The FDA has approved Eli Lilly's Mounjaro (tirzepatide) to reduce the risk of major adverse cardiovascular events (MACE-3), including cardiovascular death, nonfatal myocardial infarction or nonfatal stroke, in adults with type 2 diabetes at high risk of cardiovascular events. The approval rests on the phase 3 SURPASS-CVOT trial, which showed Mounjaro was noninferior to active comparator Trulicity in reducing MACE-3 risk.
The FDA issued an immediately-in-effect guidance on temporary policies for compounding certain starter parenteral nutrition drug products for neonates, after children's hospitals warned of a supply gap caused by two outsourcing facilities permanently shutting down. The guidance sets regulatory and enforcement priorities for state-licensed pharmacies, federal facilities and outsourcing facilities compounding under sections 503A and 503B. The FDA will keep monitoring supply needs and may take further action.
The FDA granted accelerated approval to AstraZeneca's Etcamah (camizestrant), an oral tablet, combined with a CDK4/6 inhibitor (abemaciclib, palbociclib or ribociclib) for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer whose tumors show an ESR1 mutation during aromatase inhibitor and CDK4/6 inhibitor therapy, detected by the FDA-authorized Guardant360 CDx companion diagnostic. In the pivotal trial, estimated median progression-free survival was 16 months versus 9.2 months for continuing an aromatase inhibitor plus a CDK4/6 inhibitor. The label carries a boxed warning for irregular heart rhythm, and confirmatory trials are required to verify clinical benefit.
On August 26, 2026, the FDA approved Revolution Medicines' Rasonque (daraxonrasib, formerly RMC-6236) for adults with metastatic pancreatic adenocarcinoma who have received at least one prior line of systemic therapy. It is the first FDA-approved targeted therapy aimed at the active-state RAS(ON) protein family. The RAS(ON) molecular glue approval marks one of the biggest clinical advances in pancreatic cancer in nearly a decade.
The FDA approved Ionis Pharmaceuticals' Zanvastro as the first disease-modifying therapy for Alexander disease, a rare and deadly neurological condition, for use in children and adults. In a pivotal trial, walking speed stayed stable in treated patients while the control group declined 33%, and the data hinted that treating young children may improve motor function. The drug was generally safe, with serious adverse events more common in the control group.
The FDA has approved Zanvastro (zilganersen) injection from Ionis Pharmaceuticals for Alexander disease in pediatric and adult patients, the first treatment for this rare neurological disorder and the first to target the abnormal GFAP protein buildup that drives it. Efficacy rested on a 49-patient randomized controlled study plus a 4-patient open-label substudy in infants under 2, with treated patients aged 5 and older showing better walking speed at 61 weeks. The antisense oligonucleotide is injected into the spinal canal every three months.
The FDA approved Revolution's RASONQUE (daraxonrasib), a once-daily oral tablet, for adults with metastatic pancreatic ductal adenocarcinoma who have received at least one prior systemic therapy or are not candidates for multi-drug combination systemic therapy. The approval on 26 August 2026 came about 6.5 months ahead of the original FDA target date. Daraxonrasib is the first approved RAS(ON) multi-selective inhibitor and the first RAS-targeted drug for metastatic PDAC that broadly inhibits multiple RAS mutations.
The FDA has approved Eli Lilly's tirzepatide to reduce the risk of cardiovascular events in adults with type 2 diabetes who are at high cardiovascular risk. Tirzepatide, a GIP/GLP-1 receptor agonist, is described as the first and currently only agent of its class shown to lower the risk of myocardial infarction, stroke or cardiovascular death in this population, while also providing glycemic control and weight management. The indication has not yet been approved in China.
The FDA approved Revolution Medicines' RASONQUE (daraxonrasib) on August 26 for metastatic pancreatic cancer. It is the first approved drug worldwide to directly target RAS. The approval marks a first for the RAS-targeting class in pancreatic cancer.
The FDA approved rusfertide, developed by Protagonist Therapeutics and to be sold by Takeda as Mimrylo, for polycythemia vera, a chronic blood cancer in which overproduction of red blood cells raises the risk of heart attacks, strokes and blood clots. Mimrylo is a weekly injection described as a new kind of treatment for the rare, slow-growing cancer.
JAMA reports that the FDA has approved the first oral PCSK9 inhibitor for lowering LDL cholesterol. It is the first oral formulation for this target; only the headline is usable from the original.
The FDA approved Takeda Pharmaceuticals' Mimrylo (rusfertide) for adults with polycythemia vera whose disease is not adequately controlled by existing therapies. It is the first approved treatment for the disorder that mimics hepcidin, limiting iron available for red blood cell production. In the phase 3 VERIFY trial of 293 adults, 76.9% on Mimrylo needed no phlebotomies over 32 weeks versus 32.9% on placebo; common adverse reactions were injection site reactions and anemia. Approval followed priority review.
The FDA has approved a label expansion for PharmaEssentia's Besremi, adding essential thrombocythemia to its approved indications. The original item is a feed summary and does not disclose approval details or the specific scope of the expansion.
Why it matters: PharmaEssentia's Besremi receives FDA label expansion approval, adding essential thrombocythemia as a newly approved treatment for the rare bone marrow disease.
Gilead's two-drug single-tablet regimen Bixlenvo (bictegravir 75 mg plus lenacapavir 50 mg) received FDA approval on August 27 for treating HIV-1 infection in adults, including patients previously treated with complex multi-tablet regimens. It is the smallest daily single-tablet regimen in HIV-1 and the first such regimen available for patients treated with complex multi-tablet regimens. CD4 cell counts stayed stable across both participant groups, with no treatment-emergent resistance observed.
The US FDA has approved Priovant Therapeutics' Lisraya (brepocitinib), a first-in-class TYK2/JAK1 inhibitor, for adult patients with dermatomyositis (DM). Dermatomyositis is a rare systemic autoimmune disease marked by progressive muscle weakness and widespread, painful, itchy skin lesions. The company calls the approval a major innovation in adult DM treatment after decades.
The US FDA has approved Brepocitinib, from Priovant Therapeutics, for the treatment of dermatomyositis, the company announced on August 27 local time. It is described as the first approved targeted therapy for dermatomyositis worldwide. Brepocitinib is a selective dual inhibitor of TYK2 and JAK1.
The FDA has granted early approval to Daraxonrasib, the first RAS(ON) inhibitor cleared anywhere in the world, for pancreatic cancer. The decision gives patients with KRAS-mutation-driven pancreatic cancer, a disease with a 5-year relative survival rate of just 3%, a targeted therapy option after years when no targeted drug was available. The approval moves pancreatic cancer treatment from a chemotherapy-dominated approach toward precision targeting.
The US FDA has approved Revolution Medicines' oral pan-RAS inhibitor Rasonque (daraxonrasib) for adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or are not suited to multi-drug combination systemic therapy. The approval follows overall survival benefit shown in a phase 3 trial. Rasonque works through a molecular-glue ternary complex strategy targeting RAS, a target once considered undruggable.
Eli Lilly has won FDA approval for Mounjaro to reduce the risk of heart attack or stroke in at-risk adults with type 2 diabetes. The decision adds a cardiovascular risk reduction use to the drug as Lilly competes with Novo Nordisk in diabetes and obesity. Lilly secured the approval on Friday.
The FDA has approved brepocitinib, brand name Lisraya, from Roivant and its subsidiary Priovant, for dermatomyositis, a rare autoimmune disease marked by muscle weakness and a painful skin rash. The once-daily pill affects a U.S. patient population of 40,000 to 70,000 and offers an alternative to high-dose steroids and other immune-suppressing drugs.
