Spinal Muscular Atrophy - A Pipeline Analysis Report

Overview of the drug development for spinal muscular atrophy

Spinal Muscular Atrophy (SMA) is a genetic disorder that results in the weakening of the muscles and affects muscle movement. It is common in infants, and it progresses with time. Infants with SMA have a breakdown of nerve cells in the brain and the spinal cord, due to which the brain stops sending control signals that control the muscle movement to the spinal cord. The muscles shrink and become weak, making it tough for the patients to control the head movement, sit without any external support and walk. People with a family history of SMA are at higher risks of developing the disease than the ones with no family history of SMA. The disease develops in the people with two fault copies of survival or motor neuron 1 (SMN1) gene. The people with only one fault copy of SMN1 gene are termed as the carriers of SMA. The disease does not develop in the carriers, and the profitability of the disease development in their offspring is higher. As per the data presented by the SMA Foundation, the incidence of SMA across all genders, races, and ethnic backgrounds is estimated to be 1 in every 10,000 children born. As a result, with the rising incidences of the disease, the drug development for spinal muscular atrophy is expected to rise during the next few years.

According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for spinal muscular atrophy (SMA). Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

Companies covered

This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of drug molecules for the treatment of spinal muscular atrophy. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the drug molecules discontinued by companies.

Some of the companies covered in this pipeline analysis report are –

• CYTOKINETICS
• Novartis
• Scholar Rock
• Voyager Therapeutics
• Recursion Pharmaceuticals

Therapeutic assessment of the drug development for spinal muscular atrophy by route of administration

• Oral
• Intravenous
• Intrathecal

The oral route of administration (ROA) involves the application of the drug directly through the mouth cavity, which will have a more direct effect on the target cells.

Therapeutic assessment of the drug development for spinal muscular atrophy by therapy

• Monotherapy

According to this pipeline analysis report, most of the molecules that are currently in the drug development for spinal muscular atrophy are being developed as monotherapy drugs and most of these molecules are in the pre-clinical stage of development.

Key questions answered in the report include

• What are the drug molecules in the various development stages for spinal muscular atrophy?
• What are the companies that are currently involved in the development of drug molecules for spinal muscular atrophy?
• Insight into discontinued/inactive molecules with appropriate reasoning?
• What are the major regulatory authorities approving drugs in various regions?
• Detailed profiling of each active molecule

Technavio also offers customization on reports based on specific client requirement.