AI diagnostics, brain-computer interfaces, gene editing, and the future of medicine.
58 articles
The Danish drugmaker now holds a beachhead in the youngest patients yet studied for a weight-loss medicine, a stretch of the market where rival developers are still gathering evidence.
A positive overall survival readout against standard chemotherapy would make this the first approved drug of its class, and it hands GSK a de-risked global development program.
A therapy aimed at the mutation that lets hormone-driven tumors escape treatment now ships with the diagnostic needed to spot it in time.
Drugmakers can no longer lean on overseas study sites that US reviewers cannot inspect, a stance that tightens the evidence bar after unsettling gene-therapy cases abroad.
The antisense drug, cleared 19 days ahead of schedule, stabilized gait in a Phase 3 trial of 49 patients and gives a fatal pediatric disorder its first therapy beyond supportive care.
The filing lands after the FDA reversed an earlier rejection in June and reopened an accelerated-approval route, and the company has promised four-year clinical data by the end of the third quarter.
A repurposed oncology medicine has produced a marked reduction in the hemorrhage that occurs inside injured cardiac muscle after a major attack, a benefit never shown before. Because the compound is already approved and its tolerability is known, the next, larger study may be easier to mount than for an experimental molecule.
The decision, announced on August 31 and reported from September 1, gives patients a two-week interferon option that beat anagrelide on durable response rates in a Phase 3 trial, roughly 30 years after the last therapy reached the market.
The FDA has approved PharmaEssentia's BESREMi for essential thrombocythemia, giving the rare blood-cancer patients their first new treatment option in about three decades. Trial data showed 43% of patients on the drug achieved durable platelet control, versus 6% on an older standard therapy.
The FDA on August 28 approved Eli Lilly's Mounjaro to lower the risk of major adverse cardiovascular events in adults with type 2 diabetes, making it the first GIP/GLP-1 receptor agonist with the indication, based on the 13,000-patient SURPASS-CVOT trial.
The FDA on August 27 approved daraxonrasib (Rasonque, Revolution Medicines) for previously treated metastatic pancreatic adenocarcinoma, making it the first RAS-targeted drug that does not require a companion mutation test.
The FDA approved Gilead's Bixlenvo, a once-daily bictegravir and lenacapavir tablet, for virologically suppressed adults unable to take existing single-tablet regimens, capping a late-August wave of approvals.
FDA approved Jazz Pharmaceuticals' Ziihera plus Tislelizumab and chemotherapy on August 25 for first-line HER2-positive gastroesophageal adenocarcinoma, citing median OS of 26.4 vs 19.2 months and triggering a $250 million milestone to Zymeworks.
Johnson & Johnson's nipocalimab-aahu becomes the first FDA-approved treatment for wAIHA in adults and pediatric patients 12 and older, after a Phase 2/3 trial showed roughly 3x the durable hemoglobin response versus placebo at 24 weeks.
The FDA approved three blockbuster drugs in August 2026: Bristol Myers Squibb's Zenbexus for multiple myeloma on August 14, Takeda's Orzeyful for narcolepsy type 1 on August 5, and Regeneron's Pasatru for fibrodysplasia ossificans progressiva on August 20. The approvals span oncology, neuroscience and rare disease.
Merck and Moderna said on August 19 that intismeran autogene, a personalized mRNA neoantigen therapy, met both primary and key secondary endpoints in the Phase 3 INTerpath-001 trial in resected melanoma. Moderna's market value added $45 billion in a single day.
The FDA granted accelerated approval on August 19 to Ultragenyx's GENGLYCOS (pariglasgene brecaparvovec) for adult and pediatric patients with glycogen storage disease type Ia. The approval is the company's first gene therapy and fifth FDA approval overall, and includes a Priority Review Voucher.
Genentech on August 21 announced a $750 million pharmaceutical fill-finish facility in North Carolina, expected to create 700 jobs by 2029. On the same day, Australia's OncoSil Medical received FDA approval for a brachytherapy device targeting unresectable pancreatic cancer.
The FDA on August 19 approved Regeneron's Pasatru (garetosmab-grts), a VelocImmune-derived monoclonal antibody that blocks Activin A, for adults with fibrodysplasia ossificans progressiva. The approval is the first targeted therapy for FOP and is backed by Priority Review Voucher issuance.
Genglycos, a one-time AAV gene therapy for GSD1a patients aged 8 and older, won accelerated approval on August 19. Ultragenyx set a $2.7M wholesale acquisition cost and plans to sell the accompanying priority review voucher — three vouchers sold for $180M–$200M this year.
The FDA on August 18 granted accelerated approval to iberdomide (now branded Zenbexus) in combination with daratumumab and hyaluronidase-fihj and dexamethasone for adults with relapsed or refractory multiple myeloma. Bristol Myers Squibb reported a 41 percent MRD-negative complete-response rate, and BMS's older myeloma drugs Pomalyst and Revlimid are already down 71 percent and 49 percent year-on-year.