Drug Delivery
George Tidmarsh takes over temporarily at CBER following Vinay Prasad’s abrupt departure; Replimmune trial leaders protest rejection reportedly driven by FDA’s top cancer regulator Richard Pazdur; Merck’s $3 billion savings push claims 6,000 jobs; and Pfizer CEO Albert Bourla addresses President Donald Trump’s new threats around Most Favored Nation drug pricing.
FEATURED STORIES
In the wake of multiple patient deaths from liver injuries related to Sarepta Therapeutics’ AAV gene therapy platform, some in the sector are looking for ways to improve the current technology, while others are eager to move on.
Amid a season of regulatory and scientific advances, experts reveal a culture of data hoarding among cell and gene therapy developers that is reinforcing fragmentation, stalling innovation and delaying access to treatments.
Analysts said the deal with Novo was likely giving Hims “‘credibility’ or increased consumer traffic,” adding that the “litigation risk is back on the table” now that the Danish pharma has stepped away.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
President Donald Trump’s One Big Beautiful Bill, signed into law last week, reintroduces broader exemptions for orphan drugs from the IRA’s drug price negotiation program—a move welcomed by the biopharma industry. The new tax law also cuts Medicaid funding, posing a minimal risk to pharma’s bottomlines and potentially jeopardizing hospitals’ 340B status. It does not, however, include new rules for pharmacy benefit managers that had been in an earlier draft.
California’s life sciences manufacturing jobs dipped 3.7% in 2024, according to a new Biocom California report. Still, several companies made—and continuing making—significant manufacturing investments in the state as key trends shape the discipline.
Jefferies analysts called the proxy filing, which is a standard disclosure after a merger agreement, “much more intriguing than normal” given the regulatory turmoil it revealed.
The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from sufficient to maintain momentum in the rare disease space.
The new version of the bill will still need to go through the entire House and Senate.
FDA Commissioner Marty Makary and CBER Director Vinay Prasad published an article in JAMA on Tuesday outlining the FDA’s priorities, including accelerating cures and the rapid deployment of artificial intelligence.
Despite substantial investments in groundbreaking treatments, companies struggle to communicate value early and to the right audience. Here’s how they can do better.
The overturning of the FDA’s lab-developed tests rule is just the tip of the iceberg. With the loss of Chevron deference, power has shifted from federal agencies to the courts, with potential implications for everything from the FDA shortage list to CMS drug price negotiations.
Here’s how companies can ensure they’re in compliance with new requirements that go into effect in August.
Leerink Partners previews the drugs likely to be subject to the Inflation Reduction Act’s next price negotiations as the program expands to Medicare Part B and smaller biotechs.