The MHRA will suspend the use, supply and sale of Avacopan Vifor (previously Tavneos) to new patients from 1 September 2026, after concluding that data integrity problems in the pivotal trial mean the study can no longer demonstrate efficacy. The drug, authorised on 6 May 2022 with rituximab or cyclophosphamide for severe, active GPA or MPA in adults, will be supplied only to existing patients during a six-month managed withdrawal, with the UK marketing authorisation to be revoked on 1 March 2027. Clinicians should not start new patients on the medicine and should review current patients for alternative treatments.
EMA has launched a voluntary data submission pilot that lets companies, method developers, contract research organisations and academic laboratories share data on New Approach Methodologies outside of a marketing authorisation application. Participants receive non-binding, non-decisional feedback from a group of non-clinical experts across the European medicines regulatory network, supporting regulatory acceptance of alternatives to animal testing under the 3Rs principles. Submissions run until the third quarter of 2027, after which EMA will publish a lessons-learned report.
China has approved satricabtagene autoleucel (satri-cel), the first CAR T-cell therapy for solid tumors, for patients with claudin 18.2-positive, HER2-negative gastric or gastro-esophageal junction cancer. The approval is based on a phase II trial showing improved progression-free and overall survival. Scientists welcome the decision but say more research is needed to improve CAR T-cell effectiveness against solid tumors.
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.
China's NMPA has granted conditional approval to Weiliduomab injection (brand name Weiketuo), making it the first domestically developed BCMA×CD3 bispecific antibody approved in China. Fosun Pharma's subsidiary Yaoyou Pharmaceutical, which in May 2026 licensed exclusive rights for clinical development and commercialization in mainland China, Hong Kong, Macau and Taiwan, holds the marketing authorization. The drug treats adults with relapsed or refractory multiple myeloma who have received at least three prior lines including a proteasome inhibitor, an immunomodulator and an anti-CD38 antibody. The approval rests on the Phase 2 GR1803002 trial, showing an ORR of 79.1% and an MRD negativity rate of 96.72% among patients achieving CR or sCR; further studies in RRMM are ongoing.
The FDA has approved Takeda's Mimrylo (rusfertide) for polycythemia vera, a nonmalignant rare blood cancer. Mimrylo brings a new mechanism of action to the indication and adds to CEO Julie Kim's record of regulatory wins since taking the role.
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.
China's NMPA approved Tong Yuan Kang's aumolertinib-deuterated EGFR-TKI TY-9591 (Cadaza, dextromethorphan aitinib mesylate tablets) on August 27 via priority review for first-line treatment of adults with locally advanced or metastatic NSCLC harboring EGFR exon 19 deletion or L858R mutation accompanied by CNS metastases. The company says structural optimization improves pharmacokinetics, reduces toxic metabolite formation, and showed positive efficacy and safety signals in brain metastasis and EGFR L858R populations.
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.
Hutchmed's Class 1 innovative drug, voragligratinib tartrate tablets (HMPL-453), was approved in China on August 28 for adults with advanced, metastatic or unresectable intrahepatic cholangiocarcinoma harbouring FGFR2 fusion or rearrangement who have received prior systemic therapy. The drug is the first domestically developed highly selective small-molecule FGFR1/2/3 inhibitor.
China's NMPA has approved Johnson & Johnson's Aiteya, the company's first targeted oral peptide drug, an IL-23 receptor antagonist in tablet form, for adults with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy, and for patients aged 12 and older weighing at least 40 kg. It is described as the first and only targeted oral peptide that precisely blocks the IL-23 receptor. The Center for Drug Evaluation granted it priority review in November 2025, and approval was announced on August 27, 2026.
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.