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FDA Refuse-To-File Decisions Are Rare, But CMC And Ignoring Agency Advice Are Often Triggers

 
• By Sarah Karlin-Smith

Offering a unique peak behind the curtain at data that US FDA is generally prohibited from disclosing and that companies typically choose to keep secret, an agency analysis of two decades worth of refuse-to-file letters found the decisions can add a year and a half to a drug’s approval timeline.

refusing to file - 182843126 - shutterstock
FDA analyzes 10 years of refuse-to-file letters in a new journal article • Source: Shutterstock

US Food and Drug Administration refuse-to-file letters are generally issued for “substantive” deficiencies such as missing information needed to evaluate a product’s quality, safety, and efficacy, a new agency-led analysis of 103 refuse-to-file letters issued between 2008 and 2017 found.

The letters, which are issued when the agency determines a drug application is not complete enough for a full assessment,...

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

 
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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.

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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.

More from Pathways & Standards

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.

US FDA Commissioner Makary Is Learning To Appreciate His Staff

 
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Complete Response Letter Trio Raises Prospect Of Tidal Shift In Regenerative Medicine Regulation

 
• By Bridget Silverman

Replimune's RP1 oncolytic immunotherapy became the third regenerative medicine to receive an FDA complete response letter this month as the agency appears to re-visit previous agreements about accelerated approval.