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May 29, 2020 at 11:00 am #23687DeAnn RKeymaster
It’s hard to believe I’ve been on Spinraza for almost 3 years already. Growing up I never fathomed there would be a treatment let alone more than one! It’s an exciting era in the SMA world. Today I wanted to briefly touch on what’s available and what we’re anticipating. Of course I’m only basing this on my personal experience and knowledge, so please talk to your doctors for the most accurate information.
First on the market came Spinraza. FDA approved in December 2016, the broad label doesn’t restrict use based on age or type of SMA. However, because this medication needs to cross the blood/brain barrier administration is through a lumbar puncture. Without getting too scientific Spinraza works to stop or slow the progressions by fixing the splicing error in the SMN2 gene that we rely on to make functional protein. Requiring a series of injections to start off called loading doses, then ongoing injections every 4 months are necessary. It comes at a cost of $125,000 per injection. Not including procedure costs.
Next on the market came Zolgensma. Because it’s a revolutionary gene modifying treatment it’s really exciting. Not only does it replace the missing SMN1 gene using a viral vector it’s a one-time IV administered treatment. However, it’s only approved for pediatric patients right now. Despite that, two treatment options within a couple years is incredible. Finally, parents have options! With increased infant screening and treatment options the future is brighter than ever for the kiddos. At a cost of around $2.1 million it carries a hefty price tag. Although if you compare it to Spinraza it is less expensive in the long run.
One year later, risdiplam is vying for the next treatment option. Because it’s pending FDA approval many details are unknown. Although I do know it’s an oral medication taken daily. From my understanding it works much like Spinraza in that it corrects the splicing error in the SMN2 gene. Allowing our bodies to create functional protein. Expected FDA approval is sometime in August. My suspicion is for a broad label, but time will tell. Fingers crossed cost isn’t astronomical either.
Another treatment I’m keeping my eye on is SRK-015. For this one it seems like a treatment that may work well in conjunction with other treatments. As far as I know it’s still in trials. I’m probably not the best at explaining how it works. Basically from my understanding is inhibits whatever tells your muscles to stop growing.
If you have anything to add about any of these treatment options please comment below. Likewise if you have questions, throw them out there. Are you on any of these treatments? Are you pursuing them? What do you find most intriguing? Let me know!
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