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FDA Commissioner Marty Makary intends to resign on Tuesday, according to several sources. This report follows a tumultuous 13-month tenure in which Makary oversaw the controversial rejections of several rare disease drugs and “predictable volatility” within the agency.
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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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Astellas Pharma and CytomX Therapeutics first partnered up in March 2020. For $80 million upfront, the Japanese pharma gained access to the biotech’s Probody platform to generate masked antibody therapies for cancer.
A Massachusetts judge called Kennedy’s efforts to reform the CDC’s vaccines advisory panel a “procedural failure,” adding that the new committee members do not “comport with governing law.”
In a highly competitive space, AI platforms must now prove themselves through proprietary data, focused pipelines and clinical readouts in competitive diseases. Promises of faster, cheaper drug discovery are not enough to entice strong investor engagement.
With Ascendis Pharma entering the achondroplasia space last month and BridgeBio on deck, BioMarin faces competition. Adding to the pressure, the company suffered a setback Monday when it halted two studies of Voxzogo in other growth-related conditions following multiple cases of hip injuries in other trials of the drug.
Structure Therapeutics’ oral obesity drug elicited a placebo-adjusted weight loss of 16.3% at 44 weeks in a Phase 2 trial. The biotech is planning to launch a late-stage program for the drug later this year.
Health Secretary Robert F. Kennedy Jr. has become increasingly unpopular among several government officials, largely as a result of his antivaccine rhetoric and actions. Other contentious issues reportedly include the approval of an abortion pill and other controversial FDA decisions.
Sana Biotechnology is looking to start clinical development for its type 1 diabetes therapy SC451 this year.
The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.
As the Formula 1 season kicks off in the APAC region, biopharma companies can take a page from the popular sport’s playbook around precision, adaptability, and the right team. In return, biopharma can also offer some lessons to elite motor sport on well-rounded excellence in the race to market.
Dozens of biotechs reported earnings this week. BioSpace recaps key highlights from Capricor Therapeutics, Legend Biotech, Inovio and Allogene.