척수성 근위축증 치료 시장 : 예측(2024-2029년)

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