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Duchenne Muscular Dystrophy: FDA Supports Broader Outcome Measures, Biomarkers

 
• By Brenda Sandburg

Embracing patient community recommendations, FDA draft guidance says historically controlled trials may support efficacy in some cases.

FDA has embraced many of the recommendations put forward in a community group’s proposed guidance for development of drugs to treat Duchenne muscular dystrophy, including use of biomarkers for accelerated approval and consideration of patient willingness to accept greater risks.

FDA released its 13-page draft guidance, "Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment," on June 9

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More from United States

Most Favored Nation Pricing Policy Floated In Medicaid, Coming Back To Medicare?

 
• By Cathy Kelly

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Pharma Industry Argues Tax, Other Incentives Better Than Tariffs

 
• By Jessica Merrill

Pharmaceutical industry organizations offered alternatives to tariffs that could maintain a secure domestic supply chain in public comments filed in response to a federal 232 investigation.

More from North America

US FDA Guidance Roadblock: Writing Continues, But Publication Has Slowed Or Stopped

 
• By Derrick Gingery

Cuts to the FDA’s policy and legal personnel have prevented dozens of product-specific guidances for generic drug development from being published.

FDA Adcomms Are Back: Four Cancer Drugs, COVID-19 Vaccine Formulations To Get Reviews

 
• By Sue Sutter and Bridget Silverman

The Oncologic Drugs Advisory Committee will meet for two days in mid-May, followed by a Vaccines and Related Biological Products Advisory Committee’s review of the 2025-2026 COVID-19 vaccine formulation.

Tougher Approval Standards May Follow Vinay Prasad’s Appointment To Lead US FDA’s CBER

 
• By Sarah Karlin-Smith

Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.