FDA Offers Two New Draft Guidances on Developing Targeted Therapies

Regulatory Affairs Professionals Society – The US Food and Drug Administration (FDA) on Friday published two new draft guidance documents that seek to support the development of treatments that address underlying genetic mutations that often cause or contribute to diseases, and another discussing how to determine if an in vitro diagnostic (IVD) device used in a study must undergo its own FDA review, distinct from a drug study.

"New drugs are being developed based solely on their ability to target these underlying molecular subtypes," FDA Commissioner Scott Gottlieb explained in a statement. "Moreover, this same molecular change may be present as the driving factor of many different disease phenotypes. When drugs successfully target these molecular mistakes to reverse the effects of different diseases, we need a development pathway that allows the new drug to pursue approval in each of these novel settings on the basis of the molecular marker that the drug targets. In the setting of oncology, this is often referred to as tissue agnostic drug development."

Earlier this year, FDA for the first time approved a tissue-agnostic treatment – Merck’s Keytruda (pembrolizumab) – based on a common biomarker rather than the location in the body where the tumor originated. These two draft guidance documents could help more companies take the route that Merck took to approval.

Read more at Regulatory Affairs Professionals Society.

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