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Cholesterol Endpoints Draw Scrutiny At HoFH Panels

 
• By Cathy Dombrowski and Martin Berman-Gorvine

The Metabolic and Endocrinologic Advisory Committee accepts LDL lowering as endpoint for homozygous hypercholesterolemia drugs, backing approval of Aegerion’s lomitapide by 13-2 and Genzyme’s mipomersen by 9-6, but hones in on the need for better surrogates for cardiovascular morbidity and mortality.

The two-day review of a pair of antisense drugs for treatment of a rare genetic form of high cholesterol – Aegerion Pharmaceuticals Inc.’s lomitapide, and Genzyme Corp.’s Kynamro (mipomersen) – pointed to the need to develop better surrogates for cardiovascular outcomes than the traditional cholesterol measurements.

The correlation of lipid parameters with CV morbidity/mortality has been damaged as a number of outcomes trials have failed despite...

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More from United States

A Record Year For US FDA Approvals? Commissioner Says It’s Possible

 
• By Michael McCaughan

FDA Commissioner Martin Makary suggested 60 new molecular entities could be approved in 2025, which appears possible, but can the strained staff deliver?

Real-Time Release Of CRLs Is US FDA’s Goal, Agency Leaders Affirm

 
• By Michael McCaughan

The US FDA says its release of complete response letters for products subsequently approved by the agency is just a first step toward releasing rejection letters when they are issued, assuming legal issues can be resolved.

No Surprise Here: Foreign Drug Facilities Should Expect Surprise Inspections, US FDA Says

 
• By Sue Sutter

The FDA's recent announcement of plans to expand unannounced inspections at ex-US facilities should put firms on advance notice that their next foreign facility inspection may not be preannounced, Ivy Sweeney, acting head of the agency's drug inspections office said.

Base Benefit-Risk Decisions On Data, Not Opinion And Passion, CDER’s Tidmarsh Says

 
• By Sue Sutter

New US FDA drugs center director George Tidmarsh’s data-centric comments at a Reagan-Udall Foundation meeting on unapproved fluoride products could suggest patients’ and caregivers’ experiences will carry less weight in benefit-risk decisions under his leadership.

More from North America

Pink Sheet Podcast: FDA’s New Top Drug Regulator, Sarepta’s Options After Elevidys Stand-Off

 
• By Derrick Gingery, Sarah Karlin-Smith, and Nielsen Hobbs

Pink Sheet reporter and editors discuss the potential impact George Tidmarsh could have as director of the US FDA Center for Drug Evaluation and Research and the issues threatening the future of Sarepta’s gene therapy Elevidys.

EMA Says ‘No’ To Roche/Sarepta’s Elevidys, US FDA Investigates Fourth Death

 
• By Eliza Slawther and Sarah Karlin-Smith

The European Medicines Agency recommended against pan-EU marketing authorization of Roche/Sarpeta’s gene therapy Elevidys for the treatment of Duchenne muscular dystrophy as the US reported another death in an Elevidys patient.

Merck’s Keytruda Gains Medicare Negotiation Reprieve With Expanded Orphan Exemption

 
• By Cathy Kelly

Bristol Myers Squibb’s Opdivo and Merck's Welireg also could benefit from the delay in Medicare price negotiation process eligibility.