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.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

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