FDA preps for a new and flex­i­ble ap­proach to genome edit­ing in an­i­mals

With the use of CRISPR pro­lif­er­at­ing and gene dri­ves on the hori­zon in the US, the FDA said that it’s now look­ing at a risk-based ap­proach to reg­u­lat­ing the gene edit­ing of an­i­mals.

As part of that ap­proach the agency is es­tab­lish­ing a new pi­lot pro­gram, known as the Vet­eri­nary In­no­va­tion Pro­gram (VIP). It’s charged with pro­vid­ing tech­ni­cal and pro­gram­mat­ic as­sis­tance to de­vel­op­ers seek­ing FDA ap­proval of in­ten­tion­al­ly al­tered ge­nom­ic DNA in an­i­mals and an­i­mal cells, tis­sues and cell- or tis­sue-based prod­ucts that pro­vide a ben­e­fit to hu­man health, an­i­mal health, an­i­mal well-be­ing or food pro­duc­tion.

“The goal of the VIP is to fa­cil­i­tate ad­vance­ments in the de­vel­op­ment of in­no­v­a­tive an­i­mal prod­ucts by pro­vid­ing greater cer­tain­ty in the reg­u­la­to­ry process, en­cour­ag­ing de­vel­op­ment and re­search, and sup­port­ing an ef­fi­cient and pre­dictable path­way to ap­proval for cer­tain in­no­v­a­tive an­i­mal prod­ucts,” the FDA said in a new re­port.

In 2019, the agency al­so in­tends to pub­lish guid­ance clar­i­fy­ing that risk-based cat­e­gories will in­clude: “an FDA de­ci­sion not to en­force ap­proval re­quire­ments with no pri­or re­view, an FDA de­ci­sion not to en­force ap­proval re­quire­ments fol­low­ing a re­view of da­ta that ad­dress spe­cif­ic risk ques­tions, and an FDA de­ci­sion to re­view for ap­proval with da­ta re­quire­ments pro­por­tion­ate to the risk as­so­ci­at­ed with the par­tic­u­lar prod­uct.”

This ap­proach in­cludes flex­i­bil­i­ty to trans­fer prod­ucts across these cat­e­gories based on spe­cif­ic con­di­tions and as FDA gains fa­mil­iar­i­ty with dif­fer­ent prod­uct risk pro­files. How­ev­er, FDA is still pro­hib­it­ed by Con­gress from ac­cept­ing any in­ves­ti­ga­tion­al new drug ap­pli­ca­tion for “re­search in which a hu­man em­bryo is in­ten­tion­al­ly cre­at­ed or mod­i­fied to in­clude a her­i­ta­ble ge­net­ic mod­i­fi­ca­tion.”

In an­oth­er draft guid­ance, FDA in­tends to clar­i­fy its reg­u­la­to­ry ap­proach for cat­e­gories of in­ten­tion­al­ly ge­net­i­cal­ly al­tered an­i­mals used in re­search and plans to out­line, based on risk, when FDA in­tends to ex­er­cise en­force­ment dis­cre­tion or when it in­tends to en­force the re­quire­ment for an ap­proved new an­i­mal drug ap­pli­ca­tion.

An ad­di­tion­al draft guid­ance for in­dus­try will es­tab­lish an al­ter­na­tive type of file as a repos­i­to­ry for in­for­ma­tion ex­changes with the FDA’s Cen­ter for Vet­eri­nary Med­i­cine (CVM) for prod­ucts that are in ear­ly de­vel­op­ment stages or that are de­vel­oped for re­search pur­pos­es on­ly and may nev­er progress to a mar­ketable prod­uct.

In ad­di­tion, the FDA’s CVM in­tends to hold a pub­lic we­bi­nar on 3 De­cem­ber to dis­cuss the sci­ence be­hind genome edit­ing in an­i­mals, the promis­ing us­es of this tech­nol­o­gy in an­i­mals and the po­ten­tial risks, and in­for­ma­tion about CVM’s risk-based ap­proach to the reg­u­la­tion of in­ten­tion­al ge­nom­ic al­ter­ations to an­i­mals.

And to en­hance trans­paren­cy, FDA al­so said it will list on its web­site the spe­cif­ic an­i­mals or cat­e­gories of an­i­mals with in­ten­tion­al ge­nom­ic al­ter­ations for which the agency has de­cid­ed to ex­er­cise en­force­ment dis­cre­tion with re­gard to pre­mar­ket ap­proval re­quire­ments.

First pub­lished here. reg­u­la­to­ry fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.


Zachary Brennan

managing editor, RAPS

John Hood [file photo]

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