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BioMarin’s Vimizim Secures Broad Label In Morquio A Syndrome

 
• By Sue Sutter

No limits on age or disease severity of patients appear in elosulfase alfa labeling; agency reviewers had raised the issue of baseline function at a November advisory committee meeting.

FDA cleared BioMarin Pharmaceutical Inc.’s Morquio A syndrome treatment Vimizim (elosulfase alfa) Feb. 14 with a broad label that places no limits on the enzyme replacement therapy’s use either by baseline disease severity or age – despite agency questions about efficacy in patients with better mobility and a lack of efficacy and safety data in very young children.

Two weeks ahead of its Feb. 28 PDUFA deadline, FDA approved Vimizim, a hydrolytic lysosomal glycosaminoglycan-specific enzyme, as for the...

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• By Bridget Silverman

Quick responses to late-breaking changes to US FDA COVID-19 vaccine policy kept Novavax's Nuvaxovid and Moderna's mNexspike close to expected approval timelines despite revised indications and new post-marketing trial commitments.

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• By Sarah Karlin-Smith

The once and current CBER director’s justification for his COVID-19 decision stands as a warning of the uncertainty sponsors, particularly those in the vaccine space, may now face at FDA.

GLP-1s In Crosshairs As Employers Worry About Health Costs – And Their Own Reliance On Rebates

 
• By Cathy Kelly

Business Group on Health annual employer survey highlights fast-growing Rx costs, led by the GLP-1 drugs, and payer strategies for controlling them. Policy prescriptions are unclear, though, since employers are loath to leave PBMs and nervous about pending changes to Medicare and Medicaid.