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.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,600+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,600+ biopharma pros reading Endpoints daily — and it's free.

FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Thomas Schall, ChemoCentryx CEO (file photo)

Chemo­Cen­tryx plunges as FDA rais­es ques­tions about rare dis­ease drug ahead of ad­comm

ChemoCentryx’s stock price on Wednesday was cut in half by the release of FDA briefing documents ahead of a Thursday adcomm, raising questions on the company’s clinical data to support avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

ANCA-associated vasculitides (AAV) affect small to medium-size blood vessels that can be fatal in less than a year if left untreated, according to FDA. Only Roche’s Rituxan is currently FDA-approved for the treatment of AAV, while glucocorticoids are approved for the broader indication of vasculitis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,600+ biopharma pros reading Endpoints daily — and it's free.

Stéphane Bancel at the Endpoints #JPM20 breakfast panel in San Francisco, January 2020 (Photo: Jeff Rumans, Endpoints News)

Mod­er­na says Covid-19 vac­cine boost­er in­creased im­mune re­sponse against vari­ants of con­cern

About a month after announcing their variant-specific Covid-19 vaccine boosters showed promising results in mice, Moderna says it now has some human data to back it up.

Volunteers given a booster shot about six to eight months after receiving their second dose saw increased antibody levels against SARS-CoV-2 and two variants of concern: B.1.351, which was first identified in South Africa, and P.1, first identified in Brazil, the company said on Wednesday.

Re­gen­eron’s gold­en goose Eylea may stave off biosim­i­lar com­pe­ti­tion un­til 2024 or be­yond

Almost 10 years have passed since its first FDA approval and Regeneron’s macular degeneration injection Eylea continues to pile up sales to the tune of about $5 billion per year, or more than half of Regeneron’s annual revenues.

Those billions are not expected to go anywhere anytime soon thanks to competition, even as Novartis subsidiary Sandoz announced Monday that it’s beginning a Phase III trial for an Eylea biosimilar in 460 patients across 20 countries.

Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,600+ biopharma pros reading Endpoints daily — and it's free.