News
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.
FEATURED STORIES
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.’
Leading companies spent $1.4 billion upfront on licensing deals and embarked on vast R&D programs. Clinical setbacks mean many companies are unlikely to ever recoup their investments.
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.
Job Trends
The SURVEYOR Study met its primary endpoint demonstrating a statistically significant difference as measured by the HD-Cognitive Assessment Battery ( HD-CAB) composite score at baseline between healthy participants and participants with Huntington’s Disease (HD) prior to any treatment with dalzanemdor (SAGE-718) or placebo; further underscoring the cognitive impact of HD For participants with HD that received dalzanemdor or placebo, dalzanemdor was generally well-tolerated with no new
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
During the COVID-19 pandemic, Health Secretary Robert F. Kennedy Jr.—along with FDA Commissioner Marty Makary and CBER Director Vinay Prasad—argued against vaccine mandates, partly because they limited medical choice. This week, the FDA under their leadership approved updated COVID-19 vaccines with restrictions that do the same.
THE LATEST
Brazilian authorities said the death was unlikely to have been caused by Elevidys and was instead more in line with severe infection exacerbated by immunosuppression.
Despite the failure of its Recognify-partnered inidascamine, Jefferies analysts do not expect a definitively negative stock impact on atai, given the company’s promising psychedelic pipeline.
Acknowledging the limits of disease-modifying drugs like Leqembi and Kisunla, companies like Bristol Myers Squibb, Acadia, Otsuka and Lundbeck are renewing a decades-old search for symptomatic treatments, including in high-profile drugs like Cobenfy.
These five upcoming data drops could usher in more effective and convenient therapies for Alzheimer’s disease and open up novel pathways of action to treat the memory-robbing illness.
The Commissioner’s National Priority Vouchers aim to offer accelerated pathways to drugs that meet certain criteria, perhaps including a low price-tag. But the policy is vaguely defined and was announced without public input, going against the FDA’s own published practices, experts say.
The collaboration focuses on ‘molecular gates,’ a class of molecules that the startup company Gate Bioscience says can stop pathogenic proteins from leaving the cell.
The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne muscular dystrophy.
The strategic reprioritization comes after the company hit two major hurdles in the past year, including a clinical hold for an investigational gene therapy and an FDA rejection for its lead asset.
CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an anonymous senior FDA official. Given Elevidys’ full approval, however, experts told BioSpace this path would set up a length legal battle between the regulator and Sarepta Therapeutics.
The company didn’t share specific data for the molecule, gefurulimab, but said it hit all endpoints in the Phase III PREVAIL trial and promised to share more at an upcoming scientific meeting.