New Products
The agency granted accelerated approval to zanidatamab in HER2-positive biliary tract cancer, though the company is eyeing larger indications for the drug.
Astellas's C5 inhibitor faces another approval challenge, this time for a supplemental filing in the US, following the withdrawal of a European submission, although it continues to do well in what is its only market so far.
The drug maker anticipates a potential label expansion for Revuforj (revumenib) from KMTA2-translocation acute leukemias into NPM1-mutant AML.
Following ex-US approvals, PTC obtains a US OK for Kebilidi, branded Upstaza outside the US, as a gene therapy for ultra-rare AADC deficiency. The drug is administered via neurological surgery.
Sales of its novel COPD therapy in its first quarter on the market were more than double consensus estimates, and Verona sees more to come.
Doubling some analyst projections for its second full quarter on market, Madrigal’s NASH drug appears headed to blockbuster status and a sustained market position.
The US FDA approved Orlynvah for uncomplicated urinary tract infections among adult women, despite some concerns about the potential for off-label use and resistance.
Pfizer beat GSK with a new indication for its RSV vaccine in high-risk individuals aged 18-59.
An exec said the company is confident it can ensure testing for the biomarker becomes routine for the drug, which is the first FDA-approved medicine to target CLDN18.2.
Vyalev, a 24-hour constant infusion of carbidopa and levodopa prodrugs, obtained US FDA approval and will launch at $119,000 per year.
The US FDA approved the drug for hemophilia A and B, and while it may struggle to compete in hemophilia A against Roche’s Hemlibra, it has a big convenience advantage in hemophilia B.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
While vatiquinone may have missed its primary endpoint in a Phase III trial, PTC is ready to file the drug for the debilitating, life-shortening disorder after showing that it slowed disease progression over 144 weeks.
Arexvy has become the first RSV vaccine to show it can provide protection over three years – but that might mean lower market uptake than originally expected.
Dalzanemdor missed its primary endpoint in a Phase II Parkinson’s study, Sage announced, following April’s Phase II miss in Alzheimer’s. Phase II data in Huntington’s are still expected this year.
Phase III success positions Scholar Rock’s selective myostatin inhibitor for regulatory filings in early 2025. Analysts see a blockbuster opportunity for additive therapy in spinal muscular atrophy.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
PD-1 inhibitor Opdivo has obtained US FDA approval for pre- and post-surgical treatment in operable non-small cell lung cancer, but Merck and AstraZeneca got there first.
CEO Chris Cargill talks to Scrip about Sosei Heptares’ new identity as Nxera, the ups and downs of being big pharma’s go-to small-molecule drug hunter and its move into commercialization in Japan.
Avidity hopes to complete enrollment in mid-2025 for an ongoing Phase III trial of its antibody-oligonucleotide conjugate in myotonic dystrophy type 1, which has no approved drug therapy.