Rasonque's Broad Label Is Built on Survival, Not Response
The FDA's 321-page review of Rasonque documents the survival win that earned a broad pancreatic cancer label, and the mutation subgroups whose response data ran against the drug.
Section
Pipeline
Trials, approvals, and the drugs behind them.
One Transfer RNA Molecule Controls Whether Prostate Cancer Stays Treatable
A Nature paper finds that one arginine transfer RNA, tRNA1 Arg(UCU), keeps prostate tumors on androgen receptor signaling. When it falls, cells slip into a lineage-plastic, AR-inhibitor-resistant state.
ArriVent Beat the Chemotherapy Arm It Designed and Lost to the Referee It Chose
ArriVent's firmonertinib beat chemotherapy on investigator reads in a 398-patient Phase 3, but the blinded central review it chose returned p=0.0654, and first-line EGFR exon 20 lung cancer now belongs to combinations that keep chemotherapy in the regimen.
The First Authorized Treatment for Negative Schizophrenia Symptoms Is Not a Pill
FDA cleared Motivista as the first authorized treatment for negative symptoms of schizophrenia, but the pivotal trial showed a small-to-moderate effect against a control arm that also improved. The open question is whether payers will cover an app.
Is Claudin-1 Worth $355 Million Upfront? CSL Just Placed Its Bet
CSL is paying $355 million upfront for an antibody whose best evidence is a 26-patient interim readout. The deal prices claudin-1 biology, not proven data, across kidney and liver fibrosis.
FDA Will Review the First In Vivo CRISPR Therapy Without a Public Panel
Intellia's lonvo-z is on a priority-review track to March 10, 2027 with no advisory committee planned. The one-dose gene edit cut HAE attacks 87% in Phase 3, and the debate over permanence, competition, and price will happen largely out of view.
Celcuity Ships Its First Drug Into the Corner of Breast Cancer No PI3K Label Covered
Celcuity's REVTORPYK became commercially available September 30, claiming the PIK3CA wild-type corner of second-line HR-positive breast cancer no approved PI3K or AKT label covers. The launch must now overcome an immature survival read, heavy stomatitis, and a weekly IV schedule.
Bulevirtide Raised the Bar: Mirum's Brelovitug Phase 3 Win Faces a Market That Expects More
Mirum's brelovitug hit its Phase 3 endpoint in hepatitis delta, but Gilead's Hepcludex already holds the US approval and Vir's program is in flight. A strong readout no longer guarantees the market follows.
Onswik Makes Weekly Insulin a Two-Horse Race, and Now the Hard Question Begins
Lilly's once-weekly insulin Onswik is the second FDA approval in a six-month window, after Novo's Awiqli. Both won on non-inferiority data, leaving the real question open: whether weekly dosing actually improves insulin initiation and adherence.
The FDA Approved Atebrioz on a Volume Endpoint the FOP Trial Listed as Secondary
The FDA approved Atebrioz for FOP on a change in new bone volume, a measure the trial registry listed as secondary, after the lesion-count primary missed statistical significance at week 24.
Sodium Channel Blockers Failed Genetic Epilepsy for Decades. Relutrigine Took the State-Dependent Route.
Praxis Precision Medicines bypassed non-selective sodium channel toxicity in SCN2A and SCN8A DEEs by targeting persistent current. Now the FDA is examining its pivotal trial.
BigHat Closes $75 Million Series C as Its AI-Designed ADC Enters the Clinic
BigHat Biosciences raised $75 million as BHB810, a CDH17 VHH-Fc ADC, entered a Phase 1/2 gastric cancer trial. The round tests whether wet-lab data can fix AI drug discovery's translation problem.