脊髄性筋萎縮症治療市場規模、シェア＆動向分析レポート：タイプ別（1型、2型）、治療法別（遺伝子治療、薬剤）、薬剤別（スピンラザ、ゾルゲンマ）、投与経路別、地域別、セグメント別予測、2022～2030年版

脊髄性筋萎縮症治療市場の成長と動向

Grand View Research, Inc.の新しい調査によると、世界の脊髄性筋萎縮症治療市場規模は2030年までに180億米ドルに達すると予測されています。これは、2022年から2030年にかけてCAGR18.6％で拡大すると予測されています。この成長は、製品の承認や上市が増加していることに起因していると考えられます。例えば、2020年8月には、ジェネンテック社が開発したEvrysdi（リスディプラム）が、生後2カ月以上の脊髄性筋萎縮症（SMA）の小児患者の治療薬として食品医薬品局から承認されています。

また、2020年5月、ノバルティスAGは、欧州委員会（EC）からSMAの候補であるZolgensma（onasemnogene abeparvovec）に対して、SMAの患者さんの治療に対する条件付承認を取得しました。欧州で唯一承認された遺伝子治療薬です。2021年3月には、PTCセラピューティクス社が、Evrysdi（risdiplam）について、1型、2型、3型の脊髄性筋萎縮症患者の治療を適応としてEMAから販売承認を取得しました。このように、製品承認数の増加が脊髄性筋萎縮症治療薬市場を牽引すると期待されています。

ノバルティスAGは、世界マネージドアクセスプログラム（MAP）を通じて薬を無償で提供し、SMA患者を支援しています。このプログラムでは、北米、南米、アジア、オーストラリア、欧州、アフリカなどの大陸で、対象となるSMA患者にゾルゲンスマ100回分を無料で提供しています。このように、ゾルゲンスマに対するこのような支援プログラムの存在は、プロモーション活動を強化し、収益を上げる助けにもなっています。

世界のSMAの新生児スクリーニングと診断ツールへの容易なアクセスは、日常的なアプローチに含まれる必要があります。これにより、早期診断と治療へのタイムリーなDeepLが保証されるでしょう。例えば、2021年7月、ノースカロライナ州チャペルヒルのUNCメディカルセンターで、シャーロットの赤ちゃんが、アーリーチェック新生児スクリーニングプログラムを通じて脊髄性筋萎縮症と診断され、脊髄性筋萎縮症（SMA）の画期的な遺伝子治療治療を受けた。このようなスクリーニングプログラムにより、疾患の早期診断と治療が促進されると期待されています。

さらに、製薬会社は、脊髄性筋萎縮症患者の治療のための新規治療法を開発するために、研究機関といくつかの大規模な共同研究を行っています。例えば、2021年3月、PTCセラピューティクス社、およびThe Spinal Muscular Atrophy Foundationは、科学研究の推進とSMA患者を治療するための新規治療法の開発を目的とした研究協力関係を締結しました。

しかし、治療に関連する高いコストと、臨床試験目的の参加企業の少なさが、予測期間中の市場成長を抑制する可能性があります。オーファンドラッグの研究開発に関連する高コストは、製品価格上昇の主な要因となっています。例えば、最も一般的に使用されているスピンラザは、注射1回につき約125,000米ドル、年間750,000米ドルの費用がかかります。さらに、Zolgensma（AVXS-101）は、1回の治療で210万米ドルかかります。

脊髄性筋萎縮症治療薬市場レポートハイライト

製品タイプ別では、1型脊髄性筋萎縮症患者の有病率が高く、治療用製品が広く出回っていることから、1型セグメントが2021年に最大の市場シェアを占めた

治療法別では、遺伝子治療分野が市場浸透率の上昇により予測期間中に最も成長すると予想されるゾルゲンマは、SMA患者の治療法として37カ国で承認されている唯一の遺伝子治療法です。

薬剤別では、支持的な償還政策の存在と製品に関する認知度の上昇により、2021年にはスピンラザセグメントが市場を独占しました。現在、スピンラザはイタリア、ノルウェー、オランダの欧州3カ国で全額償還の対象になっています。

投与経路別では、SMA患者への点滴投与に伴う高い安全性、有効性、忍容性から、注射剤が2021年の市場を独占

予測期間中、アジア太平洋地域が最も急速に成長すると予想されます。この地域の成長は、新製品がこの地域に参入したことに起因しています。例えば、2021年7月、F. Hoffmann-La Roche Ltdは、インドで脊髄性筋萎縮症患者の治療薬としてEvrysdi（risdiplam）を発売しました。

目次

第1章 調査手法と範囲

• 市場セグメンテーション
• 調査手法
• 情報調達
  • 購入したデータベース
  • GVRの内部データベース
  • 二次情報
  • 1次調査
  • 1次調査内容
  • 一次情報一覧
• 情報またはデータ分析
  • データ分析モデル
• 市場の策定と検証
• モデルの詳細
  • コモディティフロー分析
    • アプローチ1：コモディティフローアプローチ
    • アプローチ2：ボトムアップアプローチを使用した国別の市場推定
• 世界市場：CAGR計算
• 調査の仮定
• 二次情報一覧
• 略語一覧
• 目的

第2章 エグゼクティブサマリー

• 市場概要

第3章 市場変数、動向、範囲

• 市場系列の見通し
  • 親会社の市場見通し
• 規制の枠組み
• 浸透と成長の見通しのマッピング
• パイプライン分析
• 市場力学
  • 市場促進要因分析
  • 市場抑制要因分析
• PESTEL分析

第4章 世界の脊髄性筋萎縮症治療市場：セグメント分析、タイプ別、2018年-2030年

• 世界の脊髄性筋萎縮症治療市場：タイプ変動分析
• タイプ1
• タイプ2
• タイプ 3
• タイプ 4

第5章 世界の脊髄性筋萎縮症治療市場：セグメント分析、治療別、2018年-2030年

• 世界の脊髄性筋萎縮症治療市場：治療変動分析
• 遺伝子治療
• 薬

第6章 世界の脊髄性筋萎縮症治療市場：セグメント分析、薬剤別、2018年-2030年

• 世界の脊髄性筋萎縮症治療市場：薬物変動分析
• スピンラザ
• ゾルゲンスマ（AVXS-101）
• エヴリスディ
• その他

第7章 世界の脊髄性筋萎縮症治療市場：セグメント分析、投与経路別、2018年-2030年

• 世界の脊髄性筋萎縮症治療市場：投与経路の変動分析
• オーラル
• 注入

第8章 脊髄性筋萎縮症治療市場：セグメント分析、地域別、2018年-2030年

• 脊髄性筋萎縮症治療市場：地域変動分析
  • 北米
    • 米国
    • カナダ
  • 欧州
    • 英国
    • ドイツ
    • フランス
    • スペイン
    • イタリア
  • アジア太平洋地域
    • 日本
    • 中国
    • インド
    • オーストラリア
    • 韓国
  • ラテンアメリカ
    • ブラジル
    • メキシコ
    • アルゼンチン
  • 中東とアフリカ
    • 南アフリカ
    • サウジアラビア
    • アラブ首長国連邦

第9章 世界の脊髄性筋萎縮症治療市場：競合分析

• 主要な市場参入企業別最近の動向と影響分析
  • 主要な取引と戦略的提携の分析
    • 新製品の発売
    • 合併と買収
    • パートナーシップと契約
• 会社の分類
  • イノベーター
  • 市場のリーダー
  • 新興プレーヤー
  • ヒートマップ分析
• 会社の市況分析
• ベンダー情勢
  • 主要なディストリビューターとチャネルパートナーのリスト
  • 潜在的なエンドユーザーのリスト
  • 主要企業の市場シェア分析、2021年
• Company Market Share, 2021
  • 競合ダッシュボード分析
• 非公開会社
  • 主要新興企業一覧
  • 地域ネットワークマップ
• 企業プロファイル
  • Biogen
    • 会社概要
    • 財務実績
    • 製品のベンチマーク
    • 戦略的イニシアチブ
  • Novartis AG
    • 会社概要
    • 財務実績
    • 製品のベンチマーク
    • 戦略的イニシアチブ
  • Ionis Pharmaceuticals Inc.
    • 会社概要
    • 財務実績
    • 製品のベンチマーク
    • 戦略的イニシアチブ
  • Biohaven Pharmaceuticals
    • 会社概要
    • 財務実績
    • 製品のベンチマーク
    • 戦略的イニシアチブ
  • F. Hoffmann-La Roche Ltd
    • 会社概要
    • 財務実績
    • 製品のベンチマーク
    • 戦略的イニシアチブ
  • Cytokinetics
    • 会社概要
    • 製品のベンチマーク
    • 戦略的イニシアチブ
  • Scholar Rock, Inc.
    • 会社概要
    • 財務実績
    • 製品のベンチマーク
  • PTC Therapeutics
    • 会社概要
    • 財務実績
    • 製品のベンチマーク
    • 戦略的イニシアチブ
  • NMD Pharma A/S
    • 会社概要
    • 財務実績
    • 製品のベンチマーク
    • 戦略的イニシアチブ

List of Tables

• Table 1 List of Secondary Sources
• Table 2 List of Abbreviation
• Table 3 Global Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 4 Global Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 5 Global Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 6 Global Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 7 North America Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 8 North America Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 9 North America Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 10 North America Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 11 U.S. Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 12 U.S. Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 13 U.S. Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 14 U.S. Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 15 Canada Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 16 Canada Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 17 Canada Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 18 Canada Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 19 Europe Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 20 Europe Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 21 Europe Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 22 Europe Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 23 Germany Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 24 Germany Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 25 Germany Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 26 Germany Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 27 U.K. Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 28 U.K. Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 29 U.K. Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 30 U.K. Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 31 France Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 32 France Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 33 France Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 34 France Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 35 Italy Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 36 Italy Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 37 Italy Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 38 Italy Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 39 Spain Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 40 Spain Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 41 Spain Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 42 Spain Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 43 Asia Pacific Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 44 Asia Pacific Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 45 Asia Pacific Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 46 Asia Pacific Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 47 Japan Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 48 Japan Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 49 Japan Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 50 Japan Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 51 China Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 52 China Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 53 China Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 54 China Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 55 India Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 56 India Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 57 India Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 58 India Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 59 South Korea Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 60 South Korea Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 61 South Korea Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 62 South Korea Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 63 Australia Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 64 Australia Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 65 Australia Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 66 Australia Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 67 Latin America Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 68 Latin America Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 69 Latin America Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 70 Latin America Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 71 Brazil Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 72 Brazil Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 73 Brazil Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 74 Brazil Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 75 Mexico Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 76 Mexico Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 77 Mexico Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 78 Mexico Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 79 Argentina Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 80 Argentina Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 81 Argentina Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 82 Argentina Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 83 Middle East & Africa Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 84 Middle East & Africa Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 85 Middle East & Africa Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 86 Middle East & Africa Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 87 Saudi Arabia Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 88 Saudi Arabia Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 89 Saudi Arabia Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 90 Saudi Arabia Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 91 South Africa Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 92 South Africa Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 93 South Africa Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 94 South Africa Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)
• Table 95 UAE Spinal Muscular Atrophy Treatment Market, By Type, 2018 - 2030 (USD Million)
• Table 96 UAE Spinal Muscular Atrophy Treatment Market, By Treatment, 2018 - 2030 (USD Million)
• Table 97 UAE Spinal Muscular Atrophy Treatment Market, By Route of Administration, 2018 - 2030 (USD Million)
• Table 98 UAE Spinal Muscular Atrophy Treatment Market, By Drug, 2018 - 2030 (USD Million)

List of Figures

• Fig. 1 Spinal Muscular Atrophy Treatment market segmentation
• Fig. 2 Market research process
• Fig. 3 Data triangulation techniques
• Fig. 4 Primary research pattern
• Fig. 5 Market research approaches
• Fig. 6 Value-chain-based sizing & forecasting
• Fig. 7 QFD modeling for market share assessment
• Fig. 8 Market formulation & validation
• Fig. 9 Spinal Muscular Atrophy Treatment market snapshot
• Fig. 10 Parent market outlook
• Fig. 11 Related/ancillary market outlook, revenue, 2021, (USD Million)
• Fig. 12 Penetration and growth prospect mapping
• Fig. 13 Spinal muscular atrophy treatment market driver impact
• Fig. 14 Spinal muscular atrophy treatment market restraint impact
• Fig. 15 SWOT analysis, by factor (political & legal, economic, and technological)
• Fig. 16 Industry Analysis - Porter's
• Fig. 17 Strategy mapping
• Fig. 18 Spinal muscular atrophy treatment market: Type outlook and key takeaways
• Fig. 19 Spinal muscular atrophy treatment market: Type movement analysis & market share 2021 & 2030
• Fig. 20 Type 1 market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 21 Type 2 market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 22 Type 3 market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 23 Type 4 market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 24 Spinal muscular atrophy treatment market: Treatment outlook and key takeaways
• Fig. 25 Spinal muscular atrophy treatment market: Treatment movement analysis & market share 2021 & 2030
• Fig. 26 Gene therapy market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 27 Drug market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 28 Spinal muscular atrophy treatment market: Drug outlook and key takeaways
• Fig. 29 Spinal muscular atrophy treatment market: Drug movement analysis & market share 2021 & 2030
• Fig. 30 Spinraza market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 31 Zolgensama market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 32 Evrysdi market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 33 Other market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 34 Spinal muscular atrophy treatment market : Regional outlook and key takeaways
• Fig. 35 Regional outlook, 2021 & 2030
• Fig. 36 North America
• Fig. 37 North America market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 38 U.S.
• Fig. 39 U.S. market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 40 Canada
• Fig. 41 Canada market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 42 Europe
• Fig. 43 Europe market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 44 U.K.
• Fig. 45 U.K. market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 46 Germany
• Fig. 47 Germany market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 48 France
• Fig. 49 France market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 50 Italy
• Fig. 51 Italy market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 52 Spain
• Fig. 53 Spain market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 54 Asia Pacific
• Fig. 55 Asia Pacific market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 56 Japan
• Fig. 57 Japan market estimates and forecast, 2018 - 2030(USD Million)
• Fig. 58 China
• Fig. 59 China market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 60 India
• Fig. 61 India market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 62 South Korea
• Fig. 63 South Korea market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 64 Australia
• Fig. 65 Australia market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 66 Latin America
• Fig. 67 Latin America market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 68 Brazil
• Fig. 69 Brazil market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 70 Mexico
• Fig. 71 Mexico market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 72 Argentina
• Fig. 73 Argentina market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 74 Middle East & Africa
• Fig. 75 MEA market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 76 South Africa
• Fig. 77 South Africa market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 78 Saudi Arabia
• Fig. 79 Saudi Arabia market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 80 UAE
• Fig. 81 UAE market estimates and forecast, 2018 - 2030 (USD Million)
• Fig. 82 Market differentiators
• Fig. 83 Key company market share analysis, 2021
• Fig. 84 Company market position analysis
• Fig. 85 Competitive dashboard analysis
• Fig. 86 Regional network map

Spinal Muscular Atrophy Treatment Market Growth & Trends:

The global spinal muscular atrophy treatment market size is expected to reach USD 18.0 billion by 2030, according to a new study by Grand View Research, Inc. This is expected to expand at a CAGR of 18.6% from 2022 to 2030. This growth can be attributed to the increasing product approval and launches. For instance, in August 2020, the Food and Drug Administration approved Evrysdi (risdiplam) developed by Genentech, Inc., for the treatment of pediatric patients aged two months and older with spinal muscular atrophy (SMA).

In addition, in May 2020, Novartis AG received conditional approval to its SMA candidate Zolgensma (onasemnogene abeparvovec) from the European Commission (EC) to treat patients with SMA. It is only approved gene therapy in Europe. In March 2021, PTC Therapeutics, Inc., received marketing approval for Evrysdi (risdiplam) from the EMA for the treatment of patient with type1, 2, and 3 spinal muscular atrophy. Thus, increasing number of product approvals is expected to drive the spinal muscular atrophy treatment market.

Novartis AG is supporting SMA patients by offering medicine at free of cost through global Managed Access Program (MAP). This program provides 100 doses of Zolgensma at free of charge to eligible SMA patients across continents including North America, South America, Asia, Australia, Europe, and Africa. Thus, presence of such supportive programs for Zolgensma strengthens promotional activities and also aid in generating revenue.

Global SMA newborn screening and easy access to diagnostic tools should be included in routine approach. This would ensure early diagnosis and timely access to treatment. For instance, in July 2021, a charlotte baby received a breakthrough gene therapy treatment for Spinal Muscular Atrophy (SMA) at UNC Medical Center in Chapel Hill, N.C., after diagnosis with spinal muscular atrophy through Early Check newborn screening program. Such screening programs are expected to boost early diagnosis and treatment of disease.

Moreover, there are several extensive research collaborations undertaken by pharmaceutical companies with research institutes for developing novel therapies for the treatment of spinal muscular atrophy patients. For instance, in March 2021, PTC Therapeutics, Inc., and The Spinal Muscular Atrophy Foundation entered into research collaboration to advance scientific research and developing new treatment to treat patients with SMA.

However, high costs associated with the treatment and low availability of participant for clinical trial purposes may restrain market growth over the forecast period. The high cost related to research and development of an orphan drug is a major factor of rising prices of product. For instance, the most commonly used drug Spinraza costs around USD 125,000 per injection and USD 750,000 annually. Furthermore, Zolgensma (AVXS-101) costs USD 2.1 million for one time treatment.

Spinal Muscular Atrophy Treatment Market Report Highlights:

• By type, the type-1 segment held the largest market share in 2021 due to higher prevalence and wide availability of products for the treatment of patients with type-1 spinal muscular atrophy

• By treatment, the gene therapy segment is expected to be the fastest growing segment over the forecast period due to increase market penetration. Zolgensma is the only approved gene therapy in 37 countries to treat patient with SMA

• By drug, the Spinraza segment dominated the market in 2021 due to presence of supportive reimbursement policies and increasing awareness about products. Currently, Spinraza is applicable for full reimbursement in three European countries Italy, Norway, Netherlands

• By route of administration, the injection segment dominated the market in 2021 due to high safety, efficacy, and tolerability associated with infusion drugs to SMA patients through this route of administration

• Asia Pacific is expected to be the fastest growing region during the forecast period. The growth of region is attributable to the entry of new products into the region. For instance, in July 2021, F. Hoffmann-La Roche Ltd launched Evrysdi (risdiplam) for the treatment of patient with spinal muscular atrophy in India

Table of Contents

Chapter 1 Methodology and Scope

• 1.1 Market segmentation
  • 1.1.1 Estimates and forecast timeline
• 1.2 Research Methodology
• 1.3 Information Procurement
  • 1.3.1 Purchased database
  • 1.3.2 GVR'S internal database
  • 1.3.3 Secondary sources
  • 1.3.4 Primary research
  • 1.3.5 Details of primary research
  • 1.3.6 List of primary sources
• 1.4 Information or data analysis
  • 1.4.1 Data analysis models
• 1.5 Market Formulation & Validation
• 1.6 Model Details
  • 1.6.1 Commodity flow analysis
    • 1.6.1.1 Approach 1: Commodity Flow Approach
    • 1.6.1.2 Approach 2: Country-wise market estimation using bottom-up approach
• 1.7 Global Market: CAGR Calculation
• 1.8 Research Assumptions
• 1.9 List of Secondary Sources
• 1.10 List of Abbreviations
• 1.11 Objectives
  • 1.11.1 Objective 1
  • 1.11.2 Objective 2
  • 1.11.3 Objective 3
  • 1.11.4 Objective 4

Chapter 2 Executive Summary

• 2.1 Market Summary

Chapter 3 Market Variables, Trends & Scope

• 3.1 Market Lineage Outlook
  • 3.1.1 Parent market outlook
• 3.2 Regulatory framework
• 3.3 Penetration and Growth Prospect Mapping
• 3.4 Pipeline Analysis
• 3.5 Market Dynamics
  • 3.5.1 Market driver analysis
  • 3.5.2 Market restraint analysis
• 3.6 PESTEL Analysis

Chapter 4 Global Spinal Muscular Atrophy Treatment Market - Segment Analysis, By Type, 2018 - 2030 (USD Million)

• 4.1 Global Spinal Muscular Atrophy Treatment Market: Type Movement Analysis
• 4.2 Type 1
  • 4.2.1 Type 1 market estimates and forecast, 2018 - 2030 (USD Million)
• 4.3 Type 2
  • 4.3.1 Type 2 market estimates and forecast, 2018 - 2030 (USD Million)
• 4.4 Type 3
  • 4.4.1 Type 3 market estimates and forecast, 2018 - 2030 (USD Million)
• 4.5 Type 4
  • 4.5.1 Type 4 market estimates and forecast, 2018 - 2030 (USD Million)

Chapter 5 Global Spinal Muscular Atrophy Treatment Market - Segment Analysis, by Treatment, 2018 - 2030 (USD Million)

• 5.1 Global Spinal Muscular Atrophy Treatment Market: Treatment Movement Analysis
• 5.2 Gene Therapy
  • 5.2.1 Gene therapy market estimates and forecast, 2018 - 2030 (USD Million)
• 5.3 Drug
  • 5.3.1 Drug market estimates and forecast, 2018 - 2030 (USD Million)

Chapter 6 Global Spinal Muscular Atrophy Treatment Market - Segment Analysis, By Drug, 2018 - 2030 (USD Million)

• 6.1 Global Spinal Muscular Atrophy Treatment Market: Drug Movement Analysis
• 6.2 Spinraza
  • 6.2.1 Spinraza market estimates and forecast, 2018 - 2030 (USD Million)
• 6.3 Zolgensma (AVXS-101)
  • 6.3.1 Zolgensma (AVXS-101) market estimates and forecast, 2018 - 2030 (USD Million)
• 6.4 Evrysdi
  • 6.4.1 Evrysdi market estimates and forecast, 2018 - 2030 (USD Million)
• 6.5 Others
  • 6.5.1 Others market estimates and forecast, 2018 - 2030 (USD Million)

Chapter 7 Global Spinal Muscular Atrophy Treatment Market - Segment Analysis, by Route of Administration, 2018 - 2030 (USD Million)

• 7.1 Global Spinal Muscular Atrophy Treatment Market: Route of Administration Movement Analysis
• 7.2 Oral
  • 7.2.1 Oral market estimates and forecast, 2018 - 2030 (USD Million)
• 7.3 Injection
  • 7.3.1 Injection market estimates and forecast, 2018 - 2030 (USD Million)

Chapter 8 Spinal Muscular Atrophy Treatment Market: Segment Analysis, by Region, 2018 - 2030 (USD Million)

• 8.1 Spinal Muscular Atrophy Treatment Market: Regional Movement Analysis
  • 8.1.1 North America
    • 8.1.1.1 North America market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.1.2 U.S.
    • 8.1.1.2.1 U.S. market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.1.3 Canada
    • 8.1.1.3.1 Canada market estimates and forecast, 2018 - 2030 (USD Million)
  • 8.1.2 Europe
    • 8.1.2.1 Europe market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.2.2 U.K.
    • 8.1.2.2.1 U.K. market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.2.3 Germany
    • 8.1.2.3.1 Germany market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.2.4 France
    • 8.1.2.4.1 France market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.2.5 Spain
    • 8.1.2.5.1 Spain market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.2.6 Italy
    • 8.1.2.6.1 Italy market estimates and forecast, 2018 - 2030 (USD Million)
  • 8.1.3 Asia Pacific
    • 8.1.3.1 Asia Pacific market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.3.2 Japan
    • 8.1.3.2.1 Japan market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.3.3 China
    • 8.1.3.3.1 China market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.3.4 India
    • 8.1.3.4.1 India market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.3.5 Australia
    • 8.1.3.5.1 Australia market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.3.5 South Korea
    • 8.1.3.5.1 South Korea market estimates and forecast, 2018 - 2030 (USD Million)
  • 8.1.4 Latin America
    • 8.1.4.1 Latin America market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.4.2 Brazil
    • 8.1.4.2.1 Brazil market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.4.3 Mexico
    • 8.1.4.3.1 Mexico market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.4.4 Argentina
    • 8.1.4.4.1 Argentina market estimates and forecast, 2018 - 2030 (USD Million)
  • 8.1.5 Middle East & Africa
    • 8.1.5.1 Middle East & Africa market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.5.2 South Africa
    • 8.1.5.2.1 South Africa market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.5.3 Saudi Arabia
    • 8.1.5.3.1 Saudi Arabia market estimates and forecast, 2018 - 2030 (USD Million)
    • 8.1.5.5 United Arab Emirates
    • 8.1.5.5.1 United Arab Emirates market estimates and forecast, 2018 - 2030 (USD Million)

Chapter 9 Global Spinal Muscular Atrophy Treatment Market: Competitive Analysis

• 9.1 Recent Developments and Impact Analysis, by Key Market Participants
  • 9.1.1 Major deals & strategic alliances analysis
    • 9.1.1.1 New Product Launches
    • 9.1.1.2 Mergers and Acquisitions
    • 9.1.1.3 Partnerships & Agreements
• 9.2 Company Categorization
  • 9.2.1 Innovators
  • 9.2.2 Market Leaders
  • 9.2.3 Emerging Players
  • 9.2.4 Heat Map Analysis
• 9.3 Company Market Position Analysis
• 9.4 Vendor Landscape
  • 9.4.1 List of Key Distributors and Channel Partners
  • 9.4.2 List of Potential End Users
  • 9.4.3 Key Company Market Share Analysis, 2021
• 9.5 Company Market Share, 2021
  • 9.5.1 Competitive Dashboard Analysis
• 9.6 Private Companies
  • 9.6.1 List of Key Emerging Companies
  • 9.6.2 Regional Network Map
• 9.7 Company Profiles
  • 9.7.1 Biogen
    • 9.7.1.1 Company overview
    • 9.7.1.2 Financial performance
    • 9.7.1.3 Product benchmarking
    • 9.7.1.4 Strategic initiatives
  • 9.7.2 Novartis AG
    • 9.7.2.1 Company overview
    • 9.7.2.2 Financial performance
    • 9.7.2.3 Product benchmarking
    • 9.7.2.4 Strategic initiatives
  • 9.7.3 Ionis Pharmaceuticals Inc.
    • 9.7.3.1 Company overview
    • 9.7.3.2 Financial performance
    • 9.7.3.3 Product benchmarking
    • 9.7.3.4 Strategic initiatives
  • 9.7.4 Biohaven Pharmaceuticals
    • 9.7.4.1 Company overview
    • 9.7.4.2 Financial performance
    • 9.7.4.3 Product benchmarking
    • 9.7.4.4 Strategic initiatives
  • 9.7.5 F. Hoffmann-La Roche Ltd
    • 9.7.5.1 Company overview
    • 9.7.5.2 Financial performance
    • 9.7.5.3 Product benchmarking
    • 9.7.5.4 Strategic initiatives
  • 9.7.6 Cytokinetics
    • 9.7.6.1 Company overview
    • 9.7.6.2 Product benchmarking
    • 9.7.6.3 Strategic initiatives
  • 9.7.7 Scholar Rock, Inc.
    • 9.7.7.1 Company overview
    • 9.7.7.2 Financial performance
    • 9.7.7.3 Product benchmarking
  • 9.7.8 PTC Therapeutics
    • 9.7.8.1 Company overview
    • 9.7.8.2 Financial performance
    • 9.7.8.3 Product benchmarking
    • 9.7.8.4 Strategic initiatives
  • 9.7.9 NMD Pharma A/S
    • 9.7.9.1 Company overview
    • 9.7.9.2 Financial performance
    • 9.7.9.3 Product benchmarking
    • 9.7.9.4 Strategic initiatives