New FDA CMC Draft Guidance Released for Personalized Antisense Oligonucleotide Products

Many patients rely on personalized medicine like antisense oligonucleotide (ASO) products for complex or treatment-resistant conditions. Meeting the needs of these patients is a challenge for pharmaceutical companies, as the expenses involved and small patient sizes complicate the establishment of chemistry, manufacturing, and control (CMC) processes that the FDA will support. To rectify the situation, the FDA has released a draft guidence on establishing appropriate CMC measures for ASO products.

According to a new BioProcess Online article, “This draft guidance complements a simultaneously issued draft guidance on clinical recommendations for individualized antisense oligonucleotide (ASO) genetic treatments2 which we covered in this article. The clinical recommendations draft guidance focuses on the structure of an investigational trial for an individualized ASO treatment and notes the limited applicability of these very small investigational trials to broader patient populations. Buttressing the clinical recommendations guidance, the CMC guidance addresses the unique issues that arise when manufacturing small batches of new personalized treatments.”

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(Source: BioProcess Online, April 26^th, 2022)