Help save Spinal Muscular Atrophy (SMA) patients! Ionis Pharmaceuticals needs to apply for Accelerated Approval of Nusinersen (ISIS-SMNrx) for SMA, so they get the life-saving treatment SMA patients desperately need!
This petition had 150,118 supporters
“SMA (spinal muscular atrophy) is a disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe. It is the number one genetic cause of death for infants. SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene produces a protein that is critical to the function of the nerves that control our muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating and often fatal muscle weakness. SMA affects approximately 1 in 10,000 babies, and about 1 in every 50 Americans is a genetic carrier. SMA can affect any race or gender. There are four primary types of SMA—I, II, III, and IV—based on age of onset and highest physical milestone achieved. Individuals with SMA have difficulty performing the basic functions of life, like breathing and swallowing. However, SMA does not affect a person’s ability to think, learn, and build relationships with others” (CureSMA.org).
Currently, there is no cure. However, there is treatment in clinical trials now that is proving to be life-changing for those with SMA. Ionis Pharmaceuticals is conducting clinical trials with a drug called Nusinersen (ISIS-SMNrx) and it is currently in phase 3 of clinical trials. Only those meeting the trial criteria are allowed to participate. Data from phase 1 and 2 trials have shown positive safety results, tolerance of dose and delivery method, and substantial increases in the proper splicing of SMN2 RNA. In addition it has shown increases in SMN protein in CSF and in critically important motor neurons. Clinically significant improvements in motor function and achievement of new motor milestones such as sitting in SMA type 1 and walking in SMA type 2, and finally a statistically significant improvement in event-free survival in SMA type 1.
In 2012, Congress passed the Food and Drug Administration Safety Innovations Act (FDASIA). Section 901 of FDASIA amends the Federal Food, Drug, and Cosmetic Act (FD&C Act) to allow the FDA to base accelerated approval for drugs for serious conditions. Those conditions must fill an unmet medical need on whether the drug has an effect on a surrogate or an intermediate clinical endpoint. Based on the above positive outcomes, the clinical benefit of SMNRx has been clearly established. Various surrogate endpoints, biomarkers, and clinically meaningful outcomes including SURVIVAL have been well demonstrated (FDA.gov).
Given that SMA is an unmet medical need, we, The Fast Movement Organization (TheFastMovement.org), are petitioning Ionis Pharmaceuticals to apply with the FDA for Accelerated Approval of Nusinersen (ISIS-SMNrx). This allows those (babies, children, and adults) living with SMA treatment sooner than later. By signing this petition you are standing behind The Fast Movement and the SMA community for a chance at a better life sooner, and are helping push Ionis Pharmaceuticals to ask the FDA for Accelerated Approval for all people living with SMA.
Time is not on SMA's side. Someone living with SMA shouldn't have to wait any longer for treatment that is already changing the lives of those currently in trials.
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