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2020 Spinal Muscular Atrophy Pipeline Insights -

·5-min read

The "Spinal Muscular Atrophy - Pipeline Insight, 2020" drug pipelines has been added to's offering.

The "Spinal Muscular Atrophy - Pipeline Insight, 2020," report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in the Spinal Muscular Atrophy pipeline landscape.

It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Spinal Muscular Atrophy Emerging Drugs Chapters

This segment of the Spinal Muscular Atrophy report encloses its detailed analysis of various drugs in the different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Spinal Muscular Atrophy Emerging Drugs

Sodium valproate: Kowa

Sodium valproate is being developed by Kowa for the treatment of Spinal muscular atrophy. It is currently in phase III stage of development.

SRK-015: Scholar Rock

SRK-015, our most advanced product candidate, is a selective and local inhibitor of the activation of myostatin. Based on existing research on the mechanism of myostatin in muscle growth and strength, Scholar Rock believes the inhibition of the activation of myostatin with SRK-015 may promote a clinically beneficial increase in motor function. Scholar Rock is developing and investigating SRK-015 as a potential treatment to improve muscle strength and motor function in patients with Spinal Muscular Atrophy (SMA).it is currently in phase II stage of development.

BIIB110: Biogen

BIIB110 is a hybrid activin II receptor (ACTIIR) ligand trap that sequesters both myostatin and activins while sparing the related ligand bone morphogen protein 9 (BMP9). This targeted mechanism of action may result in greater muscle mass, function and improved safety compared to other myostatin inhibition approaches. It is currently in phase I stage of development.

Research programme: SNA based therapeutics: Exicure

SNA based therapeutics is being developed by Exicure for the treatment of Spinal muscular atrophy. It is currently in preclinical stage of development.

Spinal Muscular Atrophy: Therapeutic Assessment

This segment of the report provides insights about the different Spinal Muscular Atrophy drugs segregated based on the following parameters that define the scope of the report, such as:

Major Players in Spinal Muscular Atrophy

There are approx. 40+ key companies which are developing therapies for Spinal Muscular Atrophy. The companies which have their Spinal Muscular Atrophy drug candidates in the most advanced stage, i.e. phase III include Kowa and others.

This report covers around 40+ products under different phases of clinical development like

  • Late-stage products (Phase II and Phase II/III)

  • Mid-stage products (Phase II and Phase II/III)

  • Early-stage products (Phase I/II and Phase I) along with the details of

  • Pre-clinical and Discovery stage candidates

  • Discontinued & Inactive candidates

  • Route of Administration

Spinal Muscular Atrophy pipeline report provides therapeutic assessment of the pipeline drugs by the Route of Administration.

Products have been categorized under various ROAs such as

  • Infusion

  • Intradermal

  • Intramuscular

  • Intranasal

  • Intravenous

  • Oral

  • Parenteral

  • Subcutaneous

  • Topical.

  • Molecule Type

Products have been categorized under various Molecule types such as

  • Gene therapies

  • Small molecule

  • Vaccines

  • Polymers

  • Peptides

  • Monoclonal antibodies

  • Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Spinal Muscular Atrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Spinal Muscular Atrophy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Spinal Muscular Atrophy drugs.

Report Highlights

  • The companies and academics are working to assess challenges and seek opportunities that could influence Spinal Muscular Atrophy R&D. therapies under development are focused on novel approaches to treat/improve Spinal Muscular Atrophy.

  • In August 2020, Scholar Rock announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA).

  • In March 2018, Scholar Rock announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead antibody product candidate, SRK-015, for the treatment of spinal muscular atrophy (SMA).

  • In July 2018, Biogen announced the acquisition of the muscle enhancement programme from AliveGen, which includes candidate, BIIB 110 (previously known as ALG 801).

Spinal Muscular Atrophy Report Insights

  • Spinal Muscular Atrophy Pipeline Analysis

  • Therapeutic Assessment

  • Unmet Needs

  • Impact of Drugs

Spinal Muscular Atrophy Report Assessment

  • Pipeline Product Profiles

  • Therapeutic Assessment

  • Pipeline Assessment

  • Inactive drugs assessment

  • Unmet Needs

Key Questions Answered

Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Spinal Muscular Atrophy drugs?

  • How many Spinal Muscular Atrophy drugs are developed by each company?

  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Spinal Muscular Atrophy?

  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Spinal Muscular Atrophy therapeutics?

  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?

  • What are the clinical studies going on for Spinal Muscular Atrophy and their status?

  • What are the key designations that have been granted to the emerging drugs?

Key Players

  • Kowa

  • Scholar Rock

  • Biogen

  • Exicure

  • AveXis

  • Novartis Pharmaceuticals

  • Catalyst Pharmaceuticals

  • ReveraGen Biopharma

  • Translate Bio

Key Products

  • Sodium valproate

  • SRK-015

  • BIIB110

  • Leadiant Biosciences

  • Vamorolone

Research programme: SNA based therapeutics

  • Amifampridine Phosphate

  • Nusinersen

  • Onasemnogene

  • Branaplam

  • Valproic Acid

For more information about this drug pipelines report visit

View source version on

Laura Wood, Senior Press Manager

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