2034)
• Table 39 Market Size by Therapies of WHIM Syndrome in France, in USD million (2020-2034)
• Table 40 Total Market Size of WHIM Syndrome in Italy in USD million (2020-2034)
• Table 41 Market Size by Therapies of WHIM Syndrome in Italy, in USD million (2020-2034)
• Table 42 Total Market Size of WHIM Syndrome in Spain in USD million (2020-2034)
• Table 43 Market Size by Therapies of WHIM Syndrome in Spain, in USD million (2020-2034)
• Table 44 Total Market Size of WHIM Syndrome in the UK in USD million (2020-2034)
• Table 45 Market Size by Therapies of WHIM Syndrome in the UK, in USD million (2020-2034)
• Table 46 Total Market Size of WHIM Syndrome in Japan, in USD million (2020-2034)
• Table 47 Market Size by Therapies of WHIM Syndrome in Japan, in USD million (2020-2034)

List of Figures

• Figure 1 Model of WHIM Syndrome Molecular and Cellular Immunopathogenesis
• Figure 2 A diagnostic Flowchart of WHIM Syndrome
• Figure 3 Diagnostic Algorithm of WHIM Syndrome
• Figure 4 Treatment Regimens Used in WHIM Patients
• Figure 5 Chromothriptic Cure of WHIM Syndrome
• Figure 6 Patient Journey of WHIM Syndrome
• Figure 7 Diagnosed Prevalent cases of WHIM Syndrome in the 7MM (2020-2034)
• Figure 8 Diagnosed Prevalent Cases of WHIM Syndrome in the United States (2020-2034)
• Figure 9 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in the United States (2020-2034)
• Figure 10 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in the United States (2020-2034)
• Figure 11 Diagnosed Prevalent Cases of WHIM Syndrome in EU4 and the UK (2020-2034)
• Figure 12 Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome in EU4 and the UK (2020-2034)
• Figure 13 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in EU4 and the UK (2020-2034)
• Figure 14 Diagnosed Prevalent Cases of WHIM Syndrome in Germany (2020-2034)
• Figure 15 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Germany (2020-2034)
• Figure 16 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Germany (2020-2034)
• Figure 17 Diagnosed Prevalent Cases of WHIM Syndrome in France (2020-2034)
• Figure 18 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in France (2020-2034)
• Figure 19 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in France (2020-2034)
• Figure 20 Diagnosed Prevalent Cases of WHIM Syndrome in Italy (2020-2034)
• Figure 21 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Italy (2020-2034)
• Figure 22 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Italy (2020-2034)
• Figure 23 Diagnosed Prevalent Cases of WHIM Syndrome in Spain (2020-2034)
• Figure 24 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Spain (2020-2034)
• Figure 25 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Spain (2020-2034)
• Figure 26 Diagnosed Prevalent Cases of WHIM Syndrome in the United Kingdom (2020-2034)
• Figure 27 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in the United Kingdom (2020-2034)
• Figure 28 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in the United Kingdom (2020-2034)
• Figure 29 Diagnosed Prevalent Cases of WHIM syndrome in Japan (2020-2034)
• Figure 30 Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome in Japan (2020-2034)
• Figure 31 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Japan (2020-2034)
• Figure 32 Total Market Size of WHIM Syndrome in the 7MM, in USD million (2020-2034)
• Figure 33 Market Size of WHIM Syndrome by Therapies in the 7MM, in USD million (2020-2034)
• Figure 34 Total Market Size of WHIM syndrome in the US, in USD million (2020-2034)
• Figure 35 Market Size of WHIM Syndrome by Therapies in the US, in USD million (2020-2034)
• Figure 36 Market Size by Therapies of WHIM Syndrome in EU4 and the UK, in USD million (2020-2034)
• Figure 37 Total Market Size of WHIM Syndrome in Germany, in USD million (2020-2034)
• Figure 38 Market Size by Therapies of WHIM Syndrome in Germany, in USD million (2020-2034)
• Figure 39 Total Market Size of WHIM Syndrome in France in USD million (2020-2034)
• Figure 40 Market Size by Therapies of WHIM Syndrome in France, in USD million (2020-2034)
• Figure 41 Total Market Size of WHIM Syndrome in Italy in USD million (2020-2034)
• Figure 42 Market Size by Therapies of WHIM Syndrome in Italy, in USD million (2020-2034)
• Figure 43 Total Market Size of WHIM Syndrome in Spain in USD million (2020-2034)
• Figure 44 Market Size by Therapies of WHIM Syndrome in Spain, in USD million (2020-2034)
• Figure 45 Total Market Size of WHIM Syndrome in the UK in USD million (2020-2034)
• Figure 46 Market Size by Therapies of WHIM Syndrome in the UK, in USD million (2020-2034)
• Figure 47 Total Market Size of WHIM Syndrome in Japan, in USD million (2020-2034)
• Figure 48 Market Size by Therapies of WHIM Syndrome in Japan, in USD million (2020-2034)
• Figure 49 Market Size of WHIM Syndrome by Therapies in Japan, in USD million (2020-2034)
• Figure 50 Reimbursement Process in the United States
• Figure 51 Reimbursement Process in the United States
• Figure 52 Reimbursement Process in Germany
• Figure 53 Reimbursement Process in France
• Figure 54 Reimbursement Process in Italy
• Figure 55 Reimbursement Process in the United Kingdom
• Figure 56 Reimbursement Process in Japan

Key Highlights:

• WHIM syndrome is a rare genetic disorder caused by CXCR4 mutations, leading to immunodeficiency, warts, hypogammaglobulinemia, and myelokathexis. In 2023, the diagnosed prevalent population in the 7MM was estimated to be around 179 individuals.
• WHIM is a significant health issue in the United States, and its prevalence has been increasing over the past few decades. According to DelveInsight estimates, among the 7MM, the US has most number of cases of WHIM accounting for 68% of cases alone, followed by France in 2023.
• Assessments as per DelveInsight's analysts show that the overall diagnosed prevalent cases of WHIM syndrome in both genders in EU4 and the UK are anticipated to increase in the coming years in comparison to 19 males and 29 females, in 2023.
• The market size of WHIM syndrome is influenced by various factors, such as the prevalence of the condition, the availability and adoption of emerging therapies, regulatory approvals, treatment costs, and the overall awareness of the disease. Additionally, advancements in diagnostics and personalized medicine may also impact market dynamics.
• In 2023, the market size of WHIM was highest in the US among the 7MM, accounting for approximately USD 4.8 million which is further expected to increase by 2034.
• The market size of WHIM in Japan was nearly USD 0.3 million in 2023, accounting for only 5% of the total market of the 7MM. The total market size in the 7MM is expected to rise by 2034 at a compound annual growth rate (CAGR) of 16.4%.
• The pipeline for WHIM syndrome is notably limited, indicating a lack of ongoing research and development for new therapies. This scarcity highlights the need for increased investment and innovation in addressing this rare condition. The total market size of WHIM is anticipated to upsurge during the forecast period due to the expected entry of emerging therapies.
• In April 2024, the US FDA granted approval of X4 Pharmaceuticals' XOLREMDI (mavorixafor) capsules for WHIM syndrome, making it the first officially approved treatment for this condition. This breakthrough therapy, approved following a Priority Review of Phase III trial data, addresses CXCR4 pathway dysfunction, the root cause of WHIM syndrome. X4 also received a Rare Pediatric Disease Priority Review Voucher. This approval is a significant advancement, offering a new treatment option for patients aged 12 and older, aimed at increasing circulating mature neutrophils and lymphocytes.

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

The WHIM market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM WHIM market size from 2020 to 2034. The Report also covers current WHIM treatment practice, market drivers, market barriers, SWOT analysis, reimbursement and market access, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Geography Covered:

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

Study Period: 2020-2034

WHIM Syndrome Treatment Market

WHIM Overview

WHIM syndrome is a rare genetic disorder caused by mutations in the CXCR4 gene, leading to a triad of symptoms: immunodeficiency, warts, and hypogammaglobulinemia, often accompanied by myelokathexis, a condition where neutrophils are trapped in the bone marrow. This syndrome significantly impairs immune function and increases susceptibility to infections. The term WHIM is an acronym for the main signs of the syndrome: warts, hypogammaglobulinemia, infections, and myelokathexis.

WHIM syndrome manifests through several distinct symptoms. Individuals with this condition experience immunodeficiency, which leads to increased vulnerability to infections due to compromised immune function. Another prominent symptom is the development of persistent and recurrent warts on the skin and mucous membranes. Hypogammaglobulinemia, characterized by low levels of immunoglobulins (antibodies) in the blood, further exacerbates immune system dysfunction. Additionally, myelokathexis, a condition where neutrophils accumulate in the bone marrow but are reduced in the bloodstream, is a key feature of WHIM syndrome. These symptoms arise from mutations in the CXCR4 gene, impacting the body's ability to effectively respond to infections and maintain overall health.

WHIM Diagnosis

Diagnosing WHIM syndrome involves clinical evaluation of symptoms such as recurrent infections, warts, hypogammaglobulinemia, and myelokathexis, followed by genetic testing to confirm CXCR4 gene mutations. While genetic testing is crucial for a definitive diagnosis, there are unmet needs in this process. Many patients face delays due to lack of awareness and underdiagnosis, as symptoms may overlap with other conditions.

Additionally, the availability of specialized testing and expertise can be limited, further complicating timely diagnosis. Addressing these gaps through improved awareness, broader access to genetic testing, and enhanced diagnostic tools is essential for early intervention and effective management of WHIM syndrome.

WHIM Treatment

Treatment for WHIM syndrome focuses on managing symptoms and addressing the underlying genetic defect. Current approaches include continuous positive airway pressure (CPAP) therapy for associated respiratory issues, and medications to manage infections and immune deficiencies. CXCR4 antagonists, such as XOLREMDI (mavorixafor), aim to target the root cause by inhibiting the CXCR4 receptor, potentially improving immune function and reducing symptoms. Additionally gene therapy is being explored as a long-term solution to correct the genetic mutation.

However, significant unmet needs remain. Adherence to current therapies, such as CPAP and infection management, can be challenging for patients. Additionally, many available treatments only partially address the syndrome's complex symptoms, and there is a lack of personalized therapeutic options. More research into effective, tailored treatments and better access to emerging therapies are critical for improving outcomes and quality of life for individuals with WHIM syndrome.

WHIM Epidemiology

As the market is derived using the patient-based model, the WHIM epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Diagnosed Prevalent Cases of WHIM Syndrome, Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome, and Age-specific Diagnosed Prevalent Cases of WHIM Syndrome 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 diagnosed prevalent cases of WHIM in the US comprised approximately 121 in 2023 and are projected to increase during the forecast period.
• As per DelveInsight's estimates, the US alone accounts the highest number of total diagnosed prevalent cases, of WHIM in the 7MM, followed by EU4 and the UK and Japan, contributing to 27% and 6% of all WHIM cases respectively.
• Forecast assessments suggest a growing prevalence of diagnosed WHIM syndrome cases, projecting an increase to approximately 48 males and 73 females by 2023 in the US. This trend reflects an anticipated rise in both genders over the forecast period.
• EU4 and the UK accounted for around 48 diagnosed prevalent cases of WHIM in 2023. Of these cases, France accounted for the highest number of cases.

WHIM Drug Chapters

The drug chapter segment of the WHIM report encloses a detailed analysis of WHIM approved and off-label drugs along with late-stage (Phase III) pipeline drugs. It also helps to understand the WHIM 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.

WHIM Syndrome Marketed Drugs

XOLREMDI (mavorixafor): X4 Pharmaceuticals

XOLREMDI (mavorixafor) is the first FDA approved capsules indicated in patients with WHIM syndrome promising first-in-class, oral small molecule, functioning as a selective CXCR4 receptor antagonist. This drug enhances the mobilization and trafficking of white blood cells from the bone marrow. Beyond WHIM syndrome, Mavorixafor is being explored for severe congenital neutropenia (SCN), Waldenstrom's macroglobulinemia, breast cancer, malignant melanoma, and clear cell renal cell carcinoma (ccRCC).

In terms of regulatory milestones, XOLREMDI has garnered significant attention. The US FDA granted Fast Track Designation (FTD) in October 2020 and Breakthrough Therapy Designation (BTD) in November 2019 for treating WHIM syndrome in adults. Additionally, it received Orphan Drug Designation (ODD) from the FDA in October 2018 and Rare Pediatric Disease Designation in December 2020. The European Commission also granted ODD for WHIM syndrome in July 2019.

XOLREMDI, according to the DelveInsight's forecast model would capture a peak share of 60% in 2024 and is anticipated to take 6 years to peak with a medium-fast uptake.

Emerging WHIM Syndrome Drugs

Plerixafor: National Institute of Allergy and Infectious Diseases (NIAID)

Plerixafor (MOZOBIL, AMD3100), approved by the US FDA, mobilizes CD34+ hematopoietic stem cells for autologous transplantation in non-Hodgkin's lymphoma and multiple myeloma patients. It also addresses WHIM syndrome by partially blocking CXCR4, used at lower doses than for stem cell mobilization. The National Institute of Allergy and Infectious Diseases is advancing plerixafor for WHIM syndrome, which has completed Phase III trials. Its mechanism involves specific, reversible inhibition of the CXCR4 receptor, disrupting the binding of stromal cell-derived factor-1 (SDF-1) and affecting cellular adhesion.

However, since the conclusion of Phase II/III trials in 2021, there have been no updates or further advancements reported for this drug. This stagnation suggests a potential pause or lack of progress in its development pipeline or regulatory status.

WHIM Market Outlook

With first approved therapy for WHIM Syndrome in 2024, the market outlook for WHIM is expected to expand.

Treatment for WHIM syndrome includes both pharmacological and non-pharmacological approaches. Pharmacological treatments primarily involve Plerixafor (MOZOBIL), a CXCR4 receptor antagonist that helps manage neutropenia by partially blocking CXCR4 signaling. This is often used alongside Granulocyte-Colony Stimulating Factor (G-CSF) to enhance white blood cell mobilization. Additionally, antibiotics and antifungals are prescribed to manage infections stemming from the immune deficiencies characteristic of WHIM syndrome.

On the non-pharmacological side, supportive care is crucial, involving regular monitoring to manage and prevent infections. Lifestyle adjustments such as practicing good hygiene and minimizing infection risks are also recommended. Furthermore, psychosocial support is important to help patients cope with the emotional and psychological challenges associated with living with a chronic condition. These combined strategies aim to improve overall health outcomes and quality of life for individuals with WHIM syndrome.

• The launch of emerging therapies, such as Plerixafor (Mozobil), and others are expected to impact the market positively. The approval of these therapies could significantly impact market dynamics, although their success rates remain uncertain.
• The market size of WHIM in the 7MM was nearly USD 6.3 million in 2023, which is further anticipated to increase during the forecast period.
• The EU4 and the UK accounted for the approximately USD 1.3 million market size of WHIM approximately in 2023.
• Among the EU countries, France had the highest market size with nearly USD 0.4 million in 2023, while Spain had the lowest market size for WHIM with USD 0.1 million in 2023.
• With the expected launch of upcoming therapies, such as Plerixafor (Mozobil), among others, the total market size of WHIM is expected to show change in the upcoming years.

WHIM Pipeline Development Activities

The report provides insights into WHIM syndrome clinical trials within Phase III, Phase II, and Phase I stage. 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 WHIM emerging therapies.

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 the secondary research. Industry Experts were contacted for insights on WHIM evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility, including KOL from Associate Research Physician, National Institute of Allergy and Infectious Disease, the US; University Hospital Aachen, Germany; Department of Pediatrics, Institute of Molecular Medicine, Brescia, Italy; Department of Pediatrics, Jikei University School of Medicine, Tokyo, Japan; Immunologist, Sant Joan de Deu Research, Spain, and others.

Delveinsight's analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Their opinion helps understand and validate current and emerging therapies, treatment patterns, or WHIM 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 and Conjoint 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.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

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. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

The high cost of therapies for the treatment is a major factor restraining the growth of the global drug market. Because of the high cost, the economic burden is increasing, leading the patient to escape from proper treatment.

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 WHIM Syndrome Market Report

• The report covers a segment of key events, an executive summary, descriptive overview of WHIM syndrome, explaining its causes, signs and symptoms, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines.
• 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 WHIM market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind the 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 WHIM market.

WHIM Report Insights

• WHIM Syndrome Patient Population
• Therapeutic Approaches
• WHIM Pipeline Analysis
• WHIM Market Size and Trends
• Existing and Future Market Opportunity

WHIM Report Key Strengths

• 11 years Forecast
• The 7MM Coverage
• WHIM Epidemiology Segmentation
• Key Cross Competition
• Conjoint Analysis
• WHIM Syndrome Drugs Uptake
• Key WHIM Syndrome Market Forecast Assumptions

WHIM Report Assessment

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

Key Questions Answered In The WHIM Syndrome Market Report

WHIM Syndrome Market Insights

• What was the WHIM market share (%) distribution in 2020 and how it would look like in 2034?
• What would be the WHIM total market size as well as market size by therapies across the 7MM during the forecast period (2024-2034)?
• What are the key findings pertaining to the market across the 7MM and which country will have the largest WHIM market size during the forecast period (2024-2034)?
• At what CAGR, the WHIM market is expected to grow at the 7MM level during the forecast period (2024-2034)?
• What would be the WHIM market outlook across the 7MM during the forecast period (2024-2034)?
• What would be the WHIM market growth till 2034 and what will be the resultant market size in the year 2034?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

WHIM Syndrome Epidemiology Insights

• What is the disease risk, burden, and unmet needs of WHIM Syndrome?
• What is the historical WHIM patient population in the United States, EU5 (Germany, France, Italy, Spain, and the UK), and Japan?
• What would be the forecasted patient population of WHIM at the 7MM level?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to WHIM Syndrome?
• Out of the above-mentioned countries, which country would have the highest prevalent population of WHIM syndrome during the forecast period (2024-2034)?
• At what CAGR the population is expected to grow across the 7MM during the forecast period (2024-2034)?

Current WHIM Syndrome Treatment Scenario, Marketed Drugs, and Emerging Therapies

• What are the current options for the treatment of WHIM along with the approved therapy?
• What are the current treatment guidelines for the treatment of WHIM in the US, Europe, And Japan?
• What are the WHIM marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
• How many companies are developing therapies for the treatment of WHIM Syndrome?
• How many emerging therapies are in the mid-stage and late stages of development for the treatment of WHIM Syndrome?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the WHIM therapies?
• What are the recent therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for WHIM and their status?
• What are the key designations that have been granted for the emerging therapies for WHIM Syndrome?
• What are the 7MM historical and forecasted market of WHIM Syndrome?

Reasons to Buy WHIM Syndrome Market Report

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the WHIM 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.
• To 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.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Detailed analysis and potential of current and emerging therapies under the conjoint analysis section to provide visibility around leading emerging drugs.
• Highlights of Access and Reimbursement policies of approved therapies, barriers to accessibility of off-label expensive therapies, and patient assistance programs.
• To understand the perspective of Key Opinion Leaders around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in future.
• Detailed insights on the unmet need of the existing market so that the upcoming players can strengthen their development and launch strategy.

Frequently Asked Questions:

1. What is the forecast period covered in the report?

The WHIM Epidemiology and Market Insight report for the 7MM covers the forecast period from 2024 to 2034, providing a projection of market dynamics and trends during this timeframe.

2. Who are the key players in the WHIM market?

The WHIM market currently lacks robust development. However, the forecast period may see significant activity as major players are anticipated to enter the market and focus on developing treatments for WHIM syndrome.

3. How is the market size estimated in the forecast report?

The market size is estimated through data analysis, statistical modeling, and expert opinions. It may consider factors such as incident cases, treatment costs, revenue generated, and market trends.

4. What is the key driver of the WHIM market?

The increase in diagnosed prevalent cases of WHIM and the launch of emerging therapies are attributed to be the key drivers for increasing the WHIM market.

5. What is the expected impact of emerging therapies or advancements in WHIM treatment on the market?

Introducing new therapies, advancements in diagnostic techniques, and innovations in treatment approaches can significantly impact the WHIM treatment market. Market forecast reports may provide analysis and predictions regarding the potential impact of these developments.

6. Does the report provide insights into the competitive landscape of the market?

The market forecast report may include information on the competitive landscape, profiling key market players, their product offerings, partnerships, and strategies, and helping stakeholders understand the competitive dynamics of the WHIM market.

Table of Contents

1 Key Insights

2 Report Introduction

3 WHIM Syndrome Market Overview at a Glance

• 3.1 Market Share (%) Distribution of WHIM Syndrome in 2020
• 3.2 Market Share (%) Distribution of WHIM Syndrome in 2034

4 Epidemiology and Market Forecast Methodology

5 Key Events

6 Executive Summary of WHIM Syndrome

7 Disease Background and Overview: WHIM syndrome

• 7.1 Introduction
• 7.2 Clinical Features
• 7.3 Causes
• 7.4 Immunopathogenesis
• 7.5 Histopathological Findings
• 7.6 Diagnosis
• 7.6 1 Diagnostic Algorithm
• 7.7 Treatment

8 Patient Journey

9 Epidemiology and Patient Population of WHIM Syndrome

• 9.1 Key Findings
• 9.2 Assumptions and Rationale: The 7MM
  • 9.2.1 The United States
  • 9.2.2 EU4 and the UK
  • 9.2.3 Japan
• 9.3 Diagnosed Prevalent Cases of WHIM Syndrome in the 7MM
• 9.4 The United States
  • 9.4.1 Diagnosed Prevalent Cases of WHIM Syndrome in the US
  • 9.4.2 Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome in the US
  • 9.4.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in the US
• 9.5 EU4 Countries and the UK
  • 9.5.1 Germany
    • 9.5.1.1 Diagnosed Prevalent Cases of WHIM Syndrome in Germany
    • 9.5.1.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Germany
    • 9.5.1.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Germany
  • 9.5.2 France
    • 9.5.2.1 Diagnosed Prevalent Cases of WHIM Syndrome in France
    • 9.5.2.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in France
    • 9.5.2.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in France
  • 9.5.3 Italy
    • 9.5.3.1 Diagnosed Prevalent Cases of WHIM Syndrome in Italy
    • 9.5.3.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Italy
    • 9.5.3.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Italy
  • 9.5.4 Spain
    • 9.5.4.1 Diagnosed Prevalent Cases of WHIM Syndrome in Spain
    • 9.5.4.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Spain
    • 9.5.4.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Spain
  • 9.5.5 The United Kingdom
    • 9.5.5.1 Diagnosed Prevalent Cases of WHIM Syndrome in the United Kingdom
    • 9.5.5.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in the United Kingdom
    • 9.5.5.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in the United Kingdom
• 9.6 Japan
  • 9.6.1 Diagnosed Prevalent Cases of WHIM Syndrome in Japan
  • 9.6.2 Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome in Japan
  • 9.6.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Japan

10 WHIM Syndrome Marketed Therapies

• 10.1 XOLREMDI (mavorixafor): X4 Pharmaceuticals
  • 10.1.1 Product Profile
  • 10.1.2 Other Developmental Activities
  • 10.1.3 Clinical Development
  • 10.1.4 Clinical Trials Information
  • 10.1.5 Safety and Efficacy
  • 10.1.6 Product Profile

11 Emerging WHIM Syndrome Therapies

• 11.1 Key Cross Competition
• 11.2 Plerixafor: National Institute of Allergy and Infectious Diseases (NIAID)
  • 11.2.1 Drug Description
  • 11.2.2 Regulatory Milestone
  • 11.2.3 Other Development Activities
  • 11.2.4 Clinical Trials Information
  • 11.2.5 Safety and Efficacy
  • 11.2.6 Analysts' views

12 WHIM Syndrome: Seven Major Market Analysis

• 12.1 Key Findings
• 12.2 Market Outlook
• 12.3 Key Market Forecast Assumptions
• 12.4 Market Size of WHIM Syndrome in the 7MM
• 12.5 Market Size of WHIM Syndrome by Therapies in the 7MM
• 12.6 Market Size of WHIM Syndrome in the United States
  • 12.6.1 Total Market Size of WHIM Syndrome
  • 12.6.2 Market Size of WHIM Syndrome by Therapies
• 12.7 Market Size of WHIM Syndrome in EU4 and the UK
  • 12.7.1 Germany
    • 12.7.1.1 Total Market Size of WHIM Syndrome
    • 12.7.1.2 Market Size of WHIM Syndrome by Therapies
  • 12.7.2 France
    • 12.7.2.1 Total Market Size of WHIM Syndrome
    • 12.7.2.2 Market Size of WHIM Syndrome by Therapies
  • 12.7.3 Italy
    • 12.7.3.1 Total Market Size of WHIM Syndrome
    • 12.7.3.2 Market Size of WHIM Syndrome by Therapies
  • 12.7.4 Spain
    • 12.7.4.1 Total Market Size of WHIM Syndrome
    • 12.7.4.2 Market Size of WHIM Syndrome by Therapies
  • 12.7.5 The United Kingdom
    • 12.7.5.1 Total Market Size of WHIM Syndrome
    • 12.7.5.2 Market Size of WHIM Syndrome by Therapies
• 12.8 Market Size of WHIM Syndrome in Japan
  • 12.8.1 Total Market Size of WHIM Syndrome
  • 12.8.2 Market Size of WHIM Syndrome by Therapies

13 Key Opinion Leaders' Views

14 SWOT Analysis

15 Unmet Needs

16 Market Access and Reimbursement

• 17.1 The United States
  • 17.1.1 Centers for Medicare & Medicaid Services (CMS)
• 17.2 In EU4 and the UK
  • 17.2.1 Germany
  • 17.2.2 France
  • 17.2.3 Italy
  • 17.2.4 Spain
  • 17.2.5 The United Kingdom
• 17.3 Japan
  • 17.3.1 MHLW

18 Appendix

• 18.1 Bibliography
• 18.2 Acronyms and Abbreviations
• 18.3 Report Methodology

19 DelveInsight Capabilities

20 Disclaimer