FDA lists 205 mol­e­c­u­lar tar­gets for pe­di­atric can­cer re­search

To help with an­ti-can­cer drug de­vel­op­ment, the FDA has de­vel­oped two new lists of mol­e­c­u­lar tar­gets to guide sub­mis­sions for pe­di­atric study plans.

The two lists, post­ed Tues­day by the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence, are aimed at fos­ter­ing the de­vel­op­ment of new on­col­o­gy drugs or bi­o­log­ics for pe­di­atric pop­u­la­tions. They al­so ful­fill a com­mit­ment the agency made un­der the FDA Reau­tho­riza­tion Act of 2017 (FDARA).

Scott Got­tlieb

One list points to the mol­e­c­u­lar tar­gets that are like­ly to con­tribute to the growth or pro­gres­sion of at least one pe­di­atric can­cer, while the oth­er iden­ti­fies the tar­gets of new drugs cur­rent­ly in de­vel­op­ment that would be au­to­mat­i­cal­ly ex­empt­ed from pe­di­atric can­cer study re­quire­ments.

“Pe­di­atric can­cer drug de­vel­op­ment has lagged far be­hind de­vel­op­ment of can­cer drugs for adults,” FDA Com­mis­sion­er Scott Got­tlieb said Tues­day in a Twit­ter thread an­nounc­ing the lists.

The cre­ation of these lists is fur­ther in­tend­ed to har­ness the po­ten­tial of tu­mor ge­net­ic pro­fil­ing in pe­di­atrics as well as lever­age the amend­ments made by sec­tion 504 of FDARA to sec­tion 505B of the FD&C Act, which set forth new re­quire­ments on pe­di­atric drug de­vel­op­ment.

“Un­til the pas­sage of FDARA, sec­tion 505B of the FD&C Act has not typ­i­cal­ly been a use­ful mech­a­nism to re­quire the de­vel­op­ment of drugs for pe­di­atric can­cers since most of the on­col­o­gy drugs ap­proved for adults are used to treat can­cers that are very rarely or nev­er oc­cur in chil­dren,” the agency said. “There­fore, his­tor­i­cal­ly, drug spon­sors have re­quest­ed and ob­tained waivers for con­duct­ing the re­quired as­sess­ments of these drugs in pe­di­atric pa­tients.”

The 2017 leg­isla­tive changes ad­dressed this is­sue by nix­ing the FD&C Act’s or­phan drug des­ig­na­tion ex­emp­tions on pe­di­atric as­sess­ments. Un­der FDARA, con­duct­ing these pe­di­atric as­sess­ments are re­quired “even when the adult in­di­ca­tion has re­ceived an or­phan des­ig­na­tion, or when the adult in­di­ca­tion does not oc­cur, in the pe­di­atric pop­u­la­tion,” the FDA added.

The agency iden­ti­fied a to­tal of 205 can­di­date mol­e­c­u­lar tar­gets for the de­vel­op­ment of the new lists. Most of these mol­e­c­u­lar tar­gets (77) are clas­si­fied as “oth­ers,” fol­lowed by those that tar­get a gene ab­nor­mal­i­ty (62), a cell lin­eage de­ter­mi­nant (40) and the tu­mor mi­croen­vi­ron­ment of the im­mune sys­tem (21). On­ly five mol­e­c­u­lar tar­gets are list­ed as can­di­dates for au­to­mat­ic waivers.

The lists form part of a broad­er ef­fort at the FDA to in­cen­tivize pe­di­atric drug de­vel­op­ment. Oth­er re­cent ac­tions in the space in­clude the FDA’s adopt­ed ver­sion of an In­ter­na­tion­al Coun­cil for Har­mo­niza­tion ad­den­dum and 2017 guid­ance that pro­vides pol­i­cy clar­i­fi­ca­tions on or­phan des­ig­na­tion sta­tus for pe­di­atric sub­pop­u­la­tions of com­mon dis­eases.


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.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

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We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

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His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

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The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

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Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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