FDA
An unnamed pharma filed a citizen petition in April seeking reforms to the way the FDA publicly releases rejection letters, alleging that the policy “contravenes decades of agency practice.”
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FDA’s rare disease decisions are strongest when the patient community has a voice in advisory committee decisions.
Regulators on both sides of the Atlantic are pushing for the withdrawal of the rare disease treatment that accounted for just 1% of Amgen’s 2025 revenue. Nevertheless, Amgen continues to defend the medicine, which was acquired in the $3.7 billion buyout of ChemoCentryx.
Saol Therapeutics is the latest biotech to resubmit for approval of a drug rejected under former FDA Commissioner Marty Makary, following REGENXBIO and Replimune.
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The FDAs enforcement action against Purolea Cosmetics Lab offers an early look at how the agency expects AI-generated work to fit within existing quality systems, with experts saying human oversight and quality controls remain essential.
Merck’s once-daily pill is the first oral PCSK9 inhibitor to hit the market for high cholesterol, beating AstraZeneca in the race to develop more accessible treatment options.
Biogen’s new data, presented at the Alzheimer’s Association International Conference, supports a tau-focused approach to the intractable neurodegenerative disease; psychedelics are back in the news with more positive data from Compass Pathways and final guidance from the FDA; and the ATTR-CM space got a major shakeup with the late-stage failure of AstraZeneca and Ionis’ antisense therapeutic.
An inspection of a facility making Elevar’s rivoceranib uncovered deficiencies, leading the FDA to reject a combination therapy that includes the drug. The rebuff is the third strike for the partners after the agency issued manufacturing-related denials for the other drug in the combo.
Celcuity’s first approved drug, Revtorpyk, is indicated for the second-line treatment of advanced HER2- breast cancer patients without PIK3CA mutations—a key use distinction that sets it apart from other PIK3/AKT blockers in the space, according to Jefferies.
The agency’s finalized recommendations for clinical testing of psychedelic compounds for mental health could pave the way for companies like Compass Pathways, whose psilocybin-based therapy for treatment-resistant depression could win approval as early as this year.
Through the proposal, the FDA could clear barriers to distributed manufacturing approaches intended to enhance emergency preparedness and supply chain resilience.
Leqembi Iqlik can now be given at home throughout the entire course of treatment, setting it apart from Eli Lilly’s Kisunla, the only other anti-amyloid Alzheimer’s disease therapy on the market.
The FDA last week confirmed that it would suspend the release of complete response letters pending the resolution of a citizen petition from an unnamed pharma company. Nevertheless, 14 new CRLs are now available on the agency’s portal.
The FDA greenlit 26 novel therapies in the first half of 2026, including four for cancer and six for orphan indications. Meanwhile, AstraZeneca and Johnson and Johnson took home a combined 11 of the agency’s 79 total approvals, including supplemental nods.