FDA flips on uniQure
This was the topical news of today. It will probably continue to create a lot of discourse, no less because of what condition it is. There’s a sort of common shared reverence for how much people care about something in medicine based on what exists right now today. If its health care impact on people is tragic, severe, and without intervention, there’s a lot of grief and discussions around them are very serious.
Compare this to something that’s common and treated with a lot of different things— that typically isn’t something that’s a source of discussion. I’m definitely one of the only people on earth running around upset by the fact that spinosad has been known to be retained in the stratum corneum when topically applied to mammals ever since 1994. But it remains completely undeveloped for its use in scabies outside of America. It is a widely commercialized and heavily studied pesticide that can kill just about any bug that’s got a CNS. Natroba to boot has an unlabeled medicinal component inside of it, making registration of it quite a bit harder should a regulator simply ask why their placebo has a massive clearance rate of scabies mites.
Asset utilization like this for diseases where there’s decades of history of people crying about their topicals not working is an obscenity that doesn’t particularly mesh well with the modern research infrastructure that exists right now. Like people have gotten really, really, really smart with a lot of stuff. About every single person on earth that has ever studied infectious diseases of the skin would have known post 1994 and onwards that spinosad was an extremely good candidate to study for the condition. The call on that doesn’t get more straightforward. The medical component is fully retained at the disease site. Which is probably the only reason any good news came out of AMT-130— the drug was infused directly into the brain’s parenchyma.
Anyways, the point I’m making is there’s a lot of different treatments for scabies that have been made available. And you will probably only see these complaints come out of me. And there’s absolutely nothing at all for Huntington’s disease! Which you will see many, many people talk about in the coming days. Marty Makary has seemingly dodged a media appearance today, so he probably just doesn’t have it in him to smile at a camera right now. Can’t say I blame him, today’s news sucked. A lot.
It’s an interesting issue when regulator alignment diverges years into clinical development because this stuff ripples around the entire landscape instantly. This makes sense because everyone’s priors about trusting communication from the agency were just updated. But in general, the non-public stuff is where the losses never make themselves fully apparent to anyone but are the largest. There are projects that you’ll never even know about that don’t get started now because of the perceived risk in developing them has just changed a lot.
I don’t feel totally lost on AMT-130 here. But a huge number of HD patients probably do after about a month of celebrations. The bright side is all of the problems at uniQure or the opinions people have of their lead IND candidate will probably get better with time. Because you really don’t ever want to overstate what’s being sold to a person when it comes to medicine like this, I can’t be dissuaded by needing to continually follow its development. Because of this and because I’m okay with using a matched external control as a comparison as they did, every year that goes by I’ll be just get a little more encouraged by what the product can do for people. Other people probably will as well. In spite of neurologists who are not being blinded that may be biased when performing clinical assessment, the progression in symptoms you begin to expect at years 4, 5, 6 & 7 in early manifest HD is substantial. Some people are already at deaths door by year 10. It’s inside this long follow-up period where you get these suspiciously healthy-looking people where it gets easier to tease out an effect. So long as that trend holds together, the investigational products development will persist until it’s made available everywhere.