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市场调查报告书
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1425124

脊髓性肌肉萎缩症治疗市场－2024年至2029年预测

Spinal Muscular Atrophy Treatment Market - Forecasts from 2024 to 2029
出版日期: | 出版商: Knowledge Sourcing Intelligence | 英文 146 Pages | 商品交期: 最快1-2个工作天内

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简介目录

预计脊髓性肌肉萎缩症治疗市场在预测期内将以 12.35% 的复合年增长率成长。

大约万分之一的人患有一种称为脊髓性肌肉萎缩症 (SMA) 的遗传性疾病。大多数 SMA 是由 SMN1 片段缺陷引起的，该缺陷阻止基因产生蛋白质。运动神经元存活 (SMN) 蛋白对于维持正常运动神经元活动至关重要，主要由 SMN1 产生。 SMA 的严重程度各不相同。患有 SMA 的婴儿会出现呼吸、吞嚥、坐立和行走困难。如果延误治疗，SMA 可能会导致过早死亡。

脊髓性肌肉萎缩症治疗市场的成长动力

由于基因发现、创造性治疗方法和公众意识的提高，脊髓性肌肉萎缩症治疗行业正在迅速改变。患者流动、新生儿筛检计画和监管支持都有助于加速 SMA 治疗的进展。随着更多研究的开展和更容易获得的治疗方法，SMA 患者获得更好结果和更高生活水准的希望越来越大。

治疗方法意识不断增强

许多方法用于治疗脊髓性肌肉萎缩症（SMA）。其中包括反义寡核苷酸治疗（如 Spinraza）和基因替代疗法（如 Zolgensma），前者旨在增加 SMN 蛋白的合成，后者使 SMN1 基因发挥功能。 Evrysdi 和其他小分子药物也针对 SMN 蛋白的发育。仅增加体内 SMN 蛋白的水平不足以治疗 SMA。其他系统、途径和过程也受到 SMN 蛋白流失的影响，使这些系统成为进一步治疗的重点。这些方法通常称为非 SMN 方法。许多非 SMN 方法都专注于神经和肌肉。

加大研发力度

在医疗领域，越来越多的旨在治疗脊髓性肌肉萎缩症（SMA）的研发计划是脊髓性肌肉萎缩症治疗市场成长的关键成长要素。这些计划的范围从基因治疗到小分子药物和基因编辑技术，是由对有效治疗的迫切需求和更好地了解脊髓性肌肉萎缩症的遗传原因所驱动的。例如，FDA分别于2019年和2020年核准了两种药物Zolgensma®（onasemnogene abeparvovec-xioi）和EvrysdiTM（risdiplam）。此外，多个开发平臺目前正处于临床阶段，为 SMA 的全面復健带来希望。此外，百健（Biogen）希望透过目前的研发计划（包括 DEVOTE、RESPOND 和 ASCEND）来评估替代治疗方法并为 SMA 群体的选择提供指南。

治疗脊髓性肌肉萎缩症的临床试验

临床试验数量的增加进一步推动了脊髓性肌肉萎缩症治疗市场的发展。为了鼓励快速创建和实施影响成人和儿童的神经系统疾病的临床试验，NINDS 组建了 NeuroNext（NINDS 神经科学临床试验卓越网路）网路。该网络的目标是进行早期研究以评估新治疗方法并发现生物标记。生物标记是物理特征或物质，通常存在于血液等生理液体中，用于评估疾病的存在和严重程度。寻找 SMA 生物标记并了解该疾病的潜在机制和原因就是这样的计划之一。这项研究的资讯用于确定是否核准nusinersen (SpinrazaTM)。从这项研究中获得的知识也改进了 SMA 下一个临床试验的设计。

提高对新生儿筛检计划的认识

对新生儿筛检计划的日益关注正在推动脊髓性肌肉萎缩症治疗市场的成长。在此过程中，州公共卫生实验室使用出生后 24 至 48 小时内从婴儿脚跟采集的干血点来识别遗传性疾病，包括 SMA，作为常规新生儿筛检的一部分。技术人员进行测试以确定导致 SMA 的基因缺失是否是导致疾病的原因。如果发现 SMN1 基因异常，公共卫生实验室或临床实验室将进行额外测试以确定 SMN2 基因的拷贝数。

亚太地区脊髓性肌肉萎缩症治疗市场预计将呈现稳定成长。

亚太地区脊髓性肌肉萎缩症治疗产业可望稳定发展。基因替代疗法和小分子药物等新型疗法的引入、认识的提高以及新生儿筛检计划的製定正在帮助扩大 SMA 患者的治疗选择。随着亚太地区经济的扩张，越来越多的资金被投入医疗保健、研究和发展领域。这对于 SMA 治疗的未来以及受 SMA 影响的人们的改善是令人鼓舞的。

脊髓性肌肉萎缩症治疗市场

  • Nusinersen，也称为 SpinrazaTM，是美国食品药物管理局(FDA) 批准的第一个治疗成人和儿童SMA 的药物。该药物的目的是刺激 SMN 蛋白的合成，这对于维持运动神经元至关重要。
  • Onasemnogene aveparobec-shioi (Zolgensma TM) 基因疗法已获得 FDA 批准，用于治疗 2 岁以下儿童儿童发病的 SMA 婴儿。目标运动神经元被转移了来自安全病毒的全功能人类 SMN 基因，从而增强肌肉的存活、功能和运动。
  • Rizdiplam（也称为 Evesdi）是 FDA 批准的口服药物，用于治疗 2 个月或以上的 SMA 患者。

目录

第一章简介

  • 市场概况
  • 市场定义
  • 调查范围
  • 市场区隔
  • 货币
  • 先决条件
  • 基准年和预测年时间表

第二章调查方法

  • 调查资料
  • 先决条件

第三章执行摘要

  • 研究亮点

第四章市场动态

  • 市场驱动因素
  • 市场限制因素
  • 波特五力分析
  • 产业价值链分析

第五章脊髓性肌肉萎缩症治疗市场：依类型

  • 介绍
  • 类型1
  • 2型
  • 类型3
  • 类型4

第六章脊髓性肌肉萎缩症治疗市场：依药物分类

  • 介绍
  • 斯宾拉扎
  • 佐尔根斯马
  • 每天
  • 其他的

第七章脊髓性肌肉萎缩症治疗市场：依给药途径

  • 介绍
  • 口服
  • 注射

第八章脊髓性肌肉萎缩症治疗市场：按地区

  • 介绍
  • 北美洲
    • 美国
    • 加拿大
    • 墨西哥
  • 南美洲
    • 巴西
    • 阿根廷
    • 其他的
  • 欧洲
    • 英国
    • 德国
    • 法国
    • 西班牙
    • 其他的
  • 中东/非洲
    • 沙乌地阿拉伯
    • 阿拉伯聯合大公国
    • 以色列
    • 其他的
  • 亚太地区
    • 日本
    • 中国
    • 印度
    • 韩国
    • 印尼
    • 泰国
    • 其他的

第九章竞争环境及分析

  • 主要企业及策略分析
  • 市场占有率分析
  • 合併、收购、协议和合作

第十章 公司简介

  • Cure SMA
  • Evrysdi
  • Biogen
  • Novartis
  • Zolgensma
  • Roche
  • Fierce Pharma
  • Spinraza
  • Cleveland Clinic
  • Aster DM Healthcare
简介目录
Product Code: KSI061616224

The spinal muscular atrophy treatment market is estimated to grow at a CAGR of 12.35% during the forecast period.

Approximately 1 in 10,000 persons have a hereditary condition known as spinal muscular atrophy (SMA). The majority of SMA instances are caused by a missing SMN1 segment, which prevents the gene from producing proteins. The survival motor neuron (SMN) protein, which is essential for preserving regular motor neuron activity, is mostly produced by SMN1. The severity of SMA varies in degrees. An infant with SMA has difficulty breathing, swallowing, sitting, and walking. SMA can potentially lead to an early death if therapy is delayed.

Growth drivers for Spinal Muscular Atrophy Treatment Market

The spinal muscular atrophy treatment industry is changing quickly due to discoveries in genetics, creative therapeutic approaches, and greater public awareness. Patient activism, newborn screening programs, and regulatory backing have all contributed to the acceleration of SMA therapy advancements. With more research being conducted and more accessible therapy alternatives, there is increasing hope for better results and a higher standard of living for those with SMA.

Increasing awareness about therapies

Numerous methods are used to treat spinal muscular atrophy (SMA). These include antisense oligonucleotide treatment with Spinraza, which aims to increase the synthesis of SMN proteins, and gene replacement therapies like Zolgensma, which provides a functioning SMN1 gene. Evrysdi and other small-molecule medications also target the development of SMN proteins. Treating SMA involves more than just raising the body's level of SMN protein. Other systems, pathways, and processes are also impacted by the loss of SMN protein, and these systems are the focus of further therapies. These methods are frequently referred to as non-SMN methods. Numerous non-SMN methods concentrate on the nerves or muscles.

Increasing R&D initiatives

In the field of medicine, the increase in R&D projects aimed at treating Spinal Muscular Atrophy (SMA) offers a significant growth driver for the spinal muscular atrophy treatment market growth. These projects, which range from gene therapy to small molecule medications and gene editing technologies, are driven by the urgent need for efficient treatments and a better understanding of the genetic causes of SMA. For instance, the FDA has approved two medications, Zolgensma® (onasemnogene abeparvovec-xioi) and EvrysdiTM (risdiplam), in 2019 and 2020, respectively. Moreover, several R&D pipelines are currently in the clinical stage, which gives hope for a full recovery from SMA. Furthermore, with current R&D projects including DEVOTE, RESPOND, and ASCEND, Biogen hopes to evaluate treatment alternatives and provide guidance to the SMA community as per their choice.

Clinical Trials for Spinal Muscular Atrophy Treatment

The spinal muscular atrophy treatment market is further driven by the increasing clinical trials. To encourage the quick creation and execution of clinical trials for neurological illnesses affecting adults and/or children, NINDS formed the NeuroNext (NINDS Network for Excellence in Neuroscience Clinical Trials) network. The network's goal is to create early-stage studies that evaluate novel treatments and uncover biomarkers, which are often physical characteristics or substances found in physiological fluids like blood that may be used to assess the existence and severity of a disease. Finding biomarkers for SMA and comprehending the underlying mechanisms and causes of the illness was one of these projects. Information from this research was used to determine whether to approve nusinersen (SpinrazaTM). The design of the next clinical trials in SMA has also been improved by the knowledge gained from the study.

Growing awareness of the newborn screening program

Increasing focus on the newborn screening program is boosting the spinal muscular atrophy treatment market growth. In this process, state public health laboratories utilize dried blood spots taken from a baby's heel during the first 24 to 48 hours of life as part of normal newborn screening to identify genetic diseases, including SMA. Laboratorians run a test to see if the missing gene sequence that causes SMA is the source of the disorder. The public health laboratory or a clinical laboratory conducts additional investigations to determine the number of copies of the SMN2 gene if the SMN1 gene is shown to be abnormal.

The spinal muscular atrophy treatment market in the Asia Pacific region is anticipated to grow steadily.

The Asia Pacific region's spinal muscular atrophy treatment industry is expected to develop steadily. The advent of novel therapeutics, including gene replacement therapies and small molecule pharmaceuticals, as well as increased awareness and the deployment of newborn screening programs are all helping to expand the treatment choices available to SMA patients. More funds are being devoted to healthcare, research, and development as the economies in the Asia Pacific region expand. This is encouraging for the future of SMA therapy and for the betterment of people who are impacted by the illness.

Spinal Muscular Atrophy Treatment Market

  • Nusinersen, also known as SpinrazaTM, is the first medication authorized by the US Food and Drug Administration (FDA) to treat SMA in both adults and children. The purpose of the medication is to boost the SMN protein's synthesis, which is essential for the upkeep of motor neurons.
  • Onasemnogene abeparovec-xioi (Zolgensma TM) gene therapy was authorized by the FDA for infants with infantile-onset SMA under the age of two. The targeted motor neurons receive a fully functioning human SMN gene from a safe virus, which enhances muscle survival, function, and movement.
  • Risdiplam, also known as Evesdi, is an oral medication that the FDA has authorized for the treatment of SMA in patients two months of age and older.

Market Key Developments

  • In June 2023, new SPINRAZA® (nusinersen) data were released by Biogen Inc. with the goal of- providing important information to the spinal muscular atrophy (SMA) community. Cure SMA hosted the SMA Research & Clinical Care Meeting, where the statistics were presented.
  • In August 2021, Novartis reported that the FDA of the United States has decided that OAV-101 intrathecal (IT) clinical studies for patients with spinal muscular atrophy (SMA) can move forward.

Segmentation:

By Type

  • Type 1
  • Type 2
  • Type 3
  • Type 4

By Drug

  • Spinraza
  • Zolgensma
  • Evrysdi
  • Others

By Route of Administration

  • Oral
  • Injection

By Geography

  • North America
  • United States
  • Canada
  • Mexico
  • South America
  • Brazil
  • Argentina
  • Others
  • Europe
  • United Kingdom
  • Germany
  • France
  • Spain
  • Others
  • Middle East and Africa
  • Saudi Arabia
  • UAE
  • Israel
  • Others
  • Asia Pacific
  • Japan
  • China
  • India
  • South Korea
  • Indonesia
  • Thailand
  • Others

TABLE OF CONTENTS

1. INTRODUCTION

  • 1.1. Market Overview
  • 1.2. Market Definition
  • 1.3. Scope of the Study
  • 1.4. Market Segmentation
  • 1.5. Currency
  • 1.6. Assumptions
  • 1.7. Base, and Forecast Years Timeline

2. RESEARCH METHODOLOGY

  • 2.1. Research Data
  • 2.2. Assumptions

3. EXECUTIVE SUMMARY

  • 3.1. Research Highlights

4. MARKET DYNAMICS

  • 4.1. Market Drivers
  • 4.2. Market Restraints
  • 4.3. Porter's Five Force Analysis
    • 4.3.1. Bargaining Power of Suppliers
    • 4.3.2. Bargaining Power of Buyers
    • 4.3.3. Threat of New Entrants
    • 4.3.4. Threat of Substitutes
    • 4.3.5. Competitive Rivalry in the Industry
  • 4.4. Industry Value Chain Analysis

5. SPINAL MUSCULAR ATROPHY TREATMENT MARKET BY TYPE

  • 5.1. Introduction
  • 5.2. Type 1
  • 5.3. Type 2
  • 5.4. Type 3
  • 5.5. Type 4

6. SPINAL MUSCULAR ATROPHY TREATMENT MARKET BY DRUG

  • 6.1. Introduction
  • 6.2. Spinraza
  • 6.3. Zolgensma
  • 6.4. Evrysdi
  • 6.5. Others

7. SPINAL MUSCULAR ATROPHY TREATMENT MARKET BY ROUTE OF ADMINISTRATION

  • 7.1. Introduction
  • 7.2. Oral
  • 7.3. Injection

8. SPINAL MUSCULAR ATROPHY TREATMENT MARKET BY GEOGRAPHY

  • 8.1. Introduction
  • 8.2. North America
    • 8.2.1. United States
    • 8.2.2. Canada
    • 8.2.3. Mexico
  • 8.3. South America
    • 8.3.1. Brazil
    • 8.3.2. Argentina
    • 8.3.3. Others
  • 8.4. Europe
    • 8.4.1. United Kingdom
    • 8.4.2. Germany
    • 8.4.3. France
    • 8.4.4. Spain
    • 8.4.5. Others
  • 8.5. The Middle East and Africa
    • 8.5.1. Saudi Arabia
    • 8.5.2. UAE
    • 8.5.3. Israel
    • 8.5.4. Others
  • 8.6. Asia Pacific
    • 8.6.1. Japan
    • 8.6.2. China
    • 8.6.3. India
    • 8.6.4. South Korea
    • 8.6.5. Indonesia
    • 8.6.6. Thailand
    • 8.6.7. Others

9. COMPETITIVE ENVIRONMENT AND ANALYSIS

  • 9.1. Major Players and Strategy Analysis
  • 9.2. Market Share Analysis
  • 9.3. Mergers, Acquisitions, Agreements, and Collaborations

10. COMPANY PROFILES

  • 10.1. Cure SMA
  • 10.2. Evrysdi
  • 10.3. Biogen
  • 10.4. Novartis
  • 10.5. Zolgensma
  • 10.6. Roche
  • 10.7. Fierce Pharma
  • 10.8. Spinraza
  • 10.9. Cleveland Clinic
  • 10.10. Aster DM Healthcare