Approval of 1st FA treatment changed nothing and everything
What the FDA approval of Skyclarys really means for the FA community
Melodramatic title aside, Tuesday, Feb. 28, was a nail-biter. The U.S. Food and Drug Administration (FDA) was expected to make a decision regarding the approval of Skyclarys (omaveloxolone) for my rare disease, Friedreich’s ataxia (FA). A long and bumpy road had led to this decision, and the day was especially gut-wrenching because, if approved, Skyclarys would be the first-ever treatment for FA.
Since my diagnosis in elementary school, FA has been a grim death sentence; I could only watch as my body and abilities eroded.
A life with FA is a life of waiting — waiting to lose coordination, waiting to use the elevator, waiting for your custom wheelchair to arrive, waiting to find an accessible bathroom, waiting until someone is available to drive you around. After years of waiting for a treatment to be approved, I’d become jaded and didn’t expect any positive news regarding FA developments.
The idea that the FDA would actually approve an FA treatment seemed unrealistic.
A game-changing decision
As the morning of Feb. 28 became afternoon, there was still no announcement about Skyclarys. Just before evening, I was added to a group text with Ethan Ash, vice president of business development here at BioNews (the parent company of Friedreich’s Ataxia News), and Chris Comish, BioNews CEO. Both guys are friends of mine, and I felt lucky to be in solidarity with them as we refreshed data feeds and awaited any news from the FDA.
The group text was quiet for a while, with no news to report. My pessimism had clouded my outlook by the time my phone buzzed a little after 4 p.m. CST. Ethan had shared a screenshot of an article, published by a company I didn’t recognize, titled “FDA Approval for SKYCLARYS issued to REATA PHARMACEUTICALS INC.”
I was still skeptical (or perhaps jaded) until Chris replied a few seconds later: “Approved!!!”
Time seemed to stop for me. I mentally checked out of the group text. Before I allowed myself to be flooded with emotions, I had to assess two opposing perspectives and acknowledge that both were true.
First, nothing had changed. Even though the drug had been approved for FA patients ages 16 and older in the U.S., it’s a treatment — not a cure.
While Skyclarys has been clinically proven to slow the rate of disease progression, FA will continue to exist and have devastating outcomes. It’s tempting to hope that this drug will obliterate every challenge FA patients like me face, but that false expectation will only lead to disappointment.
Two years ago, I wrote a column about how many people, including me, wanted this drug to be a deus ex machina — a literary device in which all conflict in a story is suddenly and divinely solved.
The truth is, it may not be obvious that someone is taking Skyclarys. While the drug may help slow disease progression, it could be disappointing not to see FA patients “get better.”
But for me, another perspective supersedes that potential disappointment: With the approval of Skyclarys, everything has changed.
As I wrote on Feb. 28, this decision shifted FA’s status from untreatable to treatable. Now, instead of passively waiting for the disease to run its course, we in the FA community have an opportunity to be proactive in our care. Plus, Skyclarys was never expected to be the only treatment for FA; it’s just the first.
Going from having zero tools to having one is more than a little victory for the FA community. It means we can see ourselves as underdogs instead of victims.
That’s a real dark-horse story.
If you have have FA and are a U.S. citizen age 16 or older, talk to your healthcare provider about whether Skyclarys is a good option for you. The Reata Education, Access, and Care Helpline, or REACH, is also available to help eligible patients access the therapy.
On April 20, Reata Pharmaceuticals will host two national webcasts to share information about Skyclarys. The presentations will feature me and the “Little Victories” column. You’re invited to register here.
Note: Friedreich’s Ataxia News 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 another 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 Friedreich’s Ataxia News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Friedreich’s ataxia.
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Comments
Kathryn Renton
My grandson has FA and is 16 years old. However, he lives in Northern Ireland. Is there any news about this drug being approved for Great Britain and N. Ireland?
Heather Deville
Reata are not currently pursuing approval in GB & NI until their European application is completed - which is due to be by April 2024. Hopefully they will think again!
katie
slowing progression is not nothing.
Matthew Lafleur
I agree, and so does this column.
Shannon Barr
While this drug is something to be celebrated, my hope now becomes it will not hinder future trials for a cure. The drug companies already have a small pool of patients to pull from and if the majority of these patients, 16+ are on Skyclarys, they will have to come off to participate in a clinical trial or the clinical trial will have to allow them to remain on the drug which will most likely not happen because it could interfere with the results.
So as an FA’er, do you stay on a drug that’s approved to slow progression or do you take a chance and still participate in a clinical trial that could potentially cure? Big decisions that will have big impact, in my opinion, on future the future of FA.
Bridget
My daughter has suffered from FA since adolescence. As a caretaker and parent the announcement of Skyclary gave our family so much happiness and joy. When faced with the fact that my child will live a life with a progressive neuromuscular condition the word HOPE has been a word in our home and sometimes has been the only thing that has pushed us through. I must admit that this writing has put a pin in our HOPE balloon. I understand that it is your thoughts and feelings but how dare you try to take away happiness from believing that this medicine may help. Studies have shown that staying positive while facing difficulties can be therapeutic. Keep your bad thoughts to yourself.
Matthew Lafleur
Hey Bridget, the last thing I ever want to do is to take away hope from anyone. The point of my column as a whole is to encourage others in the FA community and to show them that even though there are setbacks in life with FA, hope is worth holding onto.
I try to balance optimism with the discouragement that often comes with FA. My goal is to always end on hope, even in this piece.
Jonathan Dorman
I like authentic opinion. The author acknowledges some reality so balloons aren't "filled" with empty platitudes. The last two sentences of this article replaced my balloon and filled it with even more hope.
"Going from having zero tools to having one is more than a little victory for the FA community. It means we can see ourselves as underdogs instead of victims." Those statements are powerful.
Laura Lampkin
I am an FA patient and I am having trouble getting a doctor to prescribe the drug! My regular doctor will not and I went to my neurologist and she will not😩 Anybody else having this problem? HELP
Svitlana Chemeris
We are now in Germany, my son has ataxia, maybe you know if it will be available to us?
Marcello Giglioni
Salve ,sono il papà di una ragazza di Roma, affetta da FA. L'annuncio dell'approvazione del farmaco, ha dato a me , e a mia moglie tanta gioia e speranza, sentimenti scomparsi in un attimo, quando abbiamo saputo il costo della terapia con Omaveloxolone, 350000€ ognmi anno.
Mi chiedo, chi potrà accedere alla terapia?
La nostra speranza, è che venga intanto approvato da EMA e AIFA, e sperare che il servizio nazionale sanitario italiano, se ne faccia carico.
Ma comunque non perdiamo la speranza.
Translation: Hi, I'm the father of a girl from Rome, affected by FA. The announcement of the approval of the drug gave me and my wife so much joy and hope, feelings that disappeared in an instant, when we learned the cost of therapy with Omaveloxolone, €350,000 each year.
I wonder, who will be able to access therapy?
Our hope is that it will be approved by EMA and AIFA in the meantime, and hope that the Italian national health service will take charge of it.
But let's not lose hope anyway.