FDA Approves New Treatment for Spinal Muscular Atrophy

motor neurons_1200.jpgSpinal muscular atrophy (SMA) is a genetic disorder characterized by loss of motor neurons, leading to weakness and wasting of muscles used for activities such as crawling, walking, sitting up, and controlling head movement. In severe cases of SMA, the muscles used for breathing and swallowing are affected. This devastating disorder affects approximately 1 in 10,000 births and is the most common genetic cause of infant mortality. SMA is caused by deletions or mutations in a gene that leads to a deficiency of survival motor neuron (SMN) protein; this protein is critical for the maintenance of motor neurons. The severity of SMA is directly related to the amount of SMN protein.

Spinraza is the first medication that’s been FDA approved for treatment of SMA. The novel agent is an antisense oligonucleotide (ASO) that is intended to increase production of SMN protein, with the goal of improving motor function in patients with SMA. 

Request the full report for Spinraza (Nusinersen) here.

Hayes Technology Prognosis tracked the regulatory process for Spinraza. As of December 23rd, what was once an investigational drug has now been approved by the Food and Drug Administration (FDA).

prognosis_1200.jpgWhether you’re a provider looking to stay abreast of health technology developments, or a payer needing assistance with your coverage policy determinations, Hayes Prognosis, our horizon scanning service, is an invaluable resource for you. Our overviews include:

  • Descriptions of the health problem addressed by the technology
  • Competing technologies
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  • Current evidence about the technology

In addition, we update our overviews as the technology progresses through clinical trial development and the regulatory approval process.

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Topics: Health Technologies, Payers, Hospitals, Government, Hayes Blog

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