GSK’s Leucovorin
what a timeline - Sept. 1999 - $GSK withdraws Wellcovorin (leucovorin) bc it's not selling Sept. 2025 - GSK is told by FDA to apply for a new indication for leucovorin after an FDA review of lit. March 2026 - FDA approves leucovorin for treating the rare genetic condition Cerebral Folate Deficiency and says GSK is the sponsor April 2026 - FDA again withdraws GSK's NDA as company says it has no intention to make or market leucovorin https://t.co/0eW9BWNB7x
Cash‑pay Market for $950 Humira Doubtful vs Cheap Biosimilars
Is there really a cash-pay market for a $950/month brand-name Humira? (...especially w/two adalimumab biosimilars priced at less than $300/mo. already on TrumpRx, and many insured folks paying $0-5/month) https://t.co/taeQUY6Rve
FDA Backs Permanent Re‑authorization of Pediatric PRV Program
FDA calls to permanently re-authorize the rare pediatric disease PRV program in Friday's 2027 budget plan -- far cry from the FDA that complained to the GAO about PRVs as distracting from their public health priorities https://t.co/i79F7X1ECw
FDA Commissioner Seeks Private Partnerships for Collaboration and Regulation
Couple interesting meetings for FDA Commissioner Makary: met with Arnold Ventures on Mar. 3 about "collaboration opportunities" https://t.co/vEyhDu5xaW and with $MNPR on "establishing a regulatory mechanism" on Mar. 13 https://t.co/DHaD6IlvCS
Prasad’s CBER Saw Frequent Overrules, Unlike Rest of FDA
caveat here is that it was routine when Prasad ran CBER (at least 4 overrules in Prasad's 10 mo. tenure) but it didn't/doesn't still seem to be happening elsewhere in FDA
CBER Updates SOPs, Enables RMAT for Held Therapies
CBER changes its standard operating procedures to open the door to RMAT designations for therapies on clinical hold - https://t.co/bZmhga8xxC
FDA Urges IRB Overhaul to Match China's 60‑day Trials
FDA commissioner Makary at a CMS conference today in Baltimore calls for “big and different” IRB reforms to catch China's speedy trial starts - pre-IND phase can run 380 days in the US, he said, while China is pushing for...
Linking U.S. Medicare Payments to Global Benchmarks Matters
CMS' CMMI dir. Abe Sutton at quality conf. in Baltimore today: "tying what we pay to what other countries pay across Medicare Part B, Medicare Part D and Medicaid really matters."
Sen. Markey Admits He Doesn
Sen. Markey (D-Mass.) tells me outside the Capitol he doesn’t know who Vinay Prasad is and would need to be briefed by staff to respond to questions on the FDA
GSK Secures Approval Yet Refuses to Produce Drug
$GSK is ostensibly the "sponsor" that won the approval but is once again saying it won't produce the drug.
Prasad’s Missteps as CBER Chief Spark Criticism
On Prasad’s last few weeks as CBER chief: 1. It was absurdly inappropriate to bring a large group of reporters together and rail on a pending application “on background” 2. The walk back of the Moderna RTF was a bad...
FDA COO Butler Retires After One Year; Deputy Assumes Role
after just one year in the position, FDA COO Butler will retire, and his deputy will take over in April - https://t.co/9qmmIiuWX2
Immedica Secures Accelerated Approval, Plans PRV Sale
Immedica wins accelerated approval for once-rejected rare disease drug, plans to sell PRV - https://t.co/wU3AfqQzjw
FDA Issues Guidance on New Individualized Therapy Pathway
Building off the Baby KJ success story: FDA unveils long-awaited guidance on new pathway for individualized therapies - https://t.co/Uetp1cGFq2