FDA
The FDA delivered two notable approvals for RSV immunization, UroGen overcame a negative advisory committee vote to secure an approval in bladder cancer, and more key regulatory nods from the past month.
The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for sevasemten in Becker muscular dystrophy.
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.
Writing in JAMA, four former government officials warn that the Trump administration’s involvement in delaying the approval of Novavax’s COVID-19 vaccine could indicate a politicization of the drug approval processes that could ‘imperil public health.’
Mike Davis previously worked as clinical team leader at the FDA’s Division of Psychiatry before serving as chief medical officer for Usona Institute, a research organization advancing psychedelic science.
Robert F. Kennedy Jr. testified in front of largely combative congresspeople on vaccine policy, his MAHA report and more; the mass leadership exodus at the FDA continues as CDER and CBER shed key staff; Kennedy’s revamped CDC vaccine advisors convene for their first meeting; Novo and Lilly present new data at the American Diabetes Association’s annual meeting; and BioSpace recaps BIO2025.
With much to cover, Democrats tackled Kennedy’s MAHA report; the firing of all members of the CDC’s ACIP committee; and much more. Little was accomplished, as Kennedy demurred and members of Congress accused him of risking American lives.
Speaking at BIO2025, rare disease leaders from Ultragenyx, Amylyx and Yale questioned the need for the new regulatory pathway proposed by FDA Commissioner Marty Makary. They acknowledged, however, that creative thinking is required to enable more treatments for patients with ultrarare diseases.
While Eli Lilly brushed off concerns about gastrointestinal side effects for oral weight loss candidate orforglipron, analysts from William Blair worried that adverse events are not tapering off as expected.
Harliku is the only FDA-approved drug for alkaptonuria, a rare condition characterized by skin discoloration, arthritis and heightened risks of heart failure and stroke.
PRESS RELEASES