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.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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