-
Has anyone heard any updates on apitegromab?
Hey all. I’m just curious. Has anyone heard any updates on apitegromab?
Last I heard, Scholar Rock would apply for FDA approval in Q1 2025. I wonder if that’s still the case.
I’ve had my eye on this drug for a while. It seems fairly promising, though I know it hasn’t been tested on adults yet. (Correct me if I’m wrong on that.)
What are your thoughts about it?
-
6 Replies
-
My neurologist believes it will be more like 3rd quarter of this year.
-
Are you referring to the filing or the drug getting FDA-approved? I’m sure it will take a while once the application is put through.
-
-
Hey Alyssa –
Scholar Rock and CureSMA just did a webinar on this on December 18, 2024. I attended it. They are still filing for FDA approval in 1Q25 and hope for an approval before the end of this year. They also received FDA approval for an expedited review after that webinar, which means it could be approved more quickly.
Of note, this requires an infusion every 4 weeks.
-
Interesting! Did they say anything else worth noting?
I’m curious how this drug will impact adults, if at all. My neurologist explained how myostatin decreases as you age, and this drug targets the myostatin on the muscle. So, there is some ambiguity there. I guess time will tell.
-
I think it’s worth noting that Spinraza clinical trials were not on adults and the FDA’s approval at the end of 2016 covered all ages. So, since it works in conjunction with Spinraza and Evrysdi, it seems to make sense that it would be approved for all age groups. Making sense may not be enough though.
The concern I have is for it to be effective, it would need functioning motor neurons. We are born with the number of motor neurons we lose them with SMA, in different locations and at different rates. In other words, I may lose motor neurons more quickly in my shoulders and another patient may lose them more quickly in their quads. So, it seems to follow that the myostatin inhibitor would be more effective in different ways for patients, in the areas with more functioning motor neurons, allowing for muscle growth to help compensate in areas of muscle loss and help strengthen areas less affected.
I had lunch with several folks in our community yesterday. One of them said they know of a 7 year old who was in the Scholar Rock clinical trials who has had significant benefit in his upper body strength and less so in the lower body. He’s a type 2, so not sure if that makes sense, but it’s certainly anecdotal.
Ultimately, I’m thankful for the ongoing efforts of those working to help us and improve our quality of life. There are studies underway and something called anthrobots, which generate new motor neurons, have been created in the lab. How that translates to animal and then human trials is TBD, but in combination with other treatments seems to point to significant progress. Plus, CRISPR and gene editing is another light at the end of the tunnel. There’s a lot of hope now and more on the horizon!
-
-
-
From what I’ve seen recently, Scholar Rock is still planning to apply for FDA approval in early 2025, so it seems like things are on track. I agree—it does look really promising, especially for what it’s targeting. And you’re right, I don’t think they’ve done trials on adults yet, but hopefully, we’ll hear more about that soon.
Log in to reply.