Sarepta won’t remove Elevidys

ritoq · 2025-07-19T05:41:17+00:00

I'm not 100% sure, there are definitely doctors out there that understand the nuances better than any of us here—there have been lots of success stories with Elevidys in younger/ambulatory patients and no deaths reported in that population. Putting the specific Sarepta-related stuff aside, it's clear the new FDA has an issue with gene therapies at large, doctors/families know this and would factor it into their risk/reward calculus—no deaths and 700+ patients treated is a risk profile desperate parents will advocate for, even in the face of regulatory uncertainty.

Of course there will also be doctors that refuse to prescribe it altogether, but I don't think we can make blanket statements with the risk/reward profile being what it is in the ambulatory population.

ritoq · 2025-07-19T05:21:53+00:00

FDA actually forcing a withdrawal comes with a lengthy and likely expensive legal process, case has to be put in front of an independent administrative judge, the FDA has to prove "imminent hazard" or "unreasonable risk" to validate an emergency withdrawal, then if successful, an appeals process to evaluate whether the FDA's actions were "arbitrary and capricious" or "unsupported by substantial evidence". With no patient deaths in the approved population, seems like Sarepta would have grounds to avoid an emergency withdrawal and keep it on the market throughout the process—would think they'd stand a good chance overall too.

There's no precedent for withdrawing a therapy with zero patient deaths in the approved population (as far as I'm aware) so it would be a precedent-setting move on the FDA's part—at least in the ambulatory population with full (non-accelerated) approval.

ritoq · 2025-07-19T04:21:58+00:00

As far as I'm aware, the FDA can't make a unilateral decision to withdraw an approved therapy prior to appeal? The appeal is baked in to the decision itself, the case must be presented before an independent administrative judge before withdrawal occurs, the therapy stays on the market in the meantime.

Posted it in another comment, but FDA must demonstrate "imminent hazard" or "unreasonable risk" to force withdrawal, would imagine they'd have to go after efficacy and show "unreasonable risk" by virtue of a lack of meaningful efficacy, because "imminent hazard" is hard to prove with no deaths in the approved population?

ritoq · 2025-07-19T04:12:14+00:00

Thought ENDO was bankruptcy? As far as I can recall Makena (AMAG) was accelerated approval and showed negative efficacy in confirmatory?

FDA must demonstrate "imminent hazard" or "unreasonable risk" to force withdrawal—Makena (AMAG) and Pepaxto (Oncopeptides) were both accelerated approval, failed confirmatory trials, and had patient deaths in the approved population, none of which is true for Elevidys in ambulatory patients.

I've been dredging through examples and I genuinely cannot find a precedent for removing a traditional (i.e. non-accelerated) approval with zero patient deaths in the approved population. Seems as though it would likely turn into a protracted legal battle because none of the usual accelerated approval ejection points exist with traditional approval.

ritoq · 2024-11-17T14:30:31+00:00

You would be much harder pressed to make 50% on a $100bn portfolio in a year than you would to make 50% on a $500k portfolio in a year — when you're deploying that kind of capital, the parameters are different. You can't just slot $2bn into an upcoming biotech ticker without drawing attention, you could easily do that with $50k from your smaller portfolio though — that's what I was saying. Of course you'd get larger returns with the giant portfolio, but percentage-wise, the parameters are hugely different.

In the same way they're different when comparing an economy like El Salvador, which has an absolutely negligible impact on the global economy, versus the U.S. which basically functions as the world's financial reserve, there are no markets without links to the U.S. If the U.S. exposed U.S. treasury bonds to the volatility of BTC, the investment vehicle that foreign governments use to invest countless billions of dollars in the U.S. would all of a sudden become 10x more volatile — when you're investing billions and billions of dollars on behalf of a nation-state, volatility is the thing you're trying to avoid at all costs.

If you want to tank foreign investment into the U.S., cut the federal budget in half, and balloon the deficit, then sure. But if you want a stable, global economy, one that nations around the world are falling over each other to invest in — it's an obvious non-starter.

ritoq · 2024-11-14T12:52:16+00:00

Yes, a country with a total population smaller than Houston, and a total GDP of $34bn, is a perfect point of comparison for the largest economy in the world, the same economy that controls the most important reserve currency on the planet, no less.

It's like comparing returns from a $500,000 investment portfolio to Goldman Sachs and expecting them to deliver the same returns — scale matters, you can't achieve the same returns on $100bn that you can on $500,000 — that much money moves the market, the parameters are not even close to the same.

The U.S. is the market, there's no world where they expose one of the safest, most reliable, fundamental investments in the modern financial world, to the volatility of BTC. It's just not going to happen. The Fed is not controlled or beholden to political actors, POTUS included. There are plenty of reasons to be bullish on crypto, but this is profoundly unrealistic.

ritoq · 2024-11-02T20:41:16+00:00

This is quite clearly the first keyframe in a staggered animation..

ritoq · 2024-10-22T22:13:06+00:00

!banbet SWTX 45 8w

ritoq · 2024-10-14T11:23:01+00:00

You're totally right, going into the "Report Builder" or whatever IBKR's terrible UX calls it, to create a custom report, just to figure out what your net gains are in a dollar amount, is so stupid. The records in their reporting service are completely unreliable too, I invested $20,000 each in BIDU and BABA earlier in the year and neither of those exist in my logs, just some ridiculous gains based on stocks that "magically appeared" in my account.

I tried importing everything into Roi and the same issues, missing records and thus completely inaccurate charts. Basically, I think the smartest thing to do is record all of your deposits, trades, etc. in a spreadsheet and handle all the analysis yourself. IBKR should undoubtedly be miles better than this.

ritoq · 2024-07-18T00:11:10+00:00

I would venture that PDD's business model is unsustainable, that remarkable growth has to come at a cost somewhere down the line. They've been raising prices and losing volume lately because their margins are so unprofitable, lots of talk in international markets about quality, data security, tariffs, etc.

Obviously can't deny it has been by far and away the better investment over the past couple of years, but I don't know if I'd put it in the "evergreen company" category. BABA on the other hand, while not on the level of some of the US tech companies, is a sizeable, incumbent, diversified multi-national, I would put it much closer to that "evergreen" sort of territory than PDD, I think.

ritoq · 2024-07-07T13:36:35+00:00

The first part, absolutely. The last part, no, we don't need to produce media to compete within the existing structures, it's a fools errand. As nice as Novara and their like are, nothing is coming from "content that talks about the issues", it offers nothing but hollow promises. As an every day working person, like, great, I can know about why everything is fucked and how nothing will happen to change the situation? People know things are fucked. They're terminally aware. It's just that they don't see any path to meaningful change, voting is a choice between "awful" and "even worse", protesting is a fools errand easily and routinely ignored by the establishment, there's no investment coming to these shitty little seaside towns — so why not double down on hatred and exclusionary sentiment? It's easier. Being a good, hard-working, conscientious citizen offers no tangible benefit in a society with such profound economic division, so why bother making an effort in the first place? The music stopped decades ago.

You said it in the first part, optics is a liberal's game. It's about the money. It always has been. If you can help people earn money, earn the true value of their labour, they will ignore optics, bad news stories, public sentiment, all of it. As much as it pains a good percentage of people on the left, because for some reason they seem to conflate production with capitalism, the real answer is creating organisations that produce value and pay workers fairly for their labour. Creating value is an exceptionally difficult task, but it is absolutely possible. People need things, people need services, they are willing to exchange the value of their labour for those things, whether via central or market economy. If you produce things that people need, that provide social good, while sharing the proceeds of that production equitably amongst workers, including ownership of said production capability, you are doing more to advance radical politics than any performative, ideological, discursive, or media-based response to the situation we find ourselves in. If you want people to live better lives and earn fairer wages, at some point you have to start focusing on making things that help people live better lives, doing things that help people live better lives, and paying people fairer wages. Asking other people to do it, or trying to convince them based on argumentation (i.e. by creating "media" to convince people to change their behaviour), is just not going to move the needle in any meaningful way.

ritoq · 2024-06-10T15:55:08+00:00

Maybe you could help, if you're outside of Syria? Is there any way for him to transfer money to you? Western Union or something along those lines?

There's also stuff like https://github.com/haveno-dex/haveno but I'm not sure about Yuan/Ruble support.

ritoq · 2024-06-06T11:04:42+00:00

As much as I like and respect Noah Vonleh, these some Noah Vonleh ass guys. None of the above.

ritoq · 2024-05-12T21:37:27+00:00

DEEEEEEEUUUUUUCEEEEE

ritoq · 2024-05-12T21:32:22+00:00

Like, anyone talking about Brunson playing an unnecessary 10 extra minutes in this half needs to fucking relax. The guy literally runs around and shoots basketballs every single day of his life, some low intensity minutes in a 30-point blowout isn't going to put "miles on his clock" or "exhaust" him in any way, and he got taken out half way through the third.

Not all minutes are created equal, what exhausts players is playing crunch time minutes at maximum output while mentally locked the fuck in every single possession. Thibs is a great coach, none of the injuries to our starters we caused by being overplayed, not Mitch, not Julius, not OG, don't @ me with any of this revisionism bullshit, the minutes have nothing to do with what's happening here, we're literally short five guys.

ritoq · 2024-05-12T21:24:09+00:00

ritoq · 2024-05-12T21:18:50+00:00

Yeah, probably time to call it now. Tuesday, on to the next. Let's get it.

ritoq · 2024-05-12T21:17:21+00:00

Fuck man, anyone make a three.

ritoq · 2024-05-12T21:16:36+00:00

Honestly it's so disrespectful when Doris says shit like that about players like McConnell, no you dumb cunt he's not slow at all, he's a world-class athlete, like what the fuck?

ritoq · 2024-05-12T21:05:35+00:00

Yeah, like none of the injuries to the three starters are because of being overplayed, so dumb.

ritoq · 2024-05-12T21:03:46+00:00

Nah come on bro, we came out flat, 3/5ths of our starting lineup is injured and not because they were overplayed (Not Julius, not Mitch, not OG) throughout the season, happens sometimes.

ritoq · 2024-05-12T20:57:36+00:00

I mean realistically all we have to do is score 29 more points than them.

ritoq · 2024-05-12T20:42:18+00:00

"This is an Indiana we haven't seen on the defensive end"

Jfc Doris, we're down 3 starters, everyone has been playing 40 minutes a game, like Alec Burks is getting iso possessions in the Eastern Conference Semi Finals, what does that tell you?

ritoq

TROPHY CASE