鎌状赤血球症（SCD）市場 - 市場の洞察、疫学、市場予測：2034年

主なハイライト

• 鎌状赤血球症（SCD）は、異常なヘモグロビンの存在により赤血球が鎌状になることを特徴とする遺伝性血液疾患です。SCDは世界の健康問題であり、特にアフリカ、中東、地中海、南アジアに祖先を持つ人々に多く見られます。
• SCDの主な原因は、ヘモグロビンの産生に影響するHBB遺伝子の突然変異です。SCDの患者は、両親から2コピーの変異遺伝子を受け継ぐため、ヘモグロビンの産生に異常をきたします。異常な赤血球は硬く粘着性になる傾向があり、血流を妨げ、鎌状赤血球クリーゼと呼ばれる激痛のエピソードを引き起こします。
• SCDは罹患者に大きな課題をもたらし、生活の質や寿命に影響を及ぼします。一般的な合併症としては、貧血、急性胸部症候群、脳卒中、臓器障害（腎臓、肝臓など）、感染症にかかりやすくなることなどが挙げられます。
• 現在、SCDの治療には非ステロイド性抗炎症薬、輸血、キレート剤、栄養補助食品、広域抗生物質が使用されています。さらに、米国FDAが鎌状赤血球症の治療薬として承認したものには、DROXIA（ヒドロキシ尿素）、ENDARI（L-グルタミン経口粉末）、ADAKVEO（クリザンリズマブ-tmca）、OXBRYTA（voxelotor）などがあります。
• SCD患者の全体的なケアと転帰の改善には、集学的アプローチが必要です。これには、包括的なヘルスケアサービス、遺伝カウンセリング、早期発見、合併症の管理などが含まれます。患者教育や支援プログラムは、SCD患者が効果的に病状を管理し、生活の質を向上させるために極めて重要です。
• SCDの新たな治療法や治療法の可能性を探るための研究開発が続けられています。遺伝子治療や遺伝子編集技術は、SCDの原因となる根本的な遺伝子変異を修正することが期待されています。これらの新しい治療法の安全性と有効性を評価するための臨床試験が進行中であり、SCDが治癒または効果的に管理される未来への希望をもたらしています。
• SCDは生命を脅かす疾患であり、有効な治療法がないことが大きな負担となっています。これほど長い間市場に出回っているにもかかわらず、治療アドヒアランスの低さと治療中止率の高さという課題が残っています。
• 最近承認された治療法では、アドヒアランスと治療費の償還もまだ困難です。最近市場に導入されたほぼ3つの治療薬、すなわちENDARI（L-グルタミン）、ADAKVEO（クリザンリズマブ）、OXBRYTA（ボクセロトール）があります。最近承認されたOXBRYTAは、簡便な投与経路でSCDの根本原因をターゲットとするクラス初の経口治療薬となっています。
• 2023年のSCDの市場規模は米国が最も大きく、EU4ヶ国および英国と比較して約92%のシェアを占めています。
• 2023年には、EU4ヶ国と英国の中ではフランスが最大の市場規模を占め、スペインのシェアは最小でした。
• 鎌状赤血球症の治療薬として最近承認されたものは少なく、CASGEVY（exagamglogene autotemcel[exa-cel]）（Vertex Pharmaceuticals/CRISPR Therapeutics）やLYFGENIA（Lovo-cel）（bluebird bio）などがあります。これらの治療薬の承認は、市場全体の成長にプラスの影響を与えると予想されます。一方、新たなパイプラインとしては、EDIT-301、Mitapivat、Inclacumab（PF-07940370）、Osivelotor（GBT-601）などの医薬品があります。
• 診断の遅れ、SCDの重篤な合併症、経済的負担、病気に対する正しい理解の欠如がSCD市場を直撃することになるとみられています。
• 結論として、SCDはヘモグロビンの異常を特徴とする複雑な遺伝性血液疾患であり、合併症や生命予後の低下につながります。しかし、治療法の進歩や現在進行中の研究努力は、管理方法の改善や治癒の可能性に希望を与えるものです。ヘルスケアサービスの強化、認知度の向上、SCD患者への支援は、主要6ヶ国における本疾患に関連する課題に取り組むための不可欠なステップです。

報告サマリー

• 当レポートは、疫学セグメント（地域別、SCDの形質、SCDの総有病者数、SCDの診断例、SCDの年齢別有病者数、SCDのタイプ別有病者数）および予測に関する広範な知識を提供し、診断率、疾患の進行、治療ガイドラインにおける将来の潜在的な成長について深い理解を示します。本書は、これらの側面に関する包括的な洞察を提供し、主題の徹底的な評価を可能にします。
• さらに、Mitapivat、EDIT-301（Renizgamglogene autogedtemcel、またはreni-cel）、Inclacumab（PF-07940370）、Osivelotor（GBT-601）のような現在の管理技術や新たな治療法の包括的な説明、および現在の治療状況に影響を与え、全体的な市場シフトをもたらす後期および中期段階（第III相および第II相）および著名な治療法の詳細なプロファイルが報告書に記載されています。
• また、SCD市場の包括的な分析を網羅し、市場規模実績と予測（2020年～2034年）を詳細に検証しています。また、治療薬の市場シェア、詳細な前提条件、調査手法の根拠も記載しています。また、主要6ヶ国地域における医薬品アウトリーチも掲載しています。
• 本レポートには、SWOT分析別動向の把握や、様々な病院や著名大学の専門家を含む専門家の洞察/KOLの見解、患者の旅路、主要6ヶ国 SCD市場の形成と推進に役立つ治療嗜好など、事業戦略を策定する際に強みとなる定性的洞察が含まれています。

市場

Pfizer、Agios Pharmaceuticals、Editas Medicine、Hoffmann-La Rocheなど、様々な主な企業がSCDの治療法の開発に携わっています。新たな治療薬やその他の治療法の上市が期待されることから、予測期間[2024-2034]中の市場規模は大幅に拡大すると思われます。

• 2023年のSCDの総市場規模は約6億5,000万米ドルであり、調査期間中（2020年～2034年）の主要6ヶ国における市場規模は2034年までに増加すると予測されます。
• 主要6ヶ国の中で、2023年のSCDの市場シェアは米国が最も高く、次いでフランス、英国でした。
• 予測期間（2024年～2034年）には、エタボピバット、インクラクマブ（PF-07940370）、レニセル（EDIT-301）、オシベローター（GBT-601）などのパイプライン候補がSCD市場規模の拡大に大きく貢献すると予想されます。
• 新興治療薬の中では、Casgevy（Exa-cel）が2034年までに主要6ヶ国で最大の市場シェアを獲得すると予想されています。
• 遺伝子治療薬には市場参入や償還など多くの課題があり、ニッチプールを対象としているため市場シェアは低いです。

上市済み薬剤

CASGEVY（exagamglogeneオートテンセル）：Vertex Pharmaceuticals/CRISPR Therapeutics

CASGEVYは、CRISPR/Cas9技術によりBCL11A遺伝子の赤血球特異的エンハンサー領域を編集した自己CD34+造血幹細胞（造血幹細胞）からなるゲノム編集細胞療法です。CASGEVYは、患者自身のCD34+細胞が赤血球系細胞のBCL11A発現を減少させ、胎児ヘモグロビン（HbF）産生を増加させるように改変された造血幹細胞移植手順による1回限りの投与を意図しています。HbFは、胎児の開発中に自然に存在する酸素運搬ヘモグロビンの形態であり、出生後に成人型ヘモグロビンに切り替わる。CASGEVYはSCD患者の血管閉塞性クリーゼを軽減または消失させることが示されています。

2023年12月、Vertex Pharmaceuticals社とCRISPR Therapeuticsは、CRISPR/Cas9ゲノム編集細胞療法であるCASGEVY（exagamglogene autotemcel[exa-cel]）を、血管閉塞性クリーゼ（VOCs）を再発する12歳以上の鎌状赤血球症（SCD）患者の治療薬として米国FDAが承認したと発表しました。

その後、CASGEVYは英国で条件付き販売承認を取得しました。バーレーンの医薬品・ヘルスケア製品規制庁および国家保健規制庁は、造血幹細胞移植が適切であり、ヒト白血球抗原が一致した関連造血幹細胞ドナーが得られない、再発性血管閉塞性クリーゼまたは輸血依存性β-サラセミア（TDT）を特徴とする12歳以上のSCD患者を対象としています。

ENDARI（L-グルタミン）：Emmaus Life Sciences

ENDARI（L-グルタミン）は、経口投与の医薬品グレードのL-グルタミン（PGLG）であり、成人および5歳以上の小児の鎌状赤血球貧血の疼痛、腫脹およびその他の合併症を緩和するアミノ酸製剤です。ENDARIは、酸化の主要な調節因子として同定された補酵素であるニコチンアミドアデニンジヌクレオチド（NAD）の酸化還元電位を改善することにより、赤血球の酸化的損傷を軽減します。

ENDARIは米国で希少疾病用医薬品指定（ODD）、EUで希少疾病用医薬品指定、FDAからファスト・トラック指定（FTD）を受けた。

2017年7月、FDAは、5歳以上の成人および小児患者における鎌状赤血球症の重篤な合併症の軽減を目的として、ENDARI（L-グルタミン経口粉末）を承認しました。

新たな治療薬

ミタピバット：Agios Pharmaceuticals

Mitapivatは、新規のファーストインクラスの経口低分子ピルビン酸キナーゼ酵素アロステリック活性化薬です。野生型および多数の変異型赤血球ピルビン酸キナーゼ（PKR）の両方を有意にアップレギュレートし、アデノシン三リン酸（ATP）産生を増加させ、2,3-ジホスホグリセリン酸レベルを低下させることが示されています。2022年2月、FDAはピルビン酸キナーゼ欠損症の成人における溶血性貧血の治療薬としてPYRUKYND（mitapivat）を承認しました。

同社は鎌状赤血球患者を対象にmitapivatを評価する第II/III相試験を開始しました。

2023年6月、Agios Pharmaceuticalsは、鎌状赤血球症を対象としたmitapivatの世界RISE UP試験の第II相試験において、mitapivat50mgおよび100mgの1日2回投与（BID）群の両群で、主要評価項目であるヘモグロビン反応性を達成したと発表しました。

インクラクマブ：Pfizer

インクラクマブは、P-セレクチンを選択的に標的とする新規の完全ヒト型モノクローナル抗体です。このタンパク質は細胞接着を媒介し、SCD患者におけるVOCによる疼痛を軽減することが臨床的に検証されています。前臨床試験の結果から、インクリクマブはSCD患者のVOCを軽減するためのクラス最高の選択肢となり得ることが示唆されており、月1回ではなく四半期ごとに投与できる可能性があります。同社は1つのフェーズⅡ試験を完了し、現在鎌状赤血球症の治療薬として臨床開発のフェーズⅢ段階にあります。

2022年10月、PfizerはGlobal Blood Therapeutics (GBT)の買収を決定しました。GBTは、鎌状赤血球症をはじめとする、十分な治療を受けていない患者コミュニティーに希望を与える、生命を変える治療法の発見、開発、提供に取り組むバイオ医薬品企業です。

鎌状赤血球症の患者にとって、効果的な治療法の確立は最も重要な課題です。現在の治療法は、疼痛や炎症の緩和、血管閉塞性クリーゼの頻度の減少、酸素供給の改善など、QOLを高める対症療法にとどまっています。また、輸血や細胞治療を繰り返すことで、SCD患者の症状管理を維持しています。SCDは米国中の多くの人々の現実です。しかし、患者は、特に専門家でないヘルスケアプロバイダーから、質の高い包括的なケアを提供するためのトレーニングを受けていない可能性がある、質の低いケアをしばしば経験します。これは、全体的な治療を提供するためのヘルスケアのインフラが乏しいことを示しており、それが満足のいかない症状管理を助長しています。

現在、治療法は薬物療法と非薬物療法に分類されます。薬理療法としては、DROXIA（ヒドロキシ尿素）、ENDARI（L-グルタミン）、ADAKVEO（クリザンリズマブ）、OXBRYTA（ボクセロトール）CASGEVY（exagamglogene autotemcel[exa-cel]）（Vertex Pharmaceuticals/CRISPR Therapeutics）、LYFGENIA（Lovo-cel）（bluebird bio）などがあります。疼痛管理薬は、オピオイド、非ステロイド性抗炎症薬（NSAIDs）、アセトアミノフェン、コルチコステロイドなどに分類されます。急性血管閉塞性クリーゼには、一般的にオピオイドと非ステロイド性抗炎症薬（NSAIDs）が使用されます。さらに、非薬物療法としては、認知行動療法、バイオフィードバック、リラクゼーション法、鍼治療、催眠療法などがあります。

ヒドロキシ尿素は経口薬であり、鎌状赤血球を減少させ、SCDのいくつかの合併症を軽減または予防することが示されています。ヒドロキシ尿素は、SCDの治療薬として初めてFDAに承認され、現在も第一選択薬として使用されています。ヒドロキシウレアで十分に症状が抑制されない場合、FDAが承認した新しい薬剤をヒドロキシウレアの治療に加えて併用療法を行うのが一般的です。

2023年12月、CRISPRベースの遺伝子治療薬であるCASGEVYとLYFGENIAが、鎌状赤血球貧血に対してFDAから承認を受けた。これらの治療法は異なる方法で作用しますが、両療法ともノーベル賞を受賞したCRISPR/Cas 9ゲノム編集技術を利用しています。

オピオイド鎮痛薬は、VOC管理における疼痛緩和の第一選択として推奨されています。オピオイド鎮痛薬には、コデイン、ヒドロコドン/パラセタモール（ヒドロコドン/アセトアミノフェン）、ヒドロコドン/イブプロフェン、オキシコドン（およびコデイン入り）、モルヒネ、ヒドロモルフォン、オキシモルフォン、メタドン、ジアモルフィン、フェンタニルなどがあります。オピオイドは一般的にヘルスケアで入手可能であり、モルヒネの静脈注射として4～6時間ごとに投与されることが多いです。しかし、鎮痛オピオイドには呼吸抑制、便秘、嘔吐、吐き気、そう痒症やじんましん、中毒、離脱症状などの副作用があり、処方が制限されています。

SCDのパイプラインには、開発後期および中期段階にある潜在的な薬剤があります。現在の主要参入企業とそれぞれの新薬候補には、Global Blood Therapeutics/Pfizer（Inclacumab）、Forma Therapeutics/Novo Nordisk（etavopivat）、Novo Nordisk（NDEC）、Bluebird Bio（lovo-cel）、Global Blood Therapeutics/Pfizer（GBT-601）、Agios Pharmaceuticals（mitapivat）、Bausch Health Americas（rifaximin）、Editas Medicine（EDIT-301）などがあります。

一言で言えば、SCDの治療法としていくつかの可能性が検討されているということです。予測期間（2024～2034年）中に市場に参入する上記の有望候補についてコメントするのは時期尚早であるとしても、この市場の将来は明るいと考えてよいと思われます。最終的に、この薬剤は今後数年間で、SCDの状況に大きな変化をもたらすと思われます。この治療領域は、世界中のヘルスケア支出数の増加の改善により、今後数年間で好影響を受けると予想されます。

鎌状赤血球症（SCD）は、ヘモグロビンに影響を及ぼす一群の終生遺伝性疾患です。赤血球内のヘモグロビン分子が重合して赤血球を鎌状（または三日月状）に変形させ（Hb S）、その結果、特徴的な血管閉塞性事象と溶血が加速する傾向を特徴とする慢性溶血性疾患です。

SCDは大血管炎に分類されますが、中動脈炎や小動脈炎も伴います。鎌状赤血球症は常染色体で遺伝し、ホモ接合体でも二重の異型接合体でも遺伝します。鎌状赤血球症は、ホモ接合体遺伝の場合、鎌状赤血球貧血（SCA）と呼ばれます。その他のSCD遺伝子型としては、ヘモグロビンSC病、鎌状赤血球プラスサラセミア、鎌状赤血球ゼロサラセミア（鎌状赤血球貧血と同様の重症度を有する）、ヘモグロビンSDパンジャブ病、ヘモグロビンSOアラブ病などが知られています。ヘモグロビンS（Hb S）は、Bグロビン遺伝子の6位がグルタミン酸からバリンに置換しているため、正常なヘモグロビン（Hb A）とは異なります。

米国では現在、出生時のHbSスクリーニングが義務付けられています。生後6ヶ月間は、胎児ヘモグロビン（HbF）レベルの上昇により、乳児はほぼ保護されます。SCDは通常、小児期の早期に様々な徴候や症状を伴って現れます。急性および慢性の疼痛、特に血管閉塞性クリーゼは、SCDの最も特徴的な臨床症状であり、しばしば骨髄梗塞による四肢長骨の骨痛を呈します。

鎌状赤血球症の症状は通常、生後4ヶ月までは現れません。この痛みは数時間から数日間続くことがあり、このような痛みのエピソードはクリーゼとも呼ばれます。毎年1回発症する人もいれば、毎年何回も発症する人もいます。クリーゼは重症化することもあり、入院を余儀なくされることもあります。鎌状赤血球症のすべての症状は酸素不足のためです。

鎌状赤血球症の診断は、欠陥遺伝子やヘモグロビン細胞を分析する血液検査から始まります。また、出生前や乳幼児期の早期診断に役立つ様々なスクリーニングプログラムもあります。SCDの管理は、疼痛エピソードやその他の合併症の予防と治療に重点を置いています。

様々なスクリーニングプログラムもあり、出生前や乳児期の早期診断に役立っています。

当レポートでは、主要6ヶ国における鎌状赤血球症（SCD）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 鎌状赤血球症（SCD）市場概要

• 2020年の主要6ヶ国におけるSCDの治療法別市場シェア（％）分布
• 2034年の主要6ヶ国におけるSCDの治療法別市場シェア（％）分布

第4章 鎌状赤血球症（SCD）のエグゼクティブサマリー

第5章 主要な出来事

第6章 疾患の背景と概要

• イントロダクション
• 鎌状赤血球症の分類
• 原因
• 関連するリスク要因
• 合併症
• 症状
• 病態生理学
• 診断
• 鎌状赤血球症の治療と管理
• 治療アルゴリズム
• 治療ガイドライン

第7章 調査手法

第8章 疫学と患者人口

• 主な調査結果
• 仮定と根拠
• 主要6ヶ国におけるSCDの総罹患数
• 主要6ヶ国におけるSCDの診断症例総数
• 主要6ヶ国におけるSCDの治療症例
• 米国
• EU4ヶ国と英国

第9章 患者動向

第10章 上市済み治療法

• キークロス競合
• ENDARI（L-グルタミン）：Emmaus Life Sciences
• ADAKVEO（クリザンリズマブ）：Novartis Pharmaceuticals
• OXBRYTA（ボクセロター）：Global Blood Therapeutics/Pfizer
• CASGEVY：Vertex Pharmaceuticals/CRISPR Therapeutics
• LYFGENIA（Lovo-cel）：Bluebird Bio

第11章 新たな治療法

• キークロス競合
• オシベロトル（GBT-601）：Global Blood Therapeutics/Pfizer
• 製造元：Agios Pharmaceuticals
• Renizgamglogene autogedtemcel（reni-cel/EDIT-301）：Editas Medicine
• インクラクマブ：Global Blood Therapeutics/Pfizer
• 製造元：Forma Therapeutics/Novo Nordisk
• NDec（デシタビンおよびテトラヒドロウリジン/EP101）：Novo Nordisk
• リファキシミン：Bausch Health

第12章 鎌状赤血球症（SCD）- 主要6ヶ国市場分析

• 主な調査結果
• 市場見通し
• コンジョイント分析
• 主要な市場予測の前提条件
• 主要6ヶ国におけるSCDの総市場規模
• 主要6ヶ国におけるSCDの市場規模（治療法別）
• 米国市場規模
• EU4ヶ国と英国の市場規模

第13章 KOLの見解

第14章 アンメットニーズ

第15章 SWOT分析

第16章 市場アクセスと償還

• 米国
• EU4ヶ国と英国

第17章 付録

第18章 報告書の調査手法

第19章 参考文献

第20章 DelveInsightのサービス内容

第21章 免責事項

第22章 DelveInsightについて

List of Tables

• Table 1: Summary of SCD Market and Epidemiology (2020-2034)
• Table 2: Molecular and Cellular Changes of Hemoglobin S.
• Table 3: Laboratory Test Schedule for Patients With SCD
• Table 4: Pharmacological Management of Pain Using WHO Three-step "Analgesic Ladder"
• Table 5: Non-narcotic Analgesics for Mild Pain in Sickle Cell Disease
• Table 6: Outline of Management of Acute Pain in Opioid Naive Adults
• Table 7: Outline of Management of Acute Pain in Opioid Naive Adults
• Table 8: Management of Acute Pain in Opioid-naive Adults
• Table 9: Effects of Therapy with Hydroxyurea
• Table 10: A German Consensus Recommendation
• Table 11: Current situation and future perspectives.
• Table 12: Recommendations from a Pan-European Consensus Conference.
• Table 13: A British Society for Hematology Guideline
• Table 14: Total Prevalent Cases of SCD in the 6MM (2020-2034)
• Table 15: Total Diagnosed Cases of SCD in the 6MM (2020-2034)
• Table 16: Treated Cases of SCD in the 6MM (2020-2034)
• Table 17: Total Prevalent Cases of SCD Trait in the United States, in thousands (2020-2034)
• Table 18: Total Prevalent Cases of SCD in the United States, (2020-2034)
• Table 19: Total Diagnosed Cases of SCD in the United States (2020-2034)
• Table 20: Age-specific Prevalent Cases of SCD in the United States (2020-2034)
• Table 21: Type-specific Prevalence of SCD in the United States (2020-2034)
• Table 22: Total Prevalent Cases of SCD Trait in EU4 and the UK, in thousands (2020-2034)
• Table 23: Total Prevalent Cases of SCD in the EU4 and the UK (2020-2034)
• Table 24: Total Diagnosed Cases of SCD in EU4 and the UK, (2020-2034)
• Table 25: Age-specific Prevalent Cases of SCD in EU4 and the UK (2020-2034)
• Table 26: Type-specific Prevalent Cases of SCD in the EU4 and the UK (2020-2034)
• Table 27: Key Cross (Marketed Therapies)
• Table 28: ENDARI (L-glutamine), Clinical Trial Description, 2024
• Table 29: Results From the ENDARI Clinical Trial in Sickle Cell Disease
• Table 30: ADAKVEO (crizanlizumab), Clinical Trial Description, 2024
• Table 31: Efficacy Results from SUSTAIN Trial in Sickle Cell Disease
• Table 32: OXBRYTA (voxelotor), Clinical Trial Description, 2024
• Table 33: Adjusted Mean Change From Baseline to Week 24 in Hemoglobin and Clinical Measures of Hemolysis
• Table 34: Exa-cel (CTX001), Clinical Trial Description, 2024
• Table 35: Lovo-cel, Clinical Trial Description, 2024
• Table 36: Key Cross (Emerging Therapies)
• Table 37: GBT-601, Clinical Trial Description, 2024
• Table 38: Mitapivat, Clinical Trial Description, 2024
• Table 39: EDIT-301, Clinical Trial Description, 2024
• Table 40: Inclacumab, Clinical Trial Description, 2024
• Table 41: Etavopivat, Clinical Trial Description, 2024
• Table 42: NDec (EPI 01), Clinical Trial Description, 2024
• Table 43: Rifaximin, Clinical Trial Description, 2024
• Table 44: Key Market Forecast Assumption of SCD in the US
• Table 45: Key Market Forecast Assumption of SCD in EU4 and the UK
• Table 46: Total Market Size of SCD in the 6MM, in USD million (2020-2034)
• Table 47: Market Size of SCD by Therapies in the 6MM, in USD million (2020-2034)
• Table 48: Total Market Size of SCD in the United States, in USD millions (2020-2034)
• Table 49: Market Size of SCD by Therapies in the United States, in USD million (2020-2034)
• Table 50: Total Market Size of SCD in EU4 and the UK, in USD million (2020-2034)

List of Figures

• Figure 1: Sickle Cell Disease Classification
• Figure 2: Sickle Cell Disease Complications
• Figure 3: Common Symptoms Associated With Sickle Cell Disease
• Figure 4: HbS Polymerization and Erythrocyte Deformation
• Figure 5: Mechanism in Sickle Cell Disease
• Figure 6: Measure of Pain Intensity and Location
• Figure 7: Treatment Algorithm
• Figure 8: Total Prevalent Cases of SCD in the 6MM (2020-2034)
• Figure 9: Total Diagnosed Cases of SCD in the 6MM (2020-2034)
• Figure 10: Treated Cases of SCD in the 6MM (2020-2034)
• Figure 11: Total Prevalent Cases of SCD Trait in the United States (2020-2034)
• Figure 12: Total Prevalent Cases of SCD in the United States (2020-2034)
• Figure 13: Total-Diagnosed Cases of SCD in the United States (2020-2034)
• Figure 14: Age-specific Prevalent Cases of SCD in the United States (2020-2034)
• Figure 15: Type-specific Prevalence of SCD in the United States (2020-2034)
• Figure 16: Total Prevalent Cases of SCD Trait in EU4 and the UK (2020-2034)
• Figure 17: Total Prevalent Cases of SCD in the EU4 and the UK (2020-2034)
• Figure 18: Total Diagnosed Cases of SCD in the EU4 and the UK (2020-2034)
• Figure 19: Age-specific Prevalent Cases of SCD in EU4 and the UK (2020-2034)
• Figure 20: Type-specific Prevalent Cases of SCD in EU4 and the UK (2020-2034)
• Figure 21: Total Market Size of SCD in the 6MM (2020-2034)
• Figure 22: Market Size of SCD by Therapies in the 6MM (2020-2034)
• Figure 23: Total Market Size of SCD in the United States (2020-2034)
• Figure 24: Market Size of SCD by Therapies in the United States (2020-2034)
• Figure 25: Total Market Size of SCD in EU4 and the UK (2020-2034)
• Figure 26: Market Size of SCD by Therapies in EU4 and the UK (2020-2034)
• Figure 27: Unmet Needs
• Figure 28: Health Technology Assessment
• Figure 29: Reimbursement Process in Germany
• Figure 30: Reimbursement Process in France
• Figure 31: Reimbursement Process in Italy
• Figure 32: Reimbursement Process in Spain
• Figure 33: Reimbursement Process in the United Kingdom

Key Highlights:

• Sickle cell disease (SCD) is a genetic blood disorder characterized by the presence of abnormal hemoglobin, causing red blood cells to become sickle-shaped. SCD is a global health concern, particularly prevalent in populations with African, Middle Eastern, Mediterranean, and South Asian ancestry.
• The primary cause of SCD is a mutation in the HBB gene, which affects the production of hemoglobin. Individuals with SCD inherit two copies of the mutated gene, one from each parent, resulting in abnormal hemoglobin production. The abnormal red blood cells tend to become rigid and sticky, obstructing blood flow and causing episodes of intense pain called sickle cell crises.
• SCD poses significant challenges for affected individuals, impacting their quality of life and life expectancy. Common complications include anemia, acute chest syndrome, stroke, organ damage (e.g., kidneys, liver), and increased susceptibility to infections.
• Currently, NSAIDs, blood transfusions, chelating agents, nutritional supplements, and broad-spectrum antibiotics are being used for the treatment of SCD. Moreover, a few therapies that the US FDA has approved for the treatment of sickle cell disease include DROXIA (hydroxyurea), ENDARI (L-glutamine oral powder), ADAKVEO (crizanlizumab-tmca), and OXBRYTA (voxelotor).
• Improving the overall care and outcomes for individuals with SCD requires a multidisciplinary approach. This includes comprehensive healthcare services, genetic counseling, early detection, and management of complications. Patient education and support programs are crucial for empowering individuals with SCD to manage their condition effectively and improve their quality of life.
• Research and development efforts are ongoing to explore new treatments and potential cures for SCD. Gene therapy and gene editing techniques hold promise in correcting the underlying genetic mutation responsible for SCD. Clinical trials are underway to assess the safety and efficacy of these emerging therapies, providing hope for a future where SCD can be cured or effectively managed.
• The disease's life-threatening nature and lack of effective therapies are a big burden, and till the last decade, only hydroxyurea was available for management. Even after being in the market for so long, there remains a challenge of low treatment adherence and high treatment discontinuation rates.
• Adherence and reimbursement of the therapies are also still challenging with the recently approved therapies. With almost three recent introductions in the market, i.e., ENDARI (L-glutamine), ADAKVEO (crizanlizumab), and OXBRYTA (voxelotor). The recently approved OXBRYTA became the first-in-class oral therapy that targets the underlying cause of SCD with a convenient route of administration.
• The United States accounted for the highest market size, with nearly ~92% of the market share of the SCD market as compared to EU4 and the UK in 2023.
• In 2023, among EU4 and the UK, France accounted for the largest market size, while Spain accounted for the smallest share.
• Few therapies have been recently approved for sickle cell disease, which include CASGEVY (exagamglogene autotemcel [exa-cel]) (Vertex Pharmaceuticals/CRISPR Therapeutics) and LYFGENIA (Lovo-cel) (bluebird bio). Approval of these therapies is expected to have a positive impact on overall growth of the market. In contrast, the emerging pipeline includes drugs like EDIT-301, Mitapivat, Inclacumab (PF-07940370), Osivelotor (GBT-601), and others.
• Delays in diagnosis, serious complications of SCD, economic burden, and lack of proper understanding of the disease will be going to hit the SCD market.
• In conclusion, SCD is a complex genetic blood disorder characterized by abnormal hemoglobin, leading to complications and reduced life expectancy. However, advancements in treatment approaches and ongoing research efforts offer hope for improved management and potential cures. Enhancing healthcare services, raising awareness, and supporting individuals with SCD are essential steps toward addressing the challenges associated with this disease in the 6MM.

Report Summary

• The report offers extensive knowledge regarding the epidemiology segments (by region, total prevalent cases of SCD trait, total prevalent cases of SCD, diagnosed cases of SCD, age-specific prevalent cases of SCD, and type-specific prevalence of SCD) and predictions, presenting a deep understanding of the potential future growth in diagnosis rates, disease progression, and treatment guidelines. It provides comprehensive insights into these aspects, enabling a thorough assessment of the subject matter.
• Additionally, an all-inclusive account of the current management techniques and emerging therapies such as Mitapivat, EDIT-301 (Renizgamglogene autogedtemcel, or reni-cel), Inclacumab (PF-07940370), Osivelotor (GBT-601) and the elaborative profiles of late and mid-stage (Phase III and Phase II) and prominent therapies that would impact the current treatment landscape and result in an overall market shift has been provided in the report.
• The report also encompasses a comprehensive analysis of the SCD market, providing an in-depth examination of its historical and projected market size (2020-2034). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology. The report also includes drug outreach coverage in the 6MM region.
• The report includes qualitative insights that provide an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, including experts from various hospitals and prominent universities, patient journey, and treatment preferences that help shape and drive the 6MM SCD market.

Market

Various key players, such as Pfizer, Agios Pharmaceuticals, Editas Medicine, Hoffmann-La Roche and others, are involved in developing therapies for SCD. The expected launch of emerging therapies and other treatments will lead to a significant increase in the market size during the forecast period [2024-2034].

• In 2023, the total market size of SCD was approximately USD 650 million, which is expected to increase by 2034 during the study period (2020-2034) in the 6MM.
• Among the 6MM, the United States accounted for the highest market share in 2023, followed by France, and the UK for SCD.
• During the forecast period (2024-2034), pipeline candidates such as Etavopivat, Inclacumab (PF-07940370), Reni-cel (EDIT-301) and Osivelotor (GBT-601) are expected to be the major contributors for driving the rise in SCD market size.
• Among the emerging therapies, Casgevy (Exa-cel) is expected to garner the largest market share by 2034 in the 6MM.
• Gene therapies come up with many challenges, such as market access and reimbursements, and target a niche pool hence having a lower market share.

SCD Drug Chapters

The section dedicated to drugs in the SCD report provides an in-depth evaluation of pipeline drugs (Phase III and Phase II) related to SCD.

The drug chapters section provides valuable information on various aspects related to clinical trials of SCD, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting SCD.

Marketed Therapies

CASGEVY (exagamglogene autotemcel): Vertex Pharmaceuticals/CRISPR Therapeutics

CASGEVY is a genome-edited cellular therapy consisting of autologous CD34+ hematopoietic stem cells (HSCs) edited by CRISPR/Cas9 technology at the erythroid-specific enhancer region of the BCL11A gene. CASGEVY is intended for one-time administration via a hematopoietic stem cell transplant procedure where the patient's own CD34+ cells are modified to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin (HbF) production. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate vaso-occlusive crises for patients with SCD.

In December 2023, Vertex Pharmaceuticals and CRISPR Therapeutics announced that the US FDA had approved CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

CASGEVY was later granted conditional marketing authorization in Great Britain by the UK. Medicines and Healthcare Products Regulatory Agency and by the National Health Regulatory Authority in Bahrain for patients 12 years of age and older with SCD characterized by recurrent vaso-occlusive crises or transfusion-dependent beta-thalassemia (TDT), for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available.

ENDARI (L-glutamine): Emmaus Life Sciences

ENDARI (L-glutamine) is an oral-administered pharmaceutical grade L-glutamine (PGLG), an amino acid formulation to relieve pain, swelling, and other complications of sickle cell anemia in adults and children 5 years and older. ENDARI reduces oxidant damage to red blood cells by improving the redox potential of nicotinamide adenine dinucleotide (NAD), a coenzyme identified as the primary regulator of oxidation.

ENDARI received Orphan Drug designation (ODD) in the US, Orphan Medicinal Product designation in the EU, and Fast Track designation (FTD) from the FDA.

In July 2017, the FDA approved ENDARI (L-glutamine oral powder) to reduce the severe complications of sickle cell disease in adult and pediatric patients aged 5 and older.

Emerging Therapies

Mitapivat: Agios Pharmaceuticals

Mitapivat is a novel, first-in-class oral small molecule allosteric activator of the pyruvate kinase enzyme. It has been shown to significantly upregulate both wild-type and numerous mutant forms of erythrocyte pyruvate kinase (PKR), increasing adenosine triphosphate (ATP) production and reducing levels of 2,3-diphosphoglycerate. In February 2022, the FDA approved PYRUKYND (mitapivat) to treat hemolytic anemia in adults with pyruvate kinase deficiency.

The company has initiated a Phase II/III trial to evaluate mitapivat in sickle cell patients.

In June 2023, Agios Pharmaceuticals announced that the Phase II portion of the global RISE UP study of mitapivat in sickle cell disease had met its primary endpoint of hemoglobin response for patients in both the 50 mg and 100 mg twice daily (BID) mitapivat arms.

Inclacumab: Pfizer

Inclacumab is a novel, fully human monoclonal antibody that selectively targets P-selectin. This protein mediates cell adhesion and is clinically validated to reduce pain due to VOCs in people with SCD. Preclinical results suggest that inclacumab can be a best-in-class option for reducing VOCs in people with SCD, with the potential for quarterly rather than monthly dosing. The company has completed one Phase II study, and it is currently in the Phase III stage of clinical development for the treatment of Sickle cell disease.

In October 2022, Pfizer competed the acquisition of Global Blood Therapeutics (GBT), a biopharmaceutical company dedicated to the discovery, development, and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease.

SCD Market Outlook

An effective cure for a disease is the utmost requirement in SCD patients. Current therapies only provide symptomatic treatment such as relief in pain crises, inflammation, reduction in the frequency of vaso-occlusive crisis, improved oxygen supply, etc., enhancing quality of life. Recurring blood transfusion and cell therapies also sustain symptom management in SCD patients. SCD is the reality of many people across the US. Yet, patients often experience poor care, especially from non-specialist healthcare providers who may lack the training to provide good, comprehensive care. It shows the scarcity of healthcare infrastructure to provide holistic treatment, which fuels unsatisfactory symptom management.

The treatment pattern currently consists of different approaches classified into pharmacologic and nonpharmacological therapies. The pharmacological therapies, including DROXIA (hydroxyurea), ENDARI (L-glutamine), ADAKVEO (crizanlizumab), OXBRYTA (voxelotor) CASGEVY (exagamglogene autotemcel [exa-cel]) (Vertex Pharmaceuticals/CRISPR Therapeutics) and LYFGENIA (Lovo-cel) (bluebird bio). Pain management agents are segregated into opioids, nonsteroidal anti-inflammatory drugs (NSAIDs), acetaminophen, corticosteroids, etc. Acute vaso-occlusive crisis is generally managed using opioids and nonsteroidal anti-inflammatory drugs (NSAIDs). Further, non-pharmacological therapies include cognitive behavioral therapy, biofeedback, relaxation techniques, acupuncture, hypnosis, etc.

Hydroxyurea is an oral medicine that has been shown to reduce sickling and reduce or prevent several complications of SCD. This was the first medication approved by the FDA to treat SCD and is still used as a first-line treatment. If hydroxyurea does not control symptoms enough, the newer FDA-approved drugs are typically added on top of hydroxyurea treatment for combination therapy.

In December 2023, CASGEVY and LYFGENIA, CRISPR-based gene therapies, received approval from the FDA for sickle cell anemia. These therapies work in different ways; however, both therapies utilize the Nobel-winning CRISPR/Cas 9 genome editing technology.

Opioid analgesics are recommended as the primary choice of pain relief in VOC management. They include codeine, hydrocodone/paracetamol (hydrocodone/acetaminophen), hydrocodone/ibuprofen, oxycodone (and with codeine), morphine, hydromorphone, oxymorphone, methadone, diamorphine, and fentanyl. Opioids are generally available in healthcare settings and are often delivered as intravenous (IV) morphine every 4-6 h. However, the adverse effects of analgesic opioids include respiratory depression, constipation, vomiting, nausea, pruritus and hives, addiction, withdrawals, etc., limit their prescriptions.

The SCD pipeline possesses potential drugs in the late and mid-development stages. The current key players and their respective drug candidates include Global Blood Therapeutics/Pfizer (Inclacumab), Forma Therapeutics/Novo Nordisk (etavopivat), Novo Nordisk (NDEC), Bluebird Bio (lovo-cel), Global Blood Therapeutics/Pfizer (GBT-601), Agios Pharmaceuticals (mitapivat), Bausch Health Americas (rifaximin), Editas Medicine (EDIT-301), and others.

In a nutshell, a few potential therapies are being investigated for the management of SCD. Even though it is too soon to comment on the above-mentioned promising candidate to enter the market during the forecast period (2024-2034), it is safe to assume that the future of this market is bright. Eventually, the drug will create a significant difference in the landscape of SCD in the coming years. The treatment space is expected to experience a positive impact in the coming years owing to the improvement in the rise in the number of healthcare spending across the world.

SCD Disease Understanding and Treatment

SCD Overview

Sickle Cell Disease (SCD) is a group of lifelong inherited conditions that affect hemoglobin. It is characterized as a chronic hemolytic disorder marked by the tendency of hemoglobin molecules within red blood cells to polymerize and deform the red cell into sickle (or crescent) shape (Hb S), resulting in characteristic vaso-occlusive events and accelerated hemolysis.

SCD is classified as a large-vessel vasculitis but also involves medium and small arteritis; sickle cell disease is inherited in an autosomal fashion, whether in the homozygous or double heterogeneous state. Sickle cell disease is called sickle cell anemia (SCA) when there is an inheritance in the homozygous state. Other known SCD genotypes include hemoglobin SC disease, sickle beta plus thalassemia, sickle beta zero thalassemia (which has similar severity with sickle cell anemia), hemoglobin SD Punjab disease, hemoglobin SO Arab disease, and others. Hemoglobin S (Hb S) differs from normal hemoglobin (Hb A) because of the substitution of valine for glutamic acid in the sixth position in the B-globin gene.

Screening for HbS at birth is currently mandatory in the United States. For the first 6 months of life, infants are largely protected by elevated levels of fetal hemoglobin (HbF). SCD usually manifests early in childhood with various signs and symptoms. Acute and chronic pain, particularly vaso-occlusive crises, are the most distinguishing clinical features of SCD, often presenting as bone pain in the long bones of the extremities due to bone marrow infarction

Symptoms of sickle cell disease usually do not occur until the age of 4 months; the prevalent symptom includes painful episodes. This pain can last from a few hours to days; these painful episodes are also known as crises. Some people have one episode every year; others have many episodes each year. Crises can be severe, which leads to hospital stays. All the symptoms of sickle cell disease are because of a lack of oxygen.

SCD Diagnosis

Sickle cell disease diagnosis starts with a blood test that is analyzed for defective genes or hemoglobin cells. Various screening programs also help in the early diagnosis of the disease during the prenatal or infancy period. SCD management focuses on preventing and treating pain episodes and other complications.

Various screening programs are also there that help in early diagnosis of the disease during the prenatal or infancy period.

Blood tests

A person can go for the screening blood test to differentiate sickle hemoglobin (hemoglobin S) or another hemoglobin (such as C, B-thalassemia, E).

Newborn screening

Diagnosing SCD early in a child is very important to prevent further complications. All babies born in most developed countries are offered screening for sickle cell disease shortly following the birth. In newborn screening programs, blood from a heel prick test is collected in "spots" on a special paper. A second test should be done to confirm the diagnosis if the test is positive.

Prenatal diagnosis

Prenatal diagnosing is done on the baby before it is born to know whether the baby is suffering from any particular disease. Different types of tests that are used include:

Chorionic villus sampling

Fetal blood sampling

Amniocentesis

DNA analysis

This test can be used to investigate alterations and mutations in the gene that produces hemoglobin components. This test may be performed to determine whether someone has one or two copies of the Hb S mutation or has two different mutations in hemoglobin genes (e.g., Hb S and Hb C). Genetic testing is most often used for prenatal testing. This is done using a sample of amniotic fluid, the liquid in the sac surrounding a growing embryo, or a tissue taken from the placenta.

SCD Treatment

The treatment goals for sickle cell disease aims to relieve pain, prevent infections, and specifically manage complications. Treatment of Sickle cell disease can be divided into First line treatment and second line treatment. The first line treatment includes management of pain, vaso-occlusive crisis, and chronic symptoms by using various medications, Second line treatment includes gene therapy and bone marrow transplantation.

Patients with SCD use medications to make their disease less severe and treat symptoms. FDA approved medications include Voxelotor, Crizanlizumab, Hydroxyurea, L-glutamine, and others.

Moreover, NSAIDS, Opioids, Iron chelating agents, Antibiotics, Folic acid and others are used for the pain and other complications associated with SCD.

Acute sickle cell crises are managed primarily with drug therapy, psychologic supportive care, including oxygen. The standard treatment approach includes opioid analgesics, adequate hydration, and rest. Initial management should be aimed at providing rapid pain control. Pain management should follow the three-step "analgesic ladder" recommended by the World Health Organization for treating cancer-related pain. The choice of analgesic and dosage should be based on the severity of pain in the individual patient.

Patients with mild pain can often be treated with oral fluids and non-narcotic analgesics at home. Acetaminophen with or without codeine or oxycodone (Roxicodone), depending on pain severity, is started first. Nonsteroidal anti-inflammatory drugs (NSAIDs) can be used unless specifically contraindicated due to peptic ulcer disease, renal disease, or hepatic dysfunction. Narcotic analgesics can be used in patients with moderate to severe pain. Pain which is sufficiently severe that require an emergency department visit or hospitalization should be treated with stronger opioids.

Supportive care with oxygen therapy, hydration using fluid replacement, antibiotics in case of infection and transfusion should be considered while managing vaso-occlusive crises in SCD patients.

The major goals in chronic disease management are symptom control and prevention of disease complications. Hydroxyurea should be used in patients with severe complications who can reliably follow the regimen. Hydroxyurea reduces the frequency of painful crises and the need for blood transfusions in patients with recurrent painful crises.

The only chance for a cure for Sickle Cell Disease is bone marrow or stem cell transplantation. The bone marrow nurtures stem cells, which are early cells that mature into red and white blood cells and platelets. Normal hemoglobin may be produced by destroying the sickle cell patient's diseased bone marrow and stem cells and transplanting healthy bone marrow from a genetically-matched donor.

SCD Epidemiology

The SCD epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of SCD trait, total prevalent cases of SCD, diagnosed cases of SCD, age-specific prevalent cases of SCD and type-specific prevalence of SCD cases in the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2020 to 2034.

• In 6MM, the United States accounted for the highest number of total prevalent cases of SCD in 2023.
• In the US, among the type-specific prevalent cases of SCD, Sickle cell anemia (hemoglobin S/S or hemoglobin S/B0-thalassemia) was the major subtype of SCD in 2023.
• Among the EU4 and the UK, France accounted for the highest number of prevalent SCD cases, followed by the UK, whereas Spain accounted for the lowest number of prevalent SCD cases.

KOL Views

To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.

We have reached out to industry experts to gather insights on various aspects of SCD, including the evolving treatment landscape, patients' reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.

Our team of analysts at Delveinsight connected with more than 10 KOLs across the 6MM. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the SCD market, which will assist our clients in analyzing the overall epidemiology and market scenario.

Qualitative Analysis

We perform Qualitative and Market Intelligence analysis using various approaches, such as SWOT analysis 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, designation, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy. In efficacy, the trial's primary and secondary outcome measures are evaluated. Based on these, the overall efficacy is evaluated.

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

Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.

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.

SCD Report Insights

• Patient Population
• Therapeutic Approaches
• SCD Market Size and Trends
• Existing Market Opportunity

SCD Report Key Strengths

• Eleven-year Forecast
• The 6MM Coverage
• SCD Epidemiology Segmentation
• Key Cross Competition

SCD Report Assessment

• Current Treatment Practices
• Reimbursements
• Market Attractiveness
• Qualitative Analysis (SWOT, Conjoint Analysis, Unmet needs)

Key Questions:

• Would there be any changes observed in the current treatment approach?
• Will there be any improvements in SCD management recommendations?
• Would research and development advances pave the way for future tests and therapies for SCD?
• Would the diagnostic testing space experience a significant impact and lead to a positive shift in the treatment landscape of SCD?
• How much market share will newly approved gene therapy will be able to capture and what are the chances of getting reimbursement across the 6MM.
• What kind of uptake will the new therapies witness in coming years in SCD patients?

Table of Contents

1. Key Insights

2. Report Introduction

3. Sickle Cell Disease (SCD) Market Overview at a Glance

• 3.1. Market Share (%) Distribution of SCD by Therapies in the 6MM in 2020
• 3.2. Market Share (%) Distribution of SCD by Therapies in the 6MM in 2034

4. Executive Summary of Sickle Cell Disease (SCD)

5. Key Events

6. Disease Background and Overview

• 6.1. Introduction
• 6.2. Classification of Sickle Cell Disease
• 6.3. Causes
• 6.4. Associated Risk Factors
• 6.5. Complications
• 6.6. Symptoms
• 6.7. Pathophysiology
• 6.8. Diagnosis
  • 6.8.1. Differential Diagnosis
• 6.9. Treatment and Management of Sickle Cell Disease
  • 6.9.1. Vaso-occlusive Crisis
  • 6.9.2. Chronic Disease
• 6.10. Treatment Algorithm
• 6.11. Treatment Guidelines
  • 6.11.1. American Society of Hematology 2021 Guidelines for Sickle Cell Disease: Stem Cell Transplantation
  • 6.11.2. American Society of Hematology 2020 Guidelines for Sickle Cell Disease: Management of Acute and Chronic Pain
  • 6.11.3. American Society of Hematology 2020 Guidelines for Sickle Cell Disease: Prevention, Diagnosis, and Treatment of Cerebrovascular Disease in Children and Adults
  • 6.11.4. American Society of Hematology 2020 Guidelines for Sickle Cell Disease: Transfusion Support
  • 6.11.5. American Society of Hematology 2019 Guidelines for Sickle Cell Disease: Cardiopulmonary and Kidney Disease
  • 6.11.6. Transition in Sickle Cell Disease (SCD): A German Consensus Recommendation
  • 6.11.7. Management of Children With Sickle Cell Disease in Europe: Current Situation and Future Perspectives
  • 6.11.8. Newborn Screening for Sickle Cell Disease in Europe: Recommendations from a Pan-European Consensus Conference
  • 6.11.9. Management of Sickle Cell Disease in Pregnancy: A British Society for Hematology Guideline

7. Methodology

8. Epidemiology and Patient Population

• 8.1. Key Findings
• 8.2. Assumptions and Rationale
• 8.3. Total Prevalent Cases of SCD in the 6MM
• 8.4. Total Diagnosed Cases of SCD in the 6MM
• 8.5. Treated Cases of SCD in the 6MM
• 8.6. The United States
  • 8.6.1. Total Prevalent Cases of SCD Trait in the United States
  • 8.6.2. Total Prevalent Cases of SCD in the United States
  • 8.6.3. Total Diagnosed Cases of SCD in the United States
  • 8.6.4. Age-specific Prevalent Cases of SCD in the United States
  • 8.6.5. Type-specific Prevalence of SCD in the United States
• 8.7. EU4 and the UK
  • 8.7.1. Total Prevalent Cases of SCD Trait in the EU4 and the UK
  • 8.7.2. Total Prevalent Cases of SCD in the EU4 and the UK
  • 8.7.3. Diagnosed Cases of SCD in the EU4 and the UK
  • 8.7.4. Age-specific Prevalent Cases of SCD in EU4 and the UK
  • 8.7.5. Type-specific Prevalent Cases of SCD in EU4 and the UK

9. Patient Journey

10. Marketed Therapies

• 10.1. Key Cross Competition
• 10.2. ENDARI (L-glutamine): Emmaus Life Sciences
  • 10.2.1. Product Description
  • 10.2.2. Regulatory Milestones
  • 10.2.3. Other Developmental Activities
  • 10.2.4. Clinical Development
  • 10.2.5. Safety and Efficacy
• 10.3. ADAKVEO (crizanlizumab): Novartis Pharmaceuticals
  • 10.3.1. Product Description
  • 10.3.2. Regulatory Milestones
  • 10.3.3. Other Developmental Activities
  • 10.3.4. Clinical Development
  • 10.3.5. Safety and Efficacy
• 10.4. OXBRYTA (voxelotor): Global Blood Therapeutics/Pfizer
  • 10.4.1. Product Description
  • 10.4.2. Regulatory Milestones
  • 10.4.3. Other Developmental Activities
  • 10.4.4. Clinical Development
  • 10.4.5. Safety and Efficacy
• 10.5. CASGEVY: Vertex Pharmaceuticals/CRISPR Therapeutics
  • 10.5.1. Product Description
  • 10.5.2. Regulatory Milestone
  • 10.5.3. Other Developmental Activities
  • 10.5.4. Clinical Developmental Activities
  • 10.5.5. Safety and Efficacy
• 10.6. LYFGENIA (Lovo-cel): Bluebird Bio
  • 10.6.1. Product Description
  • 10.6.2. Regulatory Milestone
  • 10.6.3. Other Developmental Activities
  • 10.6.4. Clinical Developmental Activities
  • 10.6.5. Safety and Efficacy

11. Emerging Therapies

• 11.1. Key Cross Competition
• 11.2. Osivelotor (GBT-601): Global Blood Therapeutics/Pfizer
  • 11.2.1. Product Description
  • 11.2.2. Other Developmental Activities
  • 11.2.3. Clinical Developmental Activities
  • 11.2.4. Safety and Efficacy
  • 11.2.5. Analyst View
• 11.3. Mitapivat: Agios Pharmaceuticals
  • 11.3.1. Product Description
  • 11.3.2. Other Developmental Activities
  • 11.3.3. Clinical Developmental Activities
  • 11.3.4. Safety and Efficacy
  • 11.3.5. Analyst View
• 11.4. Renizgamglogene autogedtemcel (reni-cel/EDIT-301): Editas Medicine
  • 11.4.1. Product Description
  • 11.4.2. Other Developmental Activities
  • 11.4.3. Clinical Developmental Activities
  • 11.4.4. Safety and Efficacy
  • 11.4.5. Analyst view
• 11.5. Inclacumab: Global Blood Therapeutics/Pfizer
  • 11.5.1. Product Description
  • 11.5.2. Other Developmental Activities
  • 11.5.3. Clinical Developmental Activities
  • 11.5.4. Safety and Efficacy
  • 11.5.5. Analyst View
• 11.6. Etavopivat: Forma Therapeutics/Novo Nordisk
  • 11.6.1. Product Description
  • 11.6.2. Other Developmental Activities
  • 11.6.3. Clinical Developmental Activities
  • 11.6.4. Safety and Efficacy
  • 11.6.5. Analyst View
• 11.7. NDec (decitabine and tetrahydrouridine/EP101): Novo Nordisk
  • 11.7.1. Product Description
  • 11.7.2. Other Developmental Activities
  • 11.7.3. Clinical Developmental Activities
  • 11.7.4. Safety and Efficacy
  • 11.7.5. Analyst View
• 11.8. Rifaximin: Bausch Health
  • 11.8.1. Product Description
  • 11.8.2. Other Developmental Activities
  • 11.8.3. Clinical Developmental Activities
  • 11.8.4. Analyst View

12. Sickle Cell Disease (SCD) - Six Major Market Analysis

• 12.1. Key Findings
• 12.2. Market Outlook
• 12.3. Conjoint Analysis
• 12.4. Key Market Forecast Assumptions
• 12.5. Total Market Size of SCD in the 6MM
• 12.6. Market Size of SCD by Therapies in the 6MM
• 12.7. United States Market Size
  • 12.7.1. Total Market Size of SCD in the United States
  • 12.7.2. Market Size of SCD by Therapies in the United States
• 12.8. EU4 and the UK Market Size
  • 12.8.1. Total Market Size of SCD in EU4 and the UK
  • 12.8.2. Market Size of SCD by Therapies in EU4 and the UK

13. KOL Views

14. Unmet Needs

15. SWOT Analysis

16. Market Access and Reimbursement

• 16.1. The United States
  • 16.1.1. Center for Medicare & Medicaid Services (CMS)
  • 16.1.2. OXBRYTA
  • 16.1.3. ENDARI
  • 16.1.4. ADAKVEO
  • 16.1.5. CASGEVY
• 16.2. EU4 and the UK
  • 16.2.1. Germany
  • 16.2.2. France
  • 16.2.3. Italy
  • 16.2.4. Spain
  • 16.2.5. United Kingdom

17. Appendix

18. Report Methodology

19. Bibliography

20. DelveInsight Capabilities

21. Disclaimer

22. About DelveInsight