首頁 > 市場調查報告書 > 製藥

市場調查報告書

商品編碼

1425124

脊髓性肌肉萎縮症治療市場－2024年至2029年預測

Spinal Muscular Atrophy Treatment Market - Forecasts from 2024 to 2029

出版日期: 2024年01月29日 | 出版商:

Knowledge Sourcing Intelligence | 英文 146 Pages | 商品交期: 最快1-2個工作天內

價格

簡介目錄

預計脊髓性肌肉萎縮症治療市場在預測期內將以 12.35% 的複合年成長率成長。

大約萬分之一的人患有一種稱為脊髓性肌肉萎縮症 (SMA) 的遺傳性疾病。大多數 SMA 是由 SMN1 片段缺陷引起的，該缺陷阻止基因產生蛋白質。運動神經元存活 (SMN) 蛋白對於維持正常運動神經元活動至關重要，主要由 SMN1 產生。 SMA 的嚴重程度各不相同。患有 SMA 的嬰兒會出現呼吸、吞嚥、坐立和行走困難。如果延誤治療，SMA 可能會導致過早死亡。

脊髓性肌肉萎縮症治療市場的成長動力

由於基因發現、創造性治療方法和公眾意識的提高，脊髓性肌肉萎縮症治療行業正在迅速改變。患者流動、新生兒篩檢計畫和監管支持都有助於加速 SMA 治療的進展。隨著更多研究的開展和更容易獲得的治療方法，SMA 患者獲得更好結果和更高生活水準的希望越來越大。

治療方法意識不斷增強

許多方法用於治療脊髓性肌肉萎縮症（SMA）。其中包括反義寡核苷酸治療（如 Spinraza）和基因替代療法（如 Zolgensma），前者旨在增加 SMN 蛋白的合成，後者使 SMN1 基因發揮功能。 Evrysdi 和其他小分子藥物也針對 SMN 蛋白的發育。僅增加體內 SMN 蛋白的水平不足以治療 SMA。其他系統、途徑和過程也受到 SMN 蛋白流失的影響，使這些系統成為進一步治療的重點。這些方法通常稱為非 SMN 方法。許多非 SMN 方法都專注於神經和肌肉。

加大研發力度

在醫療領域，越來越多的旨在治療脊髓性肌肉萎縮症（SMA）的研發計劃是脊髓性肌肉萎縮症治療市場成長的關鍵成長要素。這些計劃的範圍從基因治療到小分子藥物和基因編輯技術，是由對有效治療的迫切需求和更好地了解脊髓性肌肉萎縮症的遺傳原因所驅動的。例如，FDA分別於2019年和2020年核准了兩種藥物Zolgensma®（onasemnogene abeparvovec-xioi）和EvrysdiTM（risdiplam）。此外，多個開發平臺目前正處於臨床階段，為 SMA 的全面復健帶來希望。此外，百健（Biogen）希望透過目前的研發計劃（包括 DEVOTE、RESPOND 和 ASCEND）來評估替代治療方法並為 SMA 群體的選擇提供指南。

治療脊髓性肌肉萎縮症的臨床試驗

臨床試驗數量的增加進一步推動了脊髓性肌肉萎縮症治療市場的發展。為了鼓勵快速創建和實施影響成人和兒童的神經系統疾病的臨床試驗，NINDS 組建了 NeuroNext（NINDS 神經科學臨床試驗卓越網路）網路。該網路的目標是進行早期研究以評估新治療方法並發現生物標記。生物標記是物理特徵或物質，通常存在於血液等生理液體中，用於評估疾病的存在和嚴重程度。尋找 SMA 生物標記並了解該疾病的潛在機制和原因就是這樣的計劃之一。這項研究的資訊用於確定是否核准nusinersen (SpinrazaTM)。從這項研究中獲得的知識也改進了 SMA 下一個臨床試驗的設計。

提高對新生兒篩檢計劃的認知

對新生兒篩檢計劃的日益關注正在推動脊髓性肌肉萎縮症治療市場的成長。在此過程中，州公共衛生實驗室使用出生後 24 至 48 小時內從嬰兒腳跟採集的乾血點來識別遺傳性疾病，包括 SMA，作為常規新生兒篩檢的一部分。技術人員進行測試以確定導致 SMA 的基因缺失是否是導致疾病的原因。如果發現 SMN1 基因異常，公共衛生實驗室或臨床實驗室將進行額外測試以確定 SMN2 基因的拷貝數。

亞太地區脊髓性肌肉萎縮症治療市場預計將呈現穩定成長。

亞太地區脊髓性肌肉萎縮症治療產業可望穩定發展。基因替代療法和小分子藥物等新型療法的引入、認知的提高以及新生兒篩檢計劃的製定正在幫助擴大 SMA 患者的治療選擇。隨著亞太地區經濟的擴張，越來越多的資金被投入醫療保健、研究和發展領域。這對於 SMA 治療的未來以及受 SMA 影響的人們的改善是令人鼓舞的。

脊髓性肌肉萎縮症治療市場

Nusinersen，也稱為 SpinrazaTM，是美國食品藥物管理局(FDA) 批准的第一個治療成人和兒童SMA 的藥物。該藥物的目的是刺激 SMN 蛋白的合成，這對於維持運動神經元至關重要。
Onasemnogene aveparobec-shioi (Zolgensma TM) 基因療法已獲得 FDA 批准，用於治療 2 歲以下兒童兒童發病的 SMA 嬰兒。目標運動神經元被轉移了來自安全病毒的全功能人類 SMN 基因，從而增強肌肉的存活、功能和運動。
Rizdiplam（也稱為 Evesdi）是 FDA 批准的口服藥物，用於治療 2 個月或以上的 SMA 患者。

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

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