Spinal Muscular Atrophy Pipeline Drugs Analysis 2025: Clinical Trials, EMA, PDMA, FDA Approvals, Medication, Treatment Market, NDA Approval, Therapies, ROA, MOA and Companies by DelveInsight

Spinal Muscular Atrophy Pipeline

(Albany, USA) “Spinal Muscular Atrophy Pipeline Insight, 2025″ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Spinal Muscular Atrophy Market.

The Spinal Muscular Atrophy clinical trials report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

Spinal Muscular Atrophy pipeline constitutes 18+ key companies continuously working towards developing 20+ Spinal Muscular Atrophy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

To know more in detail about Spinal Muscular Atrophy pipeline report offerings, click here: Spinal Muscular Atrophy Clinical Trials and Pipeline

Some of the key takeaways from the Spinal Muscular Atrophy Pipeline Report:

• In March 2025, Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational muscle-targeted treatment that is being developed to provide clinically meaningful improvement in motor function for people living with spinal muscular atrophy (SMA) who are receiving an SMN-targeted treatment. The FDA will review the application under priority review and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2025. The FDA priority review designation conveys that the FDA has determined that if apitegromab is approved, it could offer significant improvement in the safety or effectiveness of treatment of the serious condition of SMA
• Spinal Muscular Atrophy Companies across the globe are diligently working toward developing novel Spinal Muscular Atrophy treatment therapies with a considerable amount of success over the years.
• Leading Spinal Muscular Atrophy companies working in the market are Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics, and others, are developing therapies for the Spinal Muscular Atrophy treatment
• Spinal Muscular Atrophy emerging therapies such as – ACTX-401, Apitegromab, GYM329, and others are expected to have a significant impact on the Spinal Muscular Atrophy market in the coming years.
• In February 2025, Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi® (risdiplam) tablet for people living with spinal muscular atrophy (SMA). Evrysdi is the only non-invasive disease-modifying treatment for SMA. The 5 mg Evrysdi tablet can either be swallowed whole or dispersed in water.
• In January 2025, Biogen Inc. (Nasdaq: BIIB) announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and the European Medicines Agency (EMA) has validated the application for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA). The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months, compared to the approved nusinersen regimen (SPINRAZA®).
• On April 2024, Hoffmann-La Roche announced a Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy
• On April 2024, Genentech announced results of a multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.
• On April 2024, Biogen announced results of a study whose primary objective is to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children’s Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C).
• On January 2024, Novartis announced results of a Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®).
• On January 2024, NMD Pharma A/S announced results of a Phase 2, Randomised, Double-blind, Placebo-controlled, 2-way Crossover Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 in Ambulatory Adults With Type 3 Spinal Muscular Atrophy
• On December 2023, Novartis announced results of a Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) – ARISER Study.
• On May 2023, Biogen announced results of a study whose primary objective is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA)

To know in detail about the Spinal Muscular Atrophy clinical trials and recent FDA approvals for Spinal Muscular Atrophy drugs, click here: Spinal Muscular Atrophy Drugs and Therapies

Spinal Muscular Atrophy Overview

Spinal Muscular Atrophy (SMA) is a rare genetic neuromuscular disorder that affects motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. It is caused by mutations in the SMN1 (Survival Motor Neuron 1) gene, which results in reduced levels of the SMN protein essential for motor neuron survival. The severity of SMA varies, and it is classified into different types (SMA 0–4) based on age of onset and disease progression.

Spinal Muscular Atrophy primarily affects voluntary muscles, particularly those involved in movement, breathing, and swallowing. Spinal Muscular Atrophy Symptoms range from severe muscle weakness in infancy (SMA Type 1, the most common and severe form) to milder symptoms with later onset (SMA Type 3 and 4). In severe cases, respiratory complications can be life-threatening.

Spinal Muscular Atrophy Diagnosis is confirmed through genetic testing. While there is no cure, disease-modifying therapies like nusinersen (Spinraza), risdiplam (Evrysdi), and gene therapy with onasemnogene abeparvovec (Zolgensma) can improve motor function and survival. Supportive care, including physical therapy, respiratory support, and nutritional management, plays a crucial role in improving the quality of life.

Spinal Muscular Atrophy Advancements in treatment and early intervention, especially through newborn screening, have significantly improved outcomes for individuals with SMA.

Spinal Muscular Atrophy Pipeline Therapeutics Assessment

• Spinal Muscular Atrophy Assessment by Product Type
• Spinal Muscular Atrophy By Stage and Product Type
• Spinal Muscular Atrophy Assessment by Route of Administration
• Spinal Muscular Atrophy By Stage and Route of Administration
• Spinal Muscular Atrophy Assessment by Molecule Type
• Spinal Muscular Atrophy by Stage and Molecule Type

DelveInsight’s Spinal Muscular Atrophy Report covers around 20+ products under different phases of clinical development like

• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Emerging Spinal Muscular Atrophy Drugs Under Different Phases of Clinical Development Include:

• ACTX-401: Novartis
• Apitegromab: Scholar Rock
• GYM329: Roche
• And Many Others

Get a Free Sample PDF Report to know more about Spinal Muscular Atrophy Pipeline Assessment- Spinal Muscular Atrophy Medication

Spinal Muscular Atrophy Pipeline Analysis:

• The Spinal Muscular Atrophy pipeline report provides insights into
• The report provides detailed insights about companies that are developing therapies for the Spinal Muscular Atrophy treatment with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Spinal Muscular Atrophy Treatment.
• Spinal Muscular Atrophy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Spinal Muscular Atrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Spinal Muscular Atrophy market.
• The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Further Spinal Muscular Atrophy product details are provided in the report. Download the Spinal Muscular Atrophy pipeline report to learn more about the emerging Spinal Muscular Atrophy therapies @ Spinal Muscular Atrophy Companies and Drugs

Scope of Spinal Muscular Atrophy Pipeline Drug Insight

• Coverage: Global
• Key Spinal Muscular Atrophy Companies: Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics, and others
• Key Spinal Muscular Atrophy Therapies: AS-202, HK001, Tegoprubart, AP-101, BLZ945, ANX005, MN-166, TW001, BIIB067, and others
• Spinal Muscular Atrophy Therapeutic Assessment: Spinal Muscular Atrophy current marketed and Spinal Muscular Atrophy emerging therapies
• Spinal Muscular Atrophy Market Dynamics: Spinal Muscular Atrophy market drivers and Spinal Muscular Atrophy market barriers

Request for Sample Report @ Spinal Muscular Atrophy Pipeline Outlook

Table of Contents

• Spinal Muscular Atrophy Report Introduction
• Spinal Muscular Atrophy Executive Summary
• Spinal Muscular Atrophy Overview
• Spinal Muscular Atrophy- Analytical Perspective In-depth Commercial Assessment
• Spinal Muscular Atrophy Pipeline Therapeutics
• Spinal Muscular Atrophy Late Stage Products (Phase II/III)
• Spinal Muscular Atrophy Mid Stage Products (Phase II)
• Spinal Muscular Atrophy Early Stage Products (Phase I)
• Spinal Muscular Atrophy Preclinical Stage Products
• Spinal Muscular Atrophy Therapeutics Assessment
• Spinal Muscular Atrophy Inactive Products
• Company-University Collaborations (Licensing/Partnering) Analysis
• Spinal Muscular Atrophy Key Companies
• Spinal Muscular Atrophy Key Products
• Spinal Muscular Atrophy Unmet Needs
• Spinal Muscular Atrophy Market Drivers and Barriers
• Spinal Muscular Atrophy Future Perspectives and Conclusion
• Spinal Muscular Atrophy Analyst Views
• Appendix
• About DelveInsight

About DelveInsight

DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences.

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