FDA
Following the dramatic late-night resignation of Peter Marks last Friday, Steele, a senior advisor to the division, takes the reins in a department inside an agency beset by cuts, layoffs, and confusion.
FEATURED STORIES
Biosimilars are essential healthcare equalizers, but their regulation is overly complicated due to lobbying by makers of branded biologics looking to maintain blockbuster revenue.
The FDA approval of Alnylam’s Amvuttra sets up a three-way race with Pfizer and BridgeBio, which both market transthyretin stabilizers for transthyretin amyloid cardiomyopathy.
The FDA’s Oncologic Drugs Advisory Committee recently voted to narrow the label for checkpoint inhibitors Keytruda and Opdivo in stomach and esophageal cancers based on PD-L1 expression levels—but the high unmet need in these patient populations should also be considered.
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Milestone Pharmaceuticals hit another bump in the road in its quest to get Cardamyst approved for paroxysmal supraventricular tachycardia when the FDA issued a Complete Response Letter on Friday.
Soleno’s Vykat XR is the first drug approved for the rare disease that directly targets its hallmark symptom.
This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.
On the agenda for the FDA this month are two RNA-based treatments for rare diseases.
Marty Makary, likely FDA commissioner under President Trump, appeared before Congress this week as the agency he’s set to lead continues to be rocked by sweeping changes and about-faces.
While Congress is renewing the priority review voucher program for rare pediatric diseases, the FDA should be required to keep public records of the passes changing hands, too.
Neffy 1 mg is the “first significant innovation” for epinephrine delivery in small children aged 4 years and up in over 35 years, according to ARS Pharmaceuticals.
The vaccine space has been battered by strong headwinds in recent weeks, including high-level disruptions to FDA and CDC advisory committee meetings.
Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple effect is already being felt across biopharma.
The approval for the first-line treatment of esophageal squamous cell carcinoma comes shortly after a label expansion for the drug in gastric and gastroesophageal cancers as BeiGene also pushes forward a pipeline of novel cancer therapies.