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One Patient, Many Questions: Milasen ‘N of 1’ Trial Provides Early Template For Individualized Genomic Medicine

 
• By Bridget Silverman

Rapid development of patient-customized oligonucleotide at Boston Children’s Hospital suggests promise and hazards of new drug development model.

The rapid development of milasen, a bespoke therapy for a single patient with a rare genetic disease described in a landmark New England Journal of Medicine article, suggests how the US Food and Drug Administration’s regulatory framework for new drugs will need to stretch to accommodate the challenge of individualized genomic medicine.

The splice-modulating antisense oligonucleotide (ASO) milasen offers a “possible template for the rapid development of patient-customized treatments,” study authors Jinkuk...

More from Approval Standards

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UK MHRA: Bacteriophage Developers Wanted ‘More Clarity’ On UK Framework

 
• By Eliza Slawther

Julian Beach, interim executive director of healthcare quality and access at the UK drug regulator, the MHRA, tells the Pink Sheet how the agency is responding to increased efforts to develop bacteriophage therapies.

LDT Final Act? US FDA Will Not Appeal District Court Decision Vacating Final Rule

 
• By Brian Bossetta

The US FDA ended efforts to regulate lab-developed tests as medical devices for now when it did not appeal a decision from the Eastern District of Texas that tossed out the agency's final rule.

MHRA Backs Bacteriophage Innovation To Fight Antimicrobial Resistance

 
• By Eliza Slawther

The UK regulator wants to help companies to develop bacteriophages for the treatment of infections. Its first guidance on this topic offers advice to researchers and sets out the regulatory requirements they will need to meet.