FDA has about one month of user fee funds left, Got­tlieb warns

As the House preps to take ac­tion on in­di­vid­ual ap­pro­pri­a­tions bills this week, in­clud­ing one that will fund the FDA, Com­mis­sion­er Scott Got­tlieb has fur­ther clar­i­fied what the agency has left in user fee funds, what re­view ac­tiv­i­ties will take place and how the agency is help­ing its em­ploy­ees con­duct in­spec­tions.

Due to the par­tial shut­down, the FDA can­not ac­cept new 2019 user fees, which means the agency can­not ac­cept new med­ical prod­uct ap­pli­ca­tions un­less they are user-fee-pro­gram re­lat­ed but don’t re­quire the pay­ment of a user fee, which is “a very small sub­set of ap­pli­ca­tions,” Got­tlieb not­ed.

And though the FDA will con­tin­ue to work on ex­ist­ing ap­pli­ca­tions where user fees were paid pri­or to 22 De­cem­ber 2018, FDA can on­ly use user fee car­ry­over bal­ances from FY 2018, and Got­tlieb not­ed that among the med­ical prod­uct user fee pro­grams, the one that will run out of car­ry­over bal­ance first is like­ly the Pre­scrip­tion Drug User Fee Act (PDU­FA) funds, which are used for new drug re­views. “We have about one month of fund­ing left,” Got­tlieb said.

Got­tlieb has promised to re­lease fur­ther in­for­ma­tion on the ac­count bal­ances, burn rate and ap­prox­i­mate­ly how long ac­tiv­i­ties can con­tin­ue un­der car­ry­over bal­ances, ac­cord­ing to the non­prof­it Al­liance for a Stronger FDA’s shut­down toolk­it.

On the gener­ic drug end, dur­ing the par­tial shut­down, the FDA will not ac­cept gener­ic drug sub­mis­sions that re­quire pay­ment of a fee.

Got­tlieb clar­i­fied that FDA will be able to process cer­tain sub­mis­sions for gener­ic drugs us­ing car­ry­over user fees, such as changes be­ing ef­fect­ed (CBE) and pri­or ap­proval sup­ple­ments, amend­ments, an­nu­al re­ports and ap­pli­ca­tions for positron emis­sion to­mog­ra­phy (PET) drugs. He al­so said that the agency can ac­cept drug mas­ter files (DMFs) to be ref­er­enced in gener­ic drug ap­pli­ca­tions, but that the agency won’t be able to con­duct ini­tial com­plete­ness as­sess­ments on Type II ac­tive phar­ma­ceu­ti­cal in­gre­di­ent (API) DMFs if the user fee has not been paid.

“Spon­sors who haven’t paid GDU­FA fa­cil­i­ty fees for FY19 shouldn’t re­mit pay­ment dur­ing the lapse pe­ri­od be­cause FDA can­not ac­cept the fees,” he added.

And if a gener­ic drug ap­pli­ca­tion ref­er­ences, for the first time af­ter 22 De­cem­ber 2018, a Type II API DMF for which the fee has not been paid, then FDA will no­ti­fy the ap­pli­cant that the fee must be paid with­in 20 cal­en­dar days. If the fee is not paid with­in 20 cal­en­dar days of that no­tice, the FDA will not re­ceive the ap­pli­ca­tion. “At this time, FDA has not de­ter­mined what ap­proach it will take if the 20-cal­en­dar-day pe­ri­od ex­pires dur­ing the lapse pe­ri­od,” Got­tlieb said.


While prais­ing the work of FDA staff con­duct­ing crit­i­cal in­spec­tions of food and drug fa­cil­i­ties who are in an “un­paid, ex­cept­ed sta­tus,” mean­ing they’re work­ing on crit­i­cal pub­lic health func­tions with­out pay or re­im­burse­ment, Got­tlieb said the em­ploy­ees’ work has not gone un­no­ticed.

“We’ll sup­port them in any way we can in their ex­e­cu­tion of these vi­tal pub­lic health func­tions. To­ward these goals we’re adopt­ing new pro­ce­dures that’ll re­duce the like­li­hood that they ac­crue bal­ances on their gov­ern­ment trav­el cred­it cards for cost of air­fare and ho­tel/lodg­ing,” he added.

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.

Im­age: Scott Got­tlieb. SHUT­TER­STOCK


Zachary Brennan

managing editor, RAPS

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

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.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.