Drugs con­tain­ing nano­ma­te­ri­als: FDA fi­nal­izes 2017 guid­ance with sev­er­al changes

The FDA on Thurs­day fi­nal­ized a draft guid­ance from 2017 ex­plain­ing the prin­ci­ples and spe­cif­ic con­sid­er­a­tions for de­vel­op­ing drugs (bi­o­log­ics and gene ther­a­pies are ex­clud­ed) con­tain­ing nano­ma­te­ri­als, in­clud­ing via ab­bre­vi­at­ed path­ways.

Con­sid­er­a­tions re­lat­ed to qual­i­ty, non­clin­i­cal, and clin­i­cal stud­ies are in­clud­ed in the 29-page fi­nal guid­ance, which al­so fea­tures “two note­wor­thy changes” from the draft ver­sion in re­sponse to stake­hold­er com­ments, ac­cord­ing to the FDA.

“First, the fi­nal guid­ance pro­vides a glos­sary of ter­mi­nol­o­gy to as­sist in un­der­stand­ing how im­por­tant terms are used in the doc­u­ment,” the FDA said. The fi­nal ver­sion now in­cludes de­f­i­n­i­tions for terms like na­noemul­sion (“A nano­ma­te­r­i­al ki­net­i­cal­ly sta­bi­lized emul­sion sys­tem, with at least one fea­ture that falls in­to the size range of 1-1,000 nm”) and “Nano­ma­te­r­i­al-as­so­ci­at­ed Drug” (i.e. a drug that’s trapped, bound, en­cap­su­lat­ed, or as­so­ci­at­ed with a nano­ma­te­r­i­al).

“Sec­ond, sev­er­al re­vi­sions were made to re­flect FDA’s cur­rent think­ing with re­spect to ab­bre­vi­at­ed ap­pli­ca­tions, in­clud­ing ab­bre­vi­at­ed new drug ap­pli­ca­tions (AN­DAs), for prod­ucts con­tain­ing nano­ma­te­ri­als,” the agency said in to­day’s Fed­er­al Reg­is­ter.

On the top­ic of gener­ics, the fi­nal guid­ance ex­plains how nano­ma­te­ri­als can range from sim­ple nanocrys­tals, or­gan­ic nano­ma­te­ri­als (e.g., li­po­some, poly­mer­ic nanopar­ti­cle), and in­or­gan­ic nano­ma­te­ri­als (e.g., gold nanopar­ti­cles), to com­plex in­te­grat­ed nanopar­ti­cles (e.g., core-shell, sur­face mod­i­fied nanopar­ti­cles), and AN­DA ap­pli­cants are “re­spon­si­ble for pro­vid­ing suf­fi­cient sci­en­tif­ic ev­i­dence based on a com­pre­hen­sive in vi­vo PK eval­u­a­tion and in vit­ro physic­o­chem­i­cal char­ac­ter­i­za­tion to demon­strate bioe­quiv­a­lence be­tween a pro­posed gener­ic drug and its nano­ma­te­r­i­al-con­tain­ing RLD.

“In ad­di­tion, for an ac­tive in­gre­di­ent that is a nano­ma­te­r­i­al, com­pre­hen­sive char­ac­ter­i­za­tion of the RLD [ref­er­ence list­ed drug] and un­der­stand­ing of the fun­da­men­tal chem­istry used to form the ac­tive in­gre­di­ent may be need­ed to demon­strate ac­tive in­gre­di­ent same­ness. Cur­rent think­ing is that any crit­i­cal struc­tur­al change in the mul­ti­ple com­po­nents of nano­ma­te­r­i­al-based prod­ucts may in­flu­ence the BE [bioe­quiv­a­lence], phar­ma­col­o­gy, and tox­i­col­o­gy pro­files,” the guid­ance adds.

But AN­DAs ref­er­enc­ing oral drugs us­ing nano­ma­te­ri­als to im­prove bioavail­abil­i­ty for poor wa­ter-sol­u­ble drugs, ac­cord­ing to the FDA, need not use that par­tic­u­lar nano­ma­te­r­i­al or any nano­ma­te­r­i­al, but may use al­ter­na­tive strate­gies to achieve the same BA en­hance­ment, the FDA adds.

In gen­er­al, how­ev­er, the guid­ance notes that a spon­sor should eval­u­ate the po­ten­tial risk(s) as­so­ci­at­ed with drugs con­tain­ing nano­ma­te­ri­als and should as­sure: “(1) ad­e­quate char­ac­ter­i­za­tion of the nano­ma­te­r­i­al, and (2) ad­e­quate un­der­stand­ing of a nano­ma­te­r­i­al’s in­tend­ed use and ap­pli­ca­tion, and of how the nano­ma­te­r­i­al at­trib­ut­es re­late to prod­uct qual­i­ty, safe­ty, and ef­fi­ca­cy. ”

In ad­di­tion to changes in re­sponse to com­ments, the FDA al­so said that the fi­nal guid­ance doc­u­ment’s dis­cus­sion re­gard­ing over-the-counter mono­graph drugs has been up­dat­ed for con­sis­ten­cy with the en­act­ment of OTC re­form pro­vi­sions of the Coro­n­avirus Aid, Re­lief, and Eco­nom­ic Se­cu­ri­ty Act.

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