Izervay: what flexibility did regulators accept, and why?

“The ocular studies were conducted in rabbit and dogs, which, as the Applicant indicated, are species in which ARC1905 is expected to have very low activity.”

“Therefore, the monkey has no advantage over the rabbit or dog, and the reviewer considers that the rabbit and dog are acceptable species.”

“The Applicant justification to omit the conduct of nonclinical carcinogenicity studies is based on avacincaptad pegol mechanism of action, route of administration, a supportive literature review, an analysis of pharmacological class effects, and nonclinical and clinical data collected to date.”

“The Applicant submitted a justification not to conduct carcinogenicity, fertility/early embryonic development, and pre-/postnatal development studies. The Pharmacology/Toxicology team concluded that the weight of evidence supports that the studies are not necessary for the intended dosing regimen and indication.”

“Nonclinical recommendations for (1) submission for a waiver request for FEED (fertility and early embryonic developmental) and PPND (pre-and postnatal developmental), along with the justification and supporting data, (2) conduct of embryofetal development studies in relevant species”

“The Applicant submitted a justification not to conduct carcinogenicity, fertility/early embryonic development, and pre-/postnatal development studies. The Pharmacology/Toxicology team concluded that the weight of evidence supports that the studies are not necessary for the intended dosing regimen and indication.”