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🚨BREAKING: Keir Starmer has told friends that he intends to stand down as Prime Minister and set out an orderly timetable for his departure
[@DailyMail]
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@Lucy3370
Looking to find hidden value in biotech's.
Katılım Ağustos 2011
197 Takip Edilen2.5K Takipçiler
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Not @stuartbuck1 deleting his post after 6 mins. He asked who is behind posts on X critical of the Arnold Ventures cartel at the FDA after cleaning house🧹🚶🏽♂️Makary, Prasad, Hoeg & Szarama, also criticizing “high costs of unproven drugs”
THERE IS NO ALGORITHM FOR A HUMAN LIFE‼️
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Dying of ALS, I'm a bit skeptical about @houmanhemmati's posts.
@SecKennedy @realDonaldTrump, your mandate to deliver treatments/cures to the American people was ignored by @MartyMakary, Despite him issuing MANY public statements similar to the post below.
I wrote the following Op-Ed in @NewsweekOpinion that outlines a few of the consequences of having FDA leaders like Marty, who say 1 thing in their confirmation hearing but do the exact opposite once they're FDA Commissioner. t.co/WMoJOvfpWz
The post below by @houmanhemmati is the mindset that is required for the future FDA Commissioner. I hope you will not forget it if you get the job, Houman.
@BillCassidy @RepGuthrie @DrOz @DrOzCMS @RepGusBilirakis @SenRonJohnson @ChuckGrassley @SecKennedy @RobertKennedyJr @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepRobinKelly @RepBarragan @RepAngieCraig @RepKimSchrier @RepLoriTrahan @SenatorBaldwin @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis
Houman David Hemmati, MD, PhD@houmanhemmati
🇺🇸 🇨🇳 I was on @foxnewsnight tonight discussing something that should concern every American in the context of the U.S.-China summit. For decades, the United States has led the world in biotechnology. Cell & gene therapies & advanced biologics — the medicines of the future — are finally delivering real hope to patients with diseases that were once untreatable or incurable. These are not incremental improvements. They are transformative. But here’s the reality we can no longer ignore: roughly half of the global investment capital in advanced biotechnology is now flowing to China. This isn’t just about money or market share. It’s about who will control the next generation of life-saving treatments. When capital, talent, & manufacturing shift overseas, American patients lose access, American innovation slows, and our strategic position in one of the most important industries of the 21st century erodes. China is not playing by the same rules. They are moving aggressively to dominate cell and gene therapy, while we continue to tie ourselves in regulatory knots and send mixed signals to the companies trying to develop these therapies here at home. We cannot afford to watch this happen. The patients who are waiting — children with rare genetic diseases, adults facing terminal conditions, families desperate for options — don’t care about bureaucracy or politics. They care about whether these treatments reach them in time. The FDA has an essential role to play in this moment. We must modernize our approach so that we can accelerate safe and effective innovation here in America, while maintaining the rigorous standards that have made our system the global gold standard. If we fail to do this, we will not only lose our leadership — we will fail the very patients we are supposed to serve. In my career, I’ve seen what’s possible when regulation keeps pace with science, and I’ve seen what happens when it doesn’t. The video of my appearance is below 👇 . I’d love to hear your thoughts.
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FDA must have the trust of the American people.
Trust must be earned through transparency.
The best form of transparency is to have everything out in the open and permit all stakeholders to view AND participate.
Decisions cannot be made in a black box.
This benefits review divisions by giving them “cover” for unpopular but scientifically/ethically sound decisions.
It benefits patients by letting them see the data and trust the process.
Yes there’s a cost. But it’s a no brainer to have AdComs.
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From a staffing & messaging perspective, it's also interesting to me how the narratives played out this week. Makary claiming to resign over the vaping situation & White House donors believing that he & the cronies he hired were fired by the White House to protect rare disease patients....
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$qure $repl $srpt this is very infuriating. How many patients are harmed by delays in approval for rare diseases??? Utmost urgency must be given to going back to original @US_FDA guide & move forward from there
Pearl Freier@PearlF
On the FDA, it had started to come out over the last few weeks that several political appointees that were in new leadership positions at FDA had a history of funding from Arnold family entities incl Makary. And Q's were raised re: recent drug rejections & the overruling of scientific staff which wasn't a regular occurrence at FDA until this group of political appointees came in. Some concerns were based on the history of how Arnold entities & affiliates like iCER appear to discriminate against a number of patient populations incl rare disease patients
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$qure $clpt
Really good article, highly recommend
“Makary’s tenure at FDA is Exhibit A. From vaccines to medications for rare diseases, he and his deputy Vinay Prasad — who left the agency, returned, and then left again — overruled staff scientists, based on the repeated assertion that they were bringing back “gold standard science and common sense.” They announced policy by press release or journal commentary rather than through official, vetted documents, which could have addressed key questions. Instead of convening advisory committees, Makary brought together rubber stamp panels to endorse the administration’s agenda.”
“But the erosion of the integrity of the agency’s decision-making has been unmistakable. By the end of his tenure, it was hard to fault those who thought all of the agency’s decisions were Makary’s alone — leaving multiple companies and advocacy groups furious at him for every disappointment.”
@RepAuchincloss
@SenRonJohnson @SenRickScott @SenGillibrand @WhiteHouse @FrankLuntz @SusieWiles @BeckyQuick @HDSA @HDBuzzFeed @Help4HDI @houmanhemmati
Rick Berke@rickberke
.@drJoshS weighs in on how Marty Makary “misunderstood” the FDA. statnews.com/2026/05/16/mar…
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This is a huge statement from the JPM 26 conference from the new acting director of CBER. It bodes well for @uniQure_NV $QURE AMT130. How can they move the goalposts and expect any company to spend the money required to bring treatments to market as they did with AMT130?
Key@KennyCh74009091
$CLPT $QURE Karim Mikhail is the new CBER! After taking a deeper dive I think this is great News! hoganlovells.com/en/publication… Karim Mikhail’s comments at JPM were actually very interesting for rare disease and gene therapy back in Jan. He basically hinted that FDA is becoming more flexible and “not thinking in black and white” anymore. He talked about: • single pivotal study pathways • flexibility for rare disease/cell & gene therapy • accelerating approvals • reducing regulatory friction Obviously he never mentioned $QURE specifically, but if you read between the lines it’s hard not to think about Huntington disease and AMT-130. You can’t apply traditional large trial logic to terminal rare diseases with tiny patient populations and years-long progression timelines. Feels like he realized there has to be some middle ground between safety and letting people die while waiting 10+ years.
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While we relax over the weekend celebrating the news of the FDA firings, I thought I would share my recap of the @uniQure_NV $QURE AMT130 study details again.
By the end of March 2026, 9 patients in hi dose cohort reached at least 4 years. That will go to 12 in early July. I mention this because in past years, the company reported data in late June/early July using the March cutoff of 9 patients. Could they use these 9 patients when meeting with FDA in upcoming Type B meeting as added support to go back to prior alignment and allow BLA filing ASAP? It is almost criminal not to.
We also know that in Sept they released 3-year data on 12 patients and an additional 3 patients did not yet hit the 3-year mark as of that July 2025 cutoff. What if they hit that by now? They can add data from those additional 3 patients in the 3-year data to further support the 3-year efficacy.
No later than the end of this year, there will be a total of 20 patients in the hi dose who hit at least 3 years with others hitting up to 5 years. The # hitting 5 years will be very small but this shows the maturity of the data. This is the only treatment for HD to demonstrate any efficacy in all the trials over the years.
With a total of 32 patients who have been treated in the hi dose to date, why would new FDA leadership need a phase 3 trial before allowing BLA filing? They should allow filing and if the data package supports AA, approve AMT130 with requirements to continue following all patients in original 4 cohorts plus data on a certain # of patients after approval to grant full approval if they feel it is necessary. Other gene therapy treatments have been granted full approval on fewer than 32 patients.
Why delay access to the ONLY disease modifying treatment ever? This is the very definition of a HUGE UNMET NEED and US citizens deserve access before more brain cells die prematurely that will never come back.
Hopefully new FDA leadership recognizes the harm done over the past 8 months and reverses course ASAP. Congress, please don't allow the FDA to continue this horror show. Force them to allow uniQure to file BLA and give the data a fair review for AA for the community.
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$CLPT $QURE Karim Mikhail is the new CBER! After taking a deeper dive I think this is great News!
hoganlovells.com/en/publication…
Karim Mikhail’s comments at JPM were actually very interesting for rare disease and gene therapy back in Jan.
He basically hinted that FDA is becoming more flexible and “not thinking in black and white” anymore. He talked about:
• single pivotal study pathways
• flexibility for rare disease/cell & gene therapy
• accelerating approvals
• reducing regulatory friction
Obviously he never mentioned $QURE specifically, but if you read between the lines it’s hard not to think about Huntington disease and AMT-130.
You can’t apply traditional large trial logic to terminal rare diseases with tiny patient populations and years-long progression timelines.
Feels like he realized there has to be some middle ground between safety and letting people die while waiting 10+ years.
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in addition to makary and prasad, without waiting for new leadership to decide, the administration went ahead and showed the rest of the leadership team associated with arnold ventures the door. this sends a strong message this administration is serious about innovation, cares about rare disease patients, and won't let american biotech lose its leadership position in the world.
Drew Armstrong@ArmstrongDrew
🚨Friday night shakeup at FDA: * Acting CDER chief Tracy Beth Høeg fired, replaced by Michael Davis * Acting CBER chief Katherine Szarama out, replaced by Karim Mikhael * COS Trafficant out, others too Full story here from me and @maxonwifi: endpoints.news/cder-chief-hoe…
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Tracy Beth Hoeg giving her exit interview to an antivaccine blogger is so very much on brand.
She was not qualified for the job; but I don’t know if the FDA will find qualified people for CBER and CDER after the mess created.
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Some more details on FDA changes:
Michael Davis, will take over as acting director of CDER.
Karim Mikhail, who joined the FDA in 2025 as a senior adviser in the office of the commissioner, will be the acting director of the Center for Biologics Evaluation and Research.
Mikhail replaces Katherine Szarama, who is also leaving the agency. He was previously CEO of the pharmaceutical company Amarin.
Lowell Zeta, another official in the commissioner’s office, will take the chief of staff role from Jim Traficant. Zeta worked as a senior adviser at the FDA in the first Trump administration.
TLDR: They are really cleaning house.
statnews.com/2026/05/15/fda…
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I picked a hell of a day to take a sick day…
That Diamantas/HHS opted to sweep out Makary’s prior leadership staff was expected. I wasn’t expecting Hoeg or Szarama to be pushed out/aside quite so quickly, though swapping for other acting directors makes sense.
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We wrote letters, met management teams, spoke with advocates and lobbyists, exposed Arnold Ventures, and went deep down the rabbit hole on this one.
Result:
Makary out.
Hoeg out.
Prasad out.
Szarama out.
All discussed on my last podcast with @twiii_podcast before it happened.
Now the important part: I fully endorse @houmanhemmati for FDA Commissioner.
We need evidence-based leadership that actually understands next-generation biologics, cell & gene therapy, and advanced medicines if America wants to stay ahead of China.
Most importantly, rare disease families deserve regulators who understand urgency, scientific nuance, and the reality that doing nothing is also a decision.
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Katherine Szarama, acting director of the biologics and vaccines center, is out as well. Karim Mikhail will replace her. Lowell Zeta will replace Jim Traficant as chief of staff. All according to an email sent to staff and obtained by STAT. Story will be updated imminently:
Lizzy Lawrence@LizzyLaw_
NEW: Acting director of the drug center, Tracy Beth Høeg, is leaving the FDA, just days after Commissioner Marty Makary resigned from the agency statnews.com/2026/05/15/fda…
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UPDATE: Katherine Szarama, acting CBER chief, also out.
FDA drug center head Tracy Beth Høeg leaves as agency faces leadership vacuum statnews.com/2026/05/15/fda… via @statnews
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🚨EXCLUSIVE: FDA official Tracy Høeg fired after refusing to resign
@TracyBethHoeg says she first learned about rumours of her departure through media reports before being told by FDA officials to resign or be terminated.
LINK below 👇👇
@MartyMakary @VPrasadMDMPH @SecKennedy
@AlecGaffney @LizzyLaw_ @maxonwifi @TheChiefNerd
@newstart_2024
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$qure I also have to think all these Arnold Ventures purges increase the odds of a positive FDA type B meeting coming in the next weeks
R@Lucy3370
Looks like the @US_FDA is purging the Arnold Ventures taint. 👇👇👇👇 $srpt $qure $srpt
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