FDA
Regulatory challenges have been even more top of mind than usual given recent upheaval at the FDA. BioSpace spoke to three industry experts about key issues, which include applying new artificial intelligence guidance. The experts also shared advice for working with regulators.
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The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding for the Immigration and Customs Enforcement’s large-scale crackdown in Minnesota and other states.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
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In what is shaping up to be a back-loaded month, the FDA is set to release a slew of regulatory decisions in February, including two that would expand the labels of blockbuster drugs.
New draft guidance from the FDA on multiple myeloma endpoints reflects the new technology available to assess disease and how patient journeys have changed with better treatments.
The FDA is currently reviewing Summit’s PD-1/VEGF bispecific as part of a chemotherapy combo for the treatment of locally advanced or metastatic non-squamous non-small cell lung cancer.
After years of contraction, investors see biotech reentering a growth cycle driven by scientific progress, asset quality and renewed conviction in oncology, obesity and neuroscience innovation.
The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
Darzalex Faspro, in combination with an anti-cancer triplet, is the first anti-CD38-based regimen for newly diagnosed patients with multiple myeloma, regardless of eligibility for stem cell transplantation.
After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
True inspection readiness is about the integrity of a company’s entire system.
The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
The U.S. regulator shared the roadmap for implementing the program, first proposed in August 2025, and teased changes made in response to industry feedback.