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US FDA Limits GMP Exemption For ‘N of 1’ Investigational New Drugs

 
• By Bowman Cox

Agency drafts guidance on how to apply requirements for chemistry, manufacturing and controls, and for good manufacturing practices, to bespoke antisense nucleotides.

N of 1
assessing "n of 1" quality • Source: Alamy

For certain bespoke therapies, the current regulatory exemption from good manufacturing practices requirements for Phase I investigational new drug products will only apply to the first batch, the US Food and Drug Administration explains in 7 December draft guidance.

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Gene Therapy Sponsor INTERACT, Pre-IND Meeting Confusion Lingers Despite FDA Guidance

 
• By Sue Sutter

PhRMA questioned whether proof-of-concept studies are needed before an INTERACT meeting and said a draft guidance describing the timing of definitive toxicology studies creates confusion.

US FDA Shows Support For Generic Drug User Fee Goals Amid Cutbacks

 
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The FDA struck an upbeat tone during its annual Generic Drugs Forum despite an unprecedented period of cutbacks and disruptions and reiterated a desire to continue meeting user fee goals.

US FDA’s Makary Floats Vague ‘Plausible Mechanism’ Approval Pathway For Rare Diseases

 
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Ascertaining the novelty and benefits of Makary’s new drug approval idea – along with what it would take to implement it – requires more clarity on the types of studies he envisions being used to grant and confirm “plausible mechanism’ approvals.

New EU Approvals

 
• By Neena Brizmohun

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Tivdak, Pfizer/Genmab’s treatment for previously treated recurrent or metastatic cervical cancer.

More from Pathways & Standards

EU Pharmacopoeia Adopts ‘Groundbreaking’ Cell Therapy Chapter, Seeks Feedback On Alternative Microbiological Tests

 
• By Neena Brizmohun

A new chapter that will be published in the European Pharmacopoeia soon is expected to provide a comprehensive framework for the production and quality control of cell-based preparations. Meanwhile, an existing chapter has been revised to facilitate the implementation of rapid microbiological testing methods.

US FDA User Fee Collection Safe In Preliminary Trump 2026 Budget Plan

 
• By Cathy Kelly

The preliminary White House 2026 budget plan cuts agency funding, but not so sharply that user fees are excluded from collection.

New EU Approvals

 
• By Neena Brizmohun

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Tivdak, Pfizer/Genmab’s treatment for previously treated recurrent or metastatic cervical cancer.