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Scientists who focus only on generating data risk missing their role in shaping strategy and driving innovation.
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New guidelines from two leading medical associations suggest that efforts to reduce bad cholesterol should focus on maintaining low levels of two key lipoproteins. Big pharma is all in, looking to improve on the standard statins to help vanquish America’s number one killer: heart disease.
The FDA’s decision last year to make complete response letters public provides new insight into why therapies sometimes fail to get the regulatory greenlight. Analysts say the information could help sponsors refine their regulatory strategies.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
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Robert F. Kennedy Jr.’s health department has consistently touted radical transparency as being key to its mission. Recent instances—the FDA’s decision not to disclose the recipients of three Commissioner’s National Priority Vouchers and FDA and CDC choices not to publish vaccine-related papers—call this intent into question.
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Representatives of companies including AbbVie, Eli Lilly, Johnson & Johnson and Merck have voiced concerns about the FDA’s approach to pre-approval inspections.
Varegacestat, a gamma secretase inhibitor, significantly improved progression-free survival while also meeting all key secondary endpoints in the pivotal RINGSIDE trial. Immunome is planning an FDA application for the second quarter of 2026.
Sanofi bought Dren’s DR-0201 program earlier this year for $600 million upfront and is running two Phase I trials in undisclosed inflammatory indications.
Vyvgart, an FcRn inhibitor already approved for generalized myasthenia gravis, is also being tested in myositis, Sjögren’s disease and the “clinically related” Graves disease.
Sanofi’s multiple sclerosis hopeful tolebrutinib faced dual setbacks on Monday, with a late-stage failure in one form of the disease and yet another regulatory setback in another.
The FDA had previously turned back the heart rhythm nasal spray twice, once in late 2023 with a refusal to file letter and again in March this year, when it flagged manufacturing issues.
For $950 million upfront, Sobi will gain ownership to pozdeutinurad, an oral URAT1 inhibitor that performed well in Phase II studies.
The FDA’s Vinay Prasad recently claimed in an internal memo that at least 10 children have died from coronavirus vaccines, but an internal safety review showed that the count was much lower.
In the midst of regulatory and political upheaval, biopharma’s R&D engine kept running, churning out highs and lows in equal parts. Here are some of this year’s most glorious clinical trial victories.
With notable therapies from Biogen, Sarepta and MacroGenics failing to show efficacy in pivotal or confirmatory trials, experts question the use of biomarker evidence for approval while one former regulator insists that a “failed trial is not a failed drug.”