AI diagnostics, brain-computer interfaces, gene editing, and the future of medicine.
105 articles
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.
Eli Lilly on August 12 filed six lawsuits against U.S. firms it accuses of illegally selling black-market versions of its experimental obesity drug retatrutide. A day later, Novo Nordisk CEO Mike Doustdar said investors are underestimating demand for differentiated obesity drugs — pushing back against the market-share narrative that has driven Lilly's stock for two quarters.
The FDA granted accelerated approval on August 13 to Bristol Myers Squibb's iberdomide, sold as Zenbexus — the first cereblon E3 ligase modulation therapy for multiple myeloma and the first new drug cleared on an MRD endpoint.
The FDA on August 12, 2026 granted Breakthrough Device Designation to Freenome's SimpleScreen Lung, an AI/ML-powered multiomic blood test for adults 50 to 80 at high risk for lung cancer — the second major FDA milestone for the company in two months after its colorectal test was approved in late July.
PhenoNet announced August 13 that the FDA issued a May Proceed notice for its Phase 3 trial of PHENOGENE-1A, an inhaled formulation of cromolyn for early Alzheimer's disease, in both APOE4 and non-APOE4 patients.
Taiho and Cullinan Therapeutics' zipalertinib plus chemotherapy met the primary endpoint of the REZILIENT3 Phase 3 trial in first-line non-small cell lung cancer, teeing up a potential regulatory filing.
A single dose of DT120 cut generalized anxiety symptoms by 5.4 placebo-adjusted points (p<0.0001) in the 214-patient Voyage study — its second late-stage win this summer — with Jefferies modeling at least $1.5 billion in peak GAD sales.
The anti-alpha-synuclein active immunotherapy for early Parkinson's disease also cleared an IND allowing its Phase 2 VacSYn trial to expand to US sites, with full 100-week data expected in the second half of 2026.
Tenax Therapeutics said its Phase 3 LEVEL study of TNX-103 failed to meet its primary endpoint, sending shares down more than 85% in pre-market trading on August 10, RTTNews reported, in the latest setback for the chronic heart failure field.
The IZAR-1 trial met its week-16 primary endpoint with 42.1% of patients on the 60mg induction dose achieving ACR50, alongside strong skin and disease-activity secondary outcomes, and no new safety signals.
The FDA on August 5 approved mFlusiva, the first mRNA vaccine for seasonal flu, for adults 50 and older, after a late-stage trial of more than 40,000 people showed it was about 27% more effective than standard shots.
Blackstone-backed healthcare revenue cycle management firm AGS Health filed updated draft papers for a $500 million initial public offering in India, Reuters reported on August 7, adding to a strong year for Indian healthcare listings.
The FDA granted accelerated approval on August 6 to vusolimogene oderparepvec-wtpg (Tudriqev) plus nivolumab for advanced melanoma that progressed on PD-1 therapy, after an advisory panel voted 10-3 in favor of the regimen.
A landmark study of more than 2.5 million people identified 26 genetic risk loci for fibromyalgia and found the strongest evidence yet that it is a nervous system disorder rather than an autoimmune disease.
A study of 12,409 people followed for an average of 26 years found that three modifiable midlife risk factors — smoking, diabetes and hypertension — are linked to roughly 13 fewer years without dementia, NYU Langone and The Guardian report.
Tudriqev from Replimune — a herpes-virus-based injection given with nivolumab — shrank tumors in 24 percent of evaluable patients for a median 14.1 months, earning accelerated approval after an advisory panel voted 10-3.
Cambridge and ICR researchers say a Boadicea-based risk assessment would flag 26.5% of under-50 women for review and catch 34.8% of those who develop breast cancer within 10 years — versus 1.4% and 4.4% under current NICE rules.