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Spinal Muscular Atrophy (SMA) Therapeutics Pipeline to Witness Significant Growth due to Positive Clinical Results in the Coming Years

According to a new research report Spinal Muscular Atrophy (SMA) Therapeutics – Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations, and Other Developments” published by Pharma Proff, SMA therapeutics currently exhibits a proliferating pipeline with 15+ therapeutic candidates.

SMA Therapeutics Pipeline Insights

Spinal muscular atrophy (SMA) is a rare inherited genetic disease that is characterized by loss of nerve cells, known as motor neurons. Motor neurons receive nerve impulses that are transmitted from brain to spinal cord, and further transmit these impulses to muscles with the help of peripheral nerves. Loss of motor neurons can lead to atrophy and weakness in the muscles, which are closest to the trunk of the body such as back, hips, and shoulders. SMA is mainly caused due to mutation in survival motor neuron 1 (SMN1) and SMN2 genes in chromosome 5. Any mutation in SMN1 gene can lead to SMA atrophy. Based on age, the disease is categorized into two types: childhood-onset SMA and adult-onset SMA.

Symptoms associated with the disease include loss of reflexes or presence of tremor in hands and fingers, respiratory difficulties, and scoliosis (curvature of the spine). The diagnosis of SMA includes physical examination, blood test, and genetic testing. Spinraza (by Biogen Inc.), a SMN2-directed antisense oligonucleotide, is the only marketed drug available for the treatment of SMA.

Insights into Pipeline Segments

According to the research findings, majority of the pipeline drug candidates are being developed to be administered by oral route. It has been observed that the oral route of medication is convenient, less risk of systemic infections, and inexpensive in nature, and also provides improved patient’s compliance. Administration of therapeutics for SMA through oral route has shown promising results in clinical studies.

Positive Clinical Trial Results are Expected to Drive the SMA Therapeutics Pipeline Advancements

Companies that are involved in developing therapeutics for SMA have shown positive clinical results in various phases of drug development. For instance, in April 2018, AveXis Inc. presented phase I clinical data of a gene therapy, AVXS-101, at the 2018 Annual Meeting of the American Academy of Neurology (AAN) in Los Angeles. The drug candidate is being developed for the treatment of SMA type I. In the clinical study, AVXS-101 was found safe and well-tolerable.

SMA Therapeutics Pipeline to Witness Significant Growth due to Advanced Regulatory Approaches

In December 2018, Novartis AG announced that the U.S. Food and Drug Administration (USFDA) has accepted the company's biologics license application (BLA) for AVXS-101, also known as ZOLGENSMA, an investigational gene replacement therapy for the treatment of SMA type I.

Browse report overview with detailed TOC on "Spinal Muscular Atrophy (SMA) Therapeutics – Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations, and Other Developments" at:

Biogen Inc., AveXis Inc., F. Hoffman La-Roche Ltd., Cytokinetics Inc., and WAVE Life Sciences Ltd. are some of the key companies involved in the development of SMA therapeutics.

SMA Therapeutics Pipeline Analysis

  • By Phase
  • By Molecule Type
  • By Route of Administration
  • By Company

The report comprises detailed pipeline analysis of therapeutics being developed for the treatment of SMA. Comprehensive insights on the pipeline products have been provided, with special focus on strategic developments of key players, information on drug licensing, designations, financing, and grants, technological advancements, patents, and upcoming conferences. In addition, the report highlights the winning strategies of companies involved in the SMA therapeutics development. Detailed regulatory approval procedures in the U.S., Europe, and Japan are also provided in the report. Furthermore, the report contains competitive analysis and extensive information on monotherapies, combination therapies, targets and mechanisms of action, and drug origin with respect to SMA.