Regulatory
The approval of Ionis Pharmaceuticals’ Tryngolza for severe hypertriglyceridemia could spur “substantial growth” for the product, according to William Blair.
Gilead’s Trodelvy can now be used as a monotherapy and in combination with Merck’s Keytruda to treat certain patients with triple-negative breast cancer.
The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.
Recent BioSpace industry conference conversations point to sponsors pushing earlier on safety, quality and performance data, leading to demand for nitrosamine analysis, IVRT/IVPT and microbiome database capabilities.
A new report from RBC Capital Markets lists Biotechnology Innovation Organization chief executive John Crowley and military physician Heidi Overton as potential candidates for the role of FDA commissioner—in addition to the agency’s current acting leader, top food regulator Kyle Diamantas.
AbbVie scooped up immunology player Apogee Therapeutics for nearly $11 billion in one of the year’s top deals to-date, while Sanofi made a big play to survive its upcoming Dupixent patent cliff; FDA uncertainty continues as the agency changes direction on gene therapies by uniQure and REGENXBIO; and Jef Akst and Annalee Armstrong report back from San Diego.
While merger and acquisition activity has been robust of late, frequent changes in guidance and leadership at the regulator add risk to any transaction.
Former FDA, CDC and NIH leaders convene at the BIO International Convention to discuss the dismantling of the Department of Health and Human Services under the Trump administration—and where we go from here.
Among the health department’s efforts is an expedited investigational new drug pilot program that would leverage collaborations with U.S. research institutions to reduce early trial timelines by as much as 12 months.
The resubmission for RGX-121, expected in the third quarter, comes as the FDA has deemed REGENXBIO’s existing data “sufficient” to support an accelerated filing. It immediately follows a similar reversal of position regarding uniQure’s embattled Huntington’s disease gene therapy.
PRESS RELEASES