News
FDA’s rare disease decisions are strongest when the patient community has a voice in advisory committee decisions.
FEATURED STORIES
Psychedelics are gaining momentum in depression, with one treating physician predicting that the drug class could “wipe out the SSRIs” if safety and durability hold up.
Saol Therapeutics is the latest biotech to resubmit for approval of a drug rejected under former FDA Commissioner Marty Makary, following REGENXBIO and Replimune.
Even as FDA approvals for biologic therapies fell in the first half of 2026, regulatory experts are optimistic about a turnaround in the rare disease space after the departure of key leaders at the agency. Still, there will continue to be tension between science and politics.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
Congressional letters sent to the CEOs of Eli Lilly, Pfizer, Merck, BMS and AbbVie this week voicing concerns about the pharmas’ clinical trials in China highlight an ongoing discrepancy in how government and industry think about the rise of the Asian country’s biotech industry.
THE LATEST
Novo Nordisk and Octagon Therapeutics announced that they forged a research collaboration focused on inflammatory diseases.
After enduring nine months of clinical holds, Merck announced that it is restarting the Phase III clinical program for islatravir, its candidate for people with HIV-1 infection.
A 3-judge panel questioned the legitimacy of Johnson & Johnson’s plan to form a subsidiary company with the purpose of absorbing legal liabilities from talc-related lawsuits.
Poxel presented positive histology data from its Phase II DESTINY-1 trial in non-alcoholic steatohepatitis (NASH), with its primary efficacy endpoint of liver fat decrease at 36 weeks.
This week’s money flowed into various therapeutic areas like cancer and inflammatory disorders, while government and philanthropic grants fund the fight against infectious diseases.
The FDA granted rare pediatric disease designation to AVR-RD-04, an experimental gene therapy treatment for the lysosomal storage disease cystinosis, AVROBIO announced Tuesday.
Shares of Spectrum Pharmaceuticals plunged 33% upon the release of briefing documents for an FDA advisory meeting regarding poziotinib
Beyond the usual challenge of delivering high-quality data that answers meaningful questions, PepGen’s Michelle Mellion is dedicated to “incorporating the patient’s voice into everything we do.”
Pres. Joe Biden declared the COVID-19 pandemic over. Biopharma stocks react as vaccines and antivirals are still in development.
Alzheon Inc. unveiled promising data from a Phase II biomarker trial of its investigational drug ALZ-801 to treat early Alzheimer’s disease.