News
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.
FEATURED STORIES
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.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
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 LATEST
Sarepta Therapeutics on Wednesday called the launch of the gene therapy for Duchenne muscular dystrophy “exceptional” but the company does not expect to see significant growth in the first half of 2024 due to its currently limited patient pool.
Incannex Healthcare on Wednesday said its investigational psychedelic psilocybin met the primary endpoint, demonstrating it can reduce Hamilton anxiety scores by more than nine points versus placebo.
On Wednesday, the CDC’s Advisory Committee on Immunization Practices voted to recommend Valneva’s chikungunya virus vaccine Ixchiq for certain high-risk patient groups.
This episode explores the drug pricing gap between consumer experience and industry norms. Join Lori and our KOLs as we explore the drivers of healthcare costs, the CBO, rebates, and overall spend in this new series.
Stakeholders Hope Bill Will Create IRA Drug Negotiation Parity Between Biologics and Small Molecules
Congressional legislation seeks to “equalize” the negotiating period between biologics and small molecules under the Inflation Reduction Act’s Drug Price Negotiation Program.
Electronic consent can personalize the patient experience in clinical trials and even improve retention. At Medable, where I am CSO, we’re committed to driving the adoption of this transformative technology.
Under the research and development agreement announced Wednesday, Viatris is securing global rights to Idorsia’s late-stage heart and lupus candidates.
The vaccine maker’s fourth-quarter revenue and earnings on Wednesday missed Wall Street expectations, while providing full-year 2024 sales guidance of flat to lower than last year.
Plus, last week Richard Gonzalez announced his retirement from AbbVie, PhRMA appointed Gilead’s O’Day to chair its board and Joseph Papa became president and CEO of Emergent.
AbbVie continued its dealmaking spree Wednesday by partnering with OSE Immunotherapeutics to develop its early-stage, first-in-class antibody for chronic and severe inflammation.