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.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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