the pipeline for spinal muscular atrophy (SMA) drugs is running dry currently. The few drugs that are present, however, are highly advanced medical technologies progressing through different phases of clinical trials.
The key drugs in the spinal muscular atrophy pipeline are RG3039, scAAV9.CB.SMN, CK-2127107, RO6885247, LMI070, Olesoxime (TRO19622), and ISIS-SMNRx.
Ionis Pharma Solution Could Vastly Improve SMA Treatment
ISIS-SMNRx is a drug being developed by Ionis Pharmaceuticals for the treatment of spinal muscular atrophy.As of 2015, it was the only drug that had reached phase 3 of clinical trials.
The studies are expected to be completed by July 2017.
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“Should this drug be approved for the treatment of spinal muscular atrophy, it will create a highly lucrative opportunity for both Ionis Pharma and Biogen Idec,” states a TMR analyst. “This drug is expected to be released in 2018 and hold high expectations from the healthcare industry for the treatment of spinal muscular atrophy.”
Growing SMA Carrier Frequency Speeds up Solutions R&D
One of the key drivers affecting the growth of the market for spinal muscular atrophy drugs is the increasing number of carriers of the defective SMA gene. As per the findings revealed by CureSMA, nearly one out of 50 people are carrying the SMA defect.
Additionally, this statistic does not include a majority of the global population that for various reasons could not be included in the study. The total percentage of SMA gene carriers could therefore be effectively higher than previously mentioned.
This is creating a greater urge for healthcare and pharmaceutical organizations to create feasible solutions to treat spinal muscular atrophy, and is also pushing the organizations to increase awareness rates of SMA among people.
Lack of Advanced Research Slows Overall Development of SMA Treatments
There are currently multiple research institutes and government agencies that are working together to keep the total number of SMA cases in check. They do not, however, distinguish between the various types of spinal muscular atrophy afflicting the patients.
This reduces the accuracy of patient diagnosis and eventually reduces the overall rate of development of treatments for spinal muscular atrophy. At the same time, it makes disease mapping for trends extremely difficult.
The resultant lack of research is therefore hindering the chances of innovative treatment solutions and the identification of disease progression.
Large Areas Still Untapped for SMA Treatment to Provide Key Opportunities
The U.S. and Europe have designated SMA as an orphan disease. Between the U.S., Europe and Japan, between 25,000 and 50,000 people were diagnosed with spinal muscular atrophy as of June 2015, according to European Pharmaceutical Reviews.
The total number of spinal muscular atrophy patients across the world is as yet unknown.
The absence of an approved drug-based treatment for spinal muscular atrophy combined with the unknown number of SMA patients leaves a major unrealized market for SMA drugs that can be tapped into for greater opportunities.
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