脊髄性筋萎縮症治療の世界市場：市場規模・シェア・動向分析 (種類別・治療法別・投与経路別・地域別・セグメント別、2025年～2030年)

脊髄性筋萎縮症治療市場の成長と動向：

Grand View Research Inc.の最新調査によると、世界の脊髄性筋萎縮症治療の市場規模は2025年から2030年にかけてCAGR 18.0%を記録し、2030年までに130億9,000万米ドルに達すると予測されています。

この成長は、製品の承認や発売が増加していることに起因しています。例えば、2020年8月、ジェネンテック社が開発したEvrysdi (risdiplam) が、脊髄性筋萎縮症 (SMA) の生後2カ月以上の小児患者の治療薬として食品医薬品局から承認されました。

さらに2020年5月、ノバルティスAGは欧州委員会 (EC) より、SMA患者を対象としたSMA候補薬ゾルゲンスマ (onasemnogene abeparvovec) の条件付き承認を取得しました。欧州で承認された唯一の遺伝子治療薬です。2021年3月、PTCセラピューティクス社は、1型、2型、3型の脊髄性筋萎縮症患者の治療薬として、Evrysdi (risdiplam) の販売承認をEMAから取得しました。このように、製品承認数の増加が脊髄性筋萎縮症治療市場を牽引すると予想されます。

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) を通じて薬を無料で提供し、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、アフリカを含む世界中の対象となるSMA患者にゾルゲンマ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、販促活動を強化し、収益を生み出す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べきです。そうすれば、早期診断と治療へのタイムリーなアクセスが保証されます。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た後、脊髄性筋萎縮症 (SMA) の画期的な遺伝子治療を受けています。このようなスクリーニングプログラムは、疾患の早期診断と治療を促進すると期待されています。

さらに、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、製薬会社が研究機関と行っている大規模な共同研究がいくつかあります。例えば、2021年3月、PTC Therapeutics, Inc.とThe Spinal Muscular Atrophy Foundationは、脊髄性筋萎縮症患者を治療するための科学的研究と新規治療開発を進めるための研究提携を締結しました。

しかし、治療に関連する費用が高く、臨床試験目的の市場参入企業が少ないことが、予測期間中の市場成長を抑制する可能性があります。希少疾病用医薬品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です。例えば、最も一般的に使用されているSpinrazaは、注射1本あたり約12万5,000米ドル、年間75万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市場：分析概要

• 種類別では、1型脊髄性筋萎縮症患者の有病率が高く、治療用製品が広く入手可能であることから、1型セグメントが2024年に最大の市場シェアを占めました。
• 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急成長すると予想されます。Zolgensmaは、SMA患者を治療するために37カ国で承認された唯一の遺伝子治療薬です。
• 薬剤別では、保険償還政策が充実していることと、製品に対する認知度が高まっていることから、2024年にはスピンラザのセグメンテーションが市場を独占します。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となっています。
• 投与経路別では、注入剤が2024年に市場を席巻しましたが、これはこの投与経路によるSMA患者への輸注薬に関連した高い安全性、有効性、忍容性によるものです。
• 予測期間中、アジア太平洋が最も急成長する見込みです。同地域の成長は、同地域への新製品の参入に起因します。例えば、2021年7月、F.ホフマン・ラ・ロシュ社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 分析方法・範囲

第2章 エグゼクティブサマリー

第3章 脊髄性筋萎縮症治療市場：変動要因・傾向・範囲

• 市場連関の見通し
• 市場力学
  • 市場促進要因の分析
  • 市場抑制要因の分析
• ビジネス環境分析
  • 業界分析：ポーターのファイブフォース分析
  • PESTEL分析

第4章 脊髄性筋萎縮症治療市場：種類別のビジネス分析

• 市場シェア：種類別 (2024年・2030年)
• 種類別ダッシュボード
• 市場規模の予測と動向分析：種類別 (2018～2030年)
• 1型
• 2型
• 3型
• 4型

第5章 脊髄性筋萎縮症治療市場：治療法別のビジネス分析

• 市場シェア：治療法別 (2024年・2030年)
• 治療法別ダッシュボード
• 市場規模の予測と動向分析：治療法別 (2018～2030年)
• 遺伝子治療
• 投薬療法

第6章 脊髄性筋萎縮症治療市場：投与経路別のビジネス分析

• 市場シェア：投与経路別 (2024年・2030年)
• 投与経路別ダッシュボード
• 市場規模の予測と動向分析：投与経路別 (2018～2030年)
• 経口
• 注入

第7章 脊髄性筋萎縮症治療市場：地域別のビジネス分析

• 市場シェア分析：地域別 (2024年・2030年)
• 市場ダッシュボード：地域別
• 市場規模の予測と動向分析 (2018～2030年)
• 北米
  • 国別、2018年-2030年
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • 国別、2018年-2030年
  • 英国
  • ドイツ
  • フランス
  • イタリア
  • スペイン
  • ノルウェー
  • デンマーク
  • スウェーデン
• アジア太平洋
  • 日本
  • 中国
  • インド
  • 韓国
  • オーストラリア
  • タイ
• ラテンアメリカ
  • 国別、2018年-2030年
  • ブラジル
  • アルゼンチン
• 中東・アフリカ
  • 国別、2018年-2030年
  • 南アフリカ
  • サウジアラビア
  • アラブ首長国連邦
  • クウェート

第8章 競合情勢

• 市場参入企業の概要
• 企業の市場ポジション分析
• 企業分類
• 戦略マッピング
• 企業プロファイル/上場企業
  • Biogen
  • Novartis AG
  • Pfizer Inc.
  • Ionis Pharmaceuticals
  • Biohaven, Ltd.
  • F. Hoffmann-La Roche Ltd
  • Cytokinetics
  • Scholar Rock, Inc.
  • PTC Therapeutics
  • NMD PHARMA A/S

List of Tables

• Table 1 List of secondary sources
• Table 2 List of abbreviations
• Table 3 Global spinal muscular atrophy treatment market, by region, 2018 - 2030 (USD Billion)
• Table 4 Global spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 5 Global spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 6 Global spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 7 North America spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
• Table 8 North America spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 9 North America spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 10 North America spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 11 U.S. spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 12 U.S. spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 13 U.S. spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 14 Canada spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 15 Canada spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 16 Canada spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 17 Mexico spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 18 Mexico spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 19 Mexico spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 20 Europe spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
• Table 21 Europe spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 22 Europe spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 23 Europe spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 24 UK spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 25 UK spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 26 UK spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 27 Germany spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 28 Germany spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 29 Germany spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 30 France spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 31 France spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 32 France spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 33 Italy spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 34 Italy spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 35 Italy spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 36 Spain spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 37 Spain spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 38 Spain spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 39 Norway spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 40 Norway spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 41 Norway spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 42 Denmark spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 43 Denmark spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 44 Denmark spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 45 Sweden spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 46 Sweden spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 47 Sweden spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 48 Asia Pacific spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
• Table 49 Asia Pacific spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 50 Asia Pacific spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 51 Asia Pacific spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 52 Japan spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 53 Japan spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 54 Japan spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 55 China spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 56 China spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 57 China spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 58 India spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 59 India spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 60 India spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 61 Australia spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 62 Australia spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 63 Australia spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 64 South Korea spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 65 South Korea spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 66 South Korea spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 67 Thailand spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 68 Thailand spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 69 Thailand spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 70 Latin America spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
• Table 71 Latin America spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 72 Latin America spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion
• Table 73 Latin America spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion
• Table 74 Brazil spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 75 Brazil spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 76 Brazil spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 77 Argentina spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 78 Argentina spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 79 Argentina spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 80 Middle East & Africa spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
• Table 81 Middle East & Africa spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 82 Middle East & Africa spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 83 Middle East & Africa spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 84 South Africa spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 85 South Africa spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 86 South Africa spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 87 Saudi Arabia spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 88 Saudi Arabia spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 89 Saudi Arabia spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 90 UAE spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 91 UAE spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 92 UAE spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
• Table 93 Kuwait spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
• Table 94 Kuwait spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
• Table 95 Kuwait spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)

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 Market formulation & validation
• Fig. 8 Market snapshot
• Fig. 9 Type outlook (USD Billion)
• Fig. 10 Treatment outlook (USD Billion)
• Fig. 11 Route of administration outlook (USD Billion)
• Fig. 12 Competitive landscape
• Fig. 13 Spinal muscular atrophy treatment market dynamics
• Fig. 14 Spinal muscular atrophy treatment market: Porter's five forces analysis
• Fig. 15 Spinal muscular atrophy treatment market: PESTLE analysis
• Fig. 16 Spinal muscular atrophy treatment market: Type segment dashboard
• Fig. 17 Spinal muscular atrophy treatment market: Type market share analysis, 2024 & 2030
• Fig. 18 Type 1 market, 2018 - 2030 (USD Billion)
• Fig. 19 Type 2 market, 2018 - 2030 (USD Billion)
• Fig. 20 Type 3 market, 2018 - 2030 (USD Billion)
• Fig. 21 Type 4 market, 2018 - 2030 (USD Billion)
• Fig. 22 Spinal muscular atrophy treatment market: Treatment segment dashboard
• Fig. 23 Spinal muscular atrophy treatment market: Treatment market share analysis, 2024 & 2030
• Fig. 24 Gene therapy market, 2018 - 2030 (USD Billion)
• Fig. 25 Drug market, 2018 - 2030 (USD Billion)
• Fig. 26 Spinraza market, 2018 - 2030 (USD Billion)
• Fig. 27 Zolgensma (AVXS-101) market, 2018 - 2030 (USD Billion)
• Fig. 28 Evrysdi market, 2018 - 2030 (USD Billion)
• Fig. 29 Others market, 2018 - 2030 (USD Billion)
• Fig. 30 Spinal muscular atrophy treatment market: Route of administration segment dashboard
• Fig. 31 Spinal muscular atrophy treatment market: Route of administration market share analysis, 2024 & 2030
• Fig. 32 Oral market, 2018 - 2030 (USD Billion)
• Fig. 33 Injection market, 2018 - 2030 (USD Billion)
• Fig. 34 Spinal muscular atrophy treatment market revenue, by region
• Fig. 35 Regional marketplace: Key takeaways
• Fig. 36 North America spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 37 U.S. country dynamics
• Fig. 38 U.S. spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 39 Canada country dynamics
• Fig. 40 Canada spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 41 Mexico country dynamics
• Fig. 42 Mexico spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 43 Europe spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 44 UK country dynamics
• Fig. 45 UK spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 46 Germany country dynamics
• Fig. 47 Germany spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 48 France country dynamics
• Fig. 49 France spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 50 Italy country dynamics
• Fig. 51 Italy spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 52 Spain country dynamics
• Fig. 53 Spain spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 54 Norway country dynamics
• Fig. 55 Norway spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 56 Denmark country dynamics
• Fig. 57 Denmark spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion
• Fig. 58 Sweden country dynamics
• Fig. 59 Sweden spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 60 Asia Pacific spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 61 Japan country dynamics
• Fig. 62 Japan spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 63 China country dynamics
• Fig. 64 China spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 65 India country dynamics
• Fig. 66 India spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 67 South Korea country dynamics
• Fig. 68 South Korea spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 69 Australia country dynamics
• Fig. 70 Australia spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 71 Thailand country dynamics
• Fig. 72 Thailand spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 73 Latin America spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 74 Brazil country dynamics
• Fig. 75 Brazil spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 76 Argentina country dynamics
• Fig. 77 Argentina spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 78 MEA spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 79 South Africa country dynamics
• Fig. 80 South Africa spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 81 Saudi Arabia country dynamics
• Fig. 82 Saudi Arabia spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 83 UAE country dynamics
• Fig. 84 UAE spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 85 Kuwait country dynamics
• Fig. 86 Kuwait spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
• Fig. 87 Company categorization
• Fig. 88 Company market position analysis
• Fig. 89 Strategic framework

Spinal Muscular Atrophy Treatment Market Growth & Trends:

The global spinal muscular atrophy treatment market size is expected to reach USD 13.09 billion by 2030, registering a CAGR of 18.0% from 2025 to 2030, according to a new study by Grand View Research Inc. 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 2024 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 2024 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 2024 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 & Scope
• 1.2. Segment Definitions
  • 1.2.1. Type
  • 1.2.2. Treatment
  • 1.2.3. Route of Administration
• 1.3. Estimates and Forecast Timeline
• 1.4. Research Methodology
• 1.5. Information Procurement
  • 1.5.1. Purchased Database
  • 1.5.2. GVR's Internal Database
  • 1.5.3. Secondary Sources
  • 1.5.4. Primary Research
• 1.6. Information Analysis
  • 1.6.1. Data Analysis Models
• 1.7. Market Formulation & Data Visualization
• 1.8. Model Details
  • 1.8.1. Commodity Flow Analysis
• 1.9. List of Secondary Sources
• 1.10. Objectives

Chapter 2. Executive Summary

• 2.1. Market Snapshot
• 2.2. Segment Snapshot
• 2.3. Competitive Landscape Snapshot

Chapter 3. Spinal Muscular Atrophy Treatment Market Variables, Trends, & Scope

• 3.1. Market Lineage Outlook
• 3.2. Market Dynamics
  • 3.2.1. Market Driver Analysis
  • 3.2.2. Market Restraint Analysis
• 3.3. Business Environment Analysis
  • 3.3.1. Industry Analysis - Porter's Five Forces Analysis
    • 3.3.1.1. Supplier Power
    • 3.3.1.2. Buyer Power
    • 3.3.1.3. Substitution Threat
    • 3.3.1.4. Threat of New Entrants
    • 3.3.1.5. Competitive Rivalry
  • 3.3.2. PESTLE Analysis

Chapter 4. Spinal Muscular Atrophy Treatment Market: Type Business Analysis

• 4.1. Type Market Share, 2024 & 2030
• 4.2. Type Segment Dashboard
• 4.3. Market Size & Forecasts and Trend Analysis, by Type, 2018 to 2030 (USD Billion)
• 4.4. Type 1
  • 4.4.1. Type 1 Market, 2018 - 2030 (USD Billion)
• 4.5. Type 2
  • 4.5.1. Type 2 Market, 2018 - 2030 (USD Billion)
• 4.6. Type 3
  • 4.6.1. Type 3 Market, 2018 - 2030 (USD Billion)
• 4.7. Type 4
  • 4.7.1. Type 4 Market, 2018 - 2030 (USD Billion)

Chapter 5. Spinal Muscular Atrophy Treatment Market: Treatment Business Analysis

• 5.1. Treatment Market Share, 2024 & 2030
• 5.2. Treatment Segment Dashboard
• 5.3. Market Size & Forecasts and Trend Analysis, by Treatment, 2018 to 2030 (USD Billion)
• 5.4. Gene Therapy
  • 5.4.1. Gene Therapy Market, 2018 - 2030 (USD Billion)
• 5.5. Drug
  • 5.5.1. Drug Market, 2018 - 2030 (USD Billion)
  • 5.5.2. Spinraza
    • 5.5.2.1. Spinraza Market, 2018 - 2030 (USD Billion)
  • 5.5.3. Zolgensma (AVXS-101)
    • 5.5.3.1. Zolgensma (AVXS-101) Market, 2018 - 2030 (USD Billion)
  • 5.5.4. Evrysdi
    • 5.5.4.1. Evrysdi Market, 2018 - 2030 (USD Billion)
  • 5.5.5. Others
    • 5.5.5.1. Others Market, 2018 - 2030 (USD Billion)

Chapter 6. Spinal Muscular Atrophy Treatment Market: Route of Administration Business Analysis

• 6.1. Route of Administration Market Share, 2024 & 2030
• 6.2. Route of Administration Segment Dashboard
• 6.3. Market Size & Forecasts and Trend Analysis, by Route of Administration, 2018 to 2030 (USD Billion)
• 6.4. Oral
  • 6.4.1. Oral Market, 2018 - 2030 (USD Billion)
• 6.5. Injection
  • 6.5.1. Injection Market, 2018 - 2030 (USD Billion)

Chapter 7. Spinal Muscular Atrophy Treatment Market: Regional Estimates & Trend Analysis

• 7.1. Regional Market Share Analysis, 2024 & 2030
• 7.2. Regional Market Dashboard
• 7.3. Market Size & Forecasts Trend Analysis, 2018 to 2030:
• 7.4. North America
  • 7.4.1. North America Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 7.4.2. U.S.
    • 7.4.2.1. Key Country Dynamics
    • 7.4.2.2. Regulatory Framework
    • 7.4.2.3. Competitive Insights
    • 7.4.2.4. U.S. Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.4.3. Canada
    • 7.4.3.1. Key Country Dynamics
    • 7.4.3.2. Regulatory Framework
    • 7.4.3.3. Competitive Insights
    • 7.4.3.4. Canada Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.4.4. Mexico
    • 7.4.4.1. Key Country Dynamics
    • 7.4.4.2. Regulatory Framework
    • 7.4.4.3. Competitive Insights
    • 7.4.4.4. Mexico Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 7.5. Europe
  • 7.5.1. Europe Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 7.5.2. UK
    • 7.5.2.1. Key Country Dynamics
    • 7.5.2.2. Regulatory Framework
    • 7.5.2.3. Competitive Insights
    • 7.5.2.4. UK Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.3. Germany
    • 7.5.3.1. Key Country Dynamics
    • 7.5.3.2. Regulatory Framework
    • 7.5.3.3. Competitive Insights
    • 7.5.3.4. Germany Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.4. France
    • 7.5.4.1. Key Country Dynamics
    • 7.5.4.2. Regulatory Framework
    • 7.5.4.3. Competitive Insights
    • 7.5.4.4. France Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.5. Italy
    • 7.5.5.1. Key Country Dynamics
    • 7.5.5.2. Regulatory Framework
    • 7.5.5.3. Competitive Insights
    • 7.5.5.4. Italy Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.6. Spain
    • 7.5.6.1. Key Country Dynamics
    • 7.5.6.2. Regulatory Framework
    • 7.5.6.3. Competitive Insights
    • 7.5.6.4. Spain Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.7. Norway
    • 7.5.7.1. Key Country Dynamics
    • 7.5.7.2. Regulatory Framework
    • 7.5.7.3. Competitive Insights
    • 7.5.7.4. Norway Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.8. Denmark
    • 7.5.8.1. Key Country Dynamics
    • 7.5.8.2. Regulatory Framework
    • 7.5.8.3. Competitive Insights
    • 7.5.8.4. Denmark Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.9. Sweden
    • 7.5.9.1. Key Country Dynamics
    • 7.5.9.2. Regulatory Framework
    • 7.5.9.3. Competitive Insights
    • 7.5.9.4. Sweden Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
• 7.6. Asia Pacific
  • 7.6.1. Asia Pacific Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.2. Japan
    • 7.6.2.1. Key Country Dynamics
    • 7.6.2.2. Regulatory Framework
    • 7.6.2.3. Competitive Insights
    • 7.6.2.4. Japan Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.3. China
    • 7.6.3.1. Key Country Dynamics
    • 7.6.3.2. Regulatory Framework
    • 7.6.3.3. Competitive Insights
    • 7.6.3.4. China Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.4. India
    • 7.6.4.1. Key Country Dynamics
    • 7.6.4.2. Regulatory Framework
    • 7.6.4.3. Competitive Insights
    • 7.6.4.4. India Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.5. South Korea
    • 7.6.5.1. Key Country Dynamics
    • 7.6.5.2. Regulatory Framework
    • 7.6.5.3. Competitive Insights
    • 7.6.5.4. South Korea Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.6. Australia
    • 7.6.6.1. Key Country Dynamics
    • 7.6.6.2. Regulatory Framework
    • 7.6.6.3. Competitive Insights
    • 7.6.6.4. Australia Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.7. Thailand
    • 7.6.7.1. Key Country Dynamics
    • 7.6.7.2. Regulatory Framework
    • 7.6.7.3. Competitive Insights
    • 7.6.7.4. Thailand Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 7.7. Latin America
  • 7.7.1. Latin America Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 7.7.2. Brazil
    • 7.7.2.1. Key Country Dynamics
    • 7.7.2.2. Regulatory Framework
    • 7.7.2.3. Competitive Insights
    • 7.7.2.4. Brazil Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.7.3. Argentina
    • 7.7.3.1. Key Country Dynamics
    • 7.7.3.2. Regulatory Framework
    • 7.7.3.3. Competitive Insights
    • 7.7.3.4. Argentina Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 7.8. MEA
  • 7.8.1. MEA Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 7.8.2. South Africa
    • 7.8.2.1. Key Country Dynamics
    • 7.8.2.2. Regulatory Framework
    • 7.8.2.3. Competitive Insights
    • 7.8.2.4. South Africa Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.8.3. Saudi Arabia
    • 7.8.3.1. Key Country Dynamics
    • 7.8.3.2. Regulatory Framework
    • 7.8.3.3. Competitive Insights
    • 7.8.3.4. Saudi Arabia Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.8.4. UAE
    • 7.8.4.1. Key Country Dynamics
    • 7.8.4.2. Regulatory Framework
    • 7.8.4.3. Competitive Insights
    • 7.8.4.4. UAE Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.8.5. Kuwait
    • 7.8.5.1. Key Country Dynamics
    • 7.8.5.2. Regulatory Framework
    • 7.8.5.3. Competitive Insights
    • 7.8.5.4. Kuwait Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)

Chapter 8. Competitive Landscape

• 8.1. Participant Overview
• 8.2. Company Market Position Analysis
• 8.3. Company Categorization
• 8.4. Strategy Mapping
• 8.5. Company Profiles/Listing
  • 8.5.1. Biogen
    • 8.5.1.1. Overview
    • 8.5.1.2. Financial Performance
    • 8.5.1.3. Product Benchmarking
    • 8.5.1.4. Strategic Initiatives
  • 8.5.2. Novartis AG
    • 8.5.2.1. Overview
    • 8.5.2.2. Financial Performance
    • 8.5.2.3. Product Benchmarking
    • 8.5.2.4. Strategic Initiatives
  • 8.5.3. Pfizer Inc.
    • 8.5.3.1. Overview
    • 8.5.3.2. Financial Performance
    • 8.5.3.3. Product Benchmarking
    • 8.5.3.4. Strategic Initiatives
  • 8.5.4. Ionis Pharmaceuticals
    • 8.5.4.1. Overview
    • 8.5.4.2. Financial Performance
    • 8.5.4.3. Product Benchmarking
    • 8.5.4.4. Strategic Initiatives
  • 8.5.5. Biohaven, Ltd.
    • 8.5.5.1. Overview
    • 8.5.5.2. Financial Performance
    • 8.5.5.3. Product Benchmarking
    • 8.5.5.4. Strategic Initiatives
  • 8.5.6. F. Hoffmann-La Roche Ltd
    • 8.5.6.1. Overview
    • 8.5.6.2. Financial Performance
    • 8.5.6.3. Product Benchmarking
    • 8.5.6.4. Strategic Initiatives
  • 8.5.7. Cytokinetics
    • 8.5.7.1. Overview
    • 8.5.7.2. Financial Performance
    • 8.5.7.3. Product Benchmarking
    • 8.5.7.4. Strategic Initiatives
  • 8.5.8. Scholar Rock, Inc.
    • 8.5.8.1. Overview
    • 8.5.8.2. Financial Performance
    • 8.5.8.3. Product Benchmarking
    • 8.5.8.4. Strategic Initiatives
  • 8.5.9. PTC Therapeutics
    • 8.5.9.1. Overview
    • 8.5.9.2. Financial Performance
    • 8.5.9.3. Product Benchmarking
    • 8.5.9.4. Strategic Initiatives
  • 8.5.10. NMD PHARMA A/S
    • 8.5.10.1. Overview
    • 8.5.10.2. Financial Performance
    • 8.5.10.3. Product Benchmarking
    • 8.5.10.4. Strategic Initiatives