Spinal Muscular Atrophy Disease Pipeline Drugs Assessment: Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2018

Spinal Muscular Atrophy Disease Pipeline Drugs Assessment

Overview:

Spinal Muscular Atrophy is a genetic neuromuscular disease which is characterized by muscle atrophy and weakness. This disease generally manifests in early ages and may leads to the genetic cause of death in toddlers and infants. SMA is caused due to the defects in Survival Motor Neuron 1 (SMN1) gene which encodes the SMN protein. The SMN protein plays a vital role in health and survival of nerve cells in spinal cord responsible for muscle contraction (motor neurons).

Symptoms of SMA includes weak arms and legs, movement problems, tremors, swallowing problems, difficulty in breathing, problems in joints and bones.Spinal Muscular Atrophy is diagnosed by molecular genetic testing, electromyogram, CT Scan, MRI, Muscle tissue biopsy.  Spirinza (Nusinersen) is the only FDA approved drug to treat SMA. Spirinza was approved in 2016 by U.S. FDA.

Segmentation:

By Trial Phase, Spinal Muscular Atrophy pipeline drugs are segmented as:

• Preclinical Trials
• Phase 1
• Phase 2
• Phase 3
• Phase 4

By Company, Spinal Muscular Atrophy pipeline drugs are segmented as:

• Novartis
• Ionis Pharmaceuticals
• Biogen
• Hoffmann-La Roche Ltd
• PTC Therapeutics
• Cytokinetics
• Sanofi
• Genzyme
• Others

By Drugs, Spinal Muscular Atrophy pipeline drugs are segmented as:

• Hydroxyurea
• Branaplam
• Pyridostigmine Bromide
• Nusinersen
• Valproic Acid
• Levocarnitine
• Celecoxib
• Sodium phenylbutyrate
• Others

By Mechanism of Action, Spinal Muscular Atrophy pipeline drugs are segmented as:

• survival of motor neuron SMN2 Splicing Modifier
• Neuroprotectant
• SMN Gene  Replacement
• Muscle  Activator
• Others

By Types of Spinal Muscular Atrophy diseases, Spinal Muscular Atrophy pipeline drugs are segmented as:

• SMA Type 0
• SMA Type 1
• SMA Type 2
• SMA Type 3
• SMA Type 4

By Route of Administration, Spinal Muscular Atrophy pipeline drugs are segmented as:

• Oral
• Parenteral

 

Space Analysis:

• In November 2017, Catalyst Pharmaceuticals, Inc initiated phase II proof-of concept study determining the safety, efficacy and tolerability of its pipeline molecule, Firdapse (amifampridine phosphate) for the treatment of spinal muscular atrophy type 3.
• In December 2017, AveXis, Inc, initiated Phase I clinical trial of AVXS-101 for patients with spinal muscular atrophy type 2 via intrathecal route. AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Types 1 and 2, designed to address the monogenic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons, providing rapid onset of effect and crossing the blood brain barrier to allow targeting of both central and systemic features.

Report Description:

Spinal Muscular Atrophy Disease Pipeline Drugs Assessment report studies the various therapeutics under clinical development for Spinal Muscular Atrophy treatment along with targets for various drug candidate. The report provides plethora of information pertaining to trail phases, companies involved in the Spinal Muscular Atrophy disease pipeline drugs development. This report studies the dynamics of the Spinal Muscular Atrophy Disease Pipeline Drugs i.e. drivers, challenges and opportunities which are significantly impacting the product development. The report provides various information pertaining the clinical trials such as designation, grants, patents, and technology among others. Moreover, the report on Spinal Muscular Atrophy disease pipeline drugs assessment comprehensively presents the geographic location, trial status information along with key players involved in the therapeutics development.

Key Features of the Report:

• Provides the information related to universities and research institutes working in the therapeutics development
• Report comprehensively covers the all active and discontinued studies
• Studies the entire pipeline with special emphasis on companies actively involved in the therapeutics development
• Presents the prominent targets for drug development in each stage of clinical trial
• Provides the in-depth analysis on the each drug candidates in the clinical trial phases

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Location

GEOGRAPHY