Zach Brennan
@ZacharyBrennan
Senior Editor, Endpoints News - covering the FDA, Capitol Hill, and biotech/pharma - reach me at [email protected], Signal: zacharybrennan.20
Recapping $SRPT chaos: - undisclosed patient death in gene therapy trial - CEO says immediate disclosure wasnt material, stock craters - FDA requests $SRPT pull a diff. DMD therapy, revokes platform desig., and adds clinical hold on gene therapy - Sarepta PR says no to pull
Political or professional? Makary's overhaul of CDER, CBER may reshape leadership norms - endpoints.news/political-or-p…
This is really clever. Marry an oncolytic approach with autologous CAR-T. The cancer-specific virus sets up the @cancer cells with a synthetic antigen and adds a special recipe of chemokines and cytokines to beckon in the T cells, then you unleash the engineered CAR-T on it.…
FDA now saying up to 5 companies can win one of these new vouchers in the pilot
Companies aligned with U.S. national priorities can now submit statements of interest for participation in the Commissioner’s National Priority Voucher (CNPV) pilot program. Statements of interest are capped at 350 words! More details here: FDA.gov/CNPV
How are @VPrasadMDMPH and @DrMakaryFDA going to fix this perception and restore trust? They are the medical establishment and that's their job now. 100 more editorials on vaccine-myocarditis won't do the trick.
Looking more and more like CBER and CDER chiefs are now political positions
Quite the uproar over Prasad's past, especially since the CBER director role is not a political one (or is it?)
🚨 ALERT 🚨 VINAY PRASAD, THE LEFTIST SABOTEUR UNDERMINING TRUMP’S @US_FDA, MUST BE FIRED NOW! We have a crisis at the FDA! A wolf in sheep’s clothing is sabotaging President Trump’s bold “Make America Healthy Again” (MAHA) agenda! Dr. Vinay Prasad @VPrasadMDMPH, a…
New rare disease treatments not getting the same reception as they did under Marks
$REPL rejection follows the rejection of $CAPR and $RARE drugs, and the FDA's actions against $SRPT. CBER Director Vinay Prasad is setting a different tone than his predecessor.
Re:direction of new CDER chief w/ $SRPT, he wrote in April: "Both the ADUHELM initial approval and the ELEVIDYS approvals demonstrate that ignoring basic tenants of the use and interpretation of clinical trial data can be very damaging to public health." realclearpolicy.com/articles/2025/…
Looking more and more like CBER and CDER chiefs are now political positions
(reposted w/ link) Returned from Japan at midnight ➡️@endpts Post-Hoc Live about @Sarepta at 1:30 pm ET today. Join me, @ArmstrongDrew and @Jared_Whitlock to catch up on all that's happened around Sarepta and its DMD gene therapy last week endpoints.news/endpoints-live…
FDA names former industry exec Tidmarsh as next CDER director - endpoints.news/fda-names-form…
FDA taps former industry exec (Horizon, La Jolla Pharmaceutical, Threshold) GEORGE TIDMARSH as next CDER Director
Important points and as we’ve seen before (eg Makena etc) it’s not that easy or quick for the FDA to pull a drug
$srpt a contrarian thought: Q3 sales of Elevidys will handily beat expectations as families rush to get it fearing it won't be available. Also positive for stock on Monday: the overhang that Elevidys will be removed from market gone; company said it would ship drug.
Pretty remarkable that trust in $SRPT management is so low that an analyst asked if there have been any other undisclosed treatment-related deaths, just to make sure, after the same question was asked earlier in the call.
😬
$SRPT CEO Doug Ingram says the company did not believe the death in the limb girdle gene therapy trial of STP-9004 was material to disclose in Sarepta’s prior call because it was not related to Elevidys, FDA was aware and hadn’t changed its position, trial has been shut down …