Guest Voice: Tempering expectations and keeping hope alive
The apitegromab approval process reminded me of some hard lessons
Joanna Buoniconti is an optimist by nature. But the SMA therapy approval process has tested her resolve. (Courtesy of Joanna Buoniconti)
For much of the time, the reality for those of us living with a chronic condition like SMA entails having something dangled in front of our faces, just out of reach — which isn’t challenging to do, considering our severe mobility limitations. Whether it’s treatments that seem to be perpetually on the horizon or a therapy that doesn’t work as effectively as it used to, hope is often snatched away from us at so many junctures. As one might imagine, it’s emotionally draining and exhausting.
Before I get into the heart of the issue, I want to preface this by saying that I’m generally an optimistic person. I’ve always had a knack for finding a silver lining in just about anything, but I had to learn the hard way not to get my hopes up too much about things. Learning this art is often an act of self-preservation.
To flesh out the scenario I want to discuss, let’s backtrack. The biopharmaceutical company Scholar Rock has been developing an investigational infusion therapy called apitegromab as an add-on treatment to improve motor function in people with SMA. The therapy has had positive results in clinical trials, and many of us in the SMA community had hoped the U.S. Food and Drug Administration (FDA) would approve it this year.
We applauded when, in January, Scholar Rock asked the FDA for priority review for its application, a process that shortens the review time to six months instead of the usual 10 months.
Unfortunately, a couple weeks ago, the FDA declined to approve apitegromab as a treatment for SMA, due to issues at a third-party fill-finish facility. Scholar Rock said it plans to resubmit its application seeking approval in the near future.
I’ve had to come to terms with similar setbacks involving Spinraza (nusinersen), some of which were harrowing experiences for me. Although Spinraza, the first approved treatment targeting the underlying cause of SMA, continues to help me, it no longer has the same effect on my muscles as it once did. So I still have a lot of hope — yes, you read that correctly — for how a medication like apitegromab could help me, given that it is specifically designed to strengthen atrophied motor muscles.
An unexpected blip happened when my mom was on an SMA forum and got wind of an expanded access program (EAP) for apitegromab at the hospital I go to. I emailed my contact in the neurology department to inquire about the program, because I really wanted to participate. My contact replied that the research coordinator for the EAP would be in touch the following week. In that moment, I was so excited and, dare I say, hopeful.
This feeling ended up biting me in the butt a couple days later when I received an email explaining that the EAP was already closed to additional participants. The silver lining, or so I thought, was that many of us hoped the therapy would be approved by the end of September anyway. If I could’ve regained some of the motor muscle function that I’d lost a decade ago, it would’ve been an early Christmas present this year. Sadly, that won’t be the case now.
It hurts, but I was able to move past the disappointment. Plus, I gained something useful from it: the topic of this column.
The important thing to remember, I think, is that having hope is a part of life, but so is learning to manage our expectations and taking care of ourselves emotionally in the aftermath.
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Note: SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of SMA News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to spinal muscular atrophy.
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