Trump will sign FDA user fee reau­tho­riza­tion bill, end­ing threat of agency lay­offs

Ahead of its sum­mer re­cess, the US Sen­ate to­day fol­lowed its House coun­ter­parts and passed a bi­par­ti­san bill to reau­tho­rize FDA user fee pro­grams for pre­scrip­tion drugs, gener­ic drugs, med­ical de­vices and biosim­i­lars for the next five years. A spokesman for Pres­i­dent Don­ald Trump told Fo­cus, “The Pres­i­dent will sign this bill, but we look for­ward to work­ing with Con­gress to ad­dress fur­ther ne­go­ti­a­tions.”

The Sen­ate’s pas­sage of the House bill by a vote of 94-1 — Bernie Sanders (D-VT) was the on­ly vote against — comes more than two years af­ter ne­go­ti­a­tions on the reau­tho­riza­tion be­gan. The bill would al­low FDA to col­lect in­dus­try user fees through 2022 to help pay for time­ly re­views of new med­ical prod­ucts, in some cas­es ex­pe­dite the re­views of cer­tain prod­ucts and to dis­close its progress in meet­ing new goals.

In­dus­try groups praised the Sen­ate’s pas­sage of the bill, though the Pro­ject on Gov­ern­ment Over­sight crit­i­cized the cozy re­la­tion­ship be­tween FDA and in­dus­try, as well as one of the bill’s pro­vi­sions that “could pro­vide pa­tients and reg­u­la­tors with less de­tailed and time­ly warn­ings about po­ten­tial­ly dead­ly haz­ards as­so­ci­at­ed with med­ical de­vices.”

Be­low is a break­down of some of the high­lights from each of the user fee reau­tho­riza­tions and oth­er pro­vi­sions.


The sixth it­er­a­tion of the Pre­scrip­tion Drug User Fee Act (PDU­FA VI) fo­cus­es on pre-mar­ket re­views, post­mar­ket safe­ty, reg­u­la­to­ry de­ci­sion tools and oth­er ways FDA is prepar­ing for the fu­ture of drug de­vel­op­ment.

In ad­di­tion, the agree­ment seeks to fur­ther in­cor­po­rate the pa­tient voice in­to drug de­vel­op­ment, en­hance the use of re­al-world ev­i­dence, im­prove ben­e­fit-risk as­sess­ments and ex­pand post­mar­ket safe­ty da­ta and eval­u­a­tions.


The sec­ond it­er­a­tion of the Gener­ic Drug User Fee Amend­ments (GUD­FA II), which cre­ates a new user fee struc­ture and aims to help small busi­ness­es, al­so will speed the re­view of gener­ic drugs and in­crease in­ter­ac­tions be­tween the agency and gener­ics de­vel­op­ers work­ing on com­plex gener­ics.


The fourth it­er­a­tion of the Med­ical De­vice User Fee Act (MD­U­FA IV) would in­crease user fees for de­vice man­u­fac­tur­ers by more than $320 mil­lion over the next five years in ex­change for re­duc­ing the av­er­age to­tal time to de­ci­sions for pre­mar­ket ap­pli­ca­tions and 510(k) sub­mis­sions, among oth­er pro­vi­sions.


The sec­ond it­er­a­tion of the Biosim­i­lar User Fee Act (Bs­U­FA II) would al­low for ad­di­tion­al com­mu­ni­ca­tion be­tween FDA re­view teams and biosim­i­lar ap­pli­cants in the form of pre­sub­mis­sion meet­ings, mid-cy­cle com­mu­ni­ca­tions and late-cy­cle meet­ings, while al­so adding 60 days to the re­view time­frame to ac­com­mo­date for ad­di­tion­al in­ter­ac­tions. In ad­di­tion, the next Bs­U­FA would al­low, in cer­tain cir­cum­stances, spon­sors to re­quest a writ­ten re­sponse to ques­tions rather than con­duct a face-to-face or tele­con­fer­ence meet­ing.

Oth­er Pro­vi­sions

The bill al­so re­quires that FDA an­nu­al­ly pub­lish in­for­ma­tion re­gard­ing the pre­vi­ous year’s in­spec­tions of drug or med­ical de­vice fa­cil­i­ties, in­clud­ing the amount of time be­tween steps in the in­spec­tion process.

In ad­di­tion, the bill re­vis­es re­quire­ments for per­for­mance re­ports un­der all the user fee pro­grams to in­clude re­quir­ing quar­ter­ly pub­li­ca­tion of in­for­ma­tion re­gard­ing guid­ance and meet­ings.

Among the amend­ments adopt­ed by the House En­er­gy and Com­merce Com­mit­tee were pro­pos­als for risk-based clas­si­fi­ca­tion for med­ical de­vice ac­ces­sorieseas­ing re­quire­ments for med­ical imag­ing de­vices and con­trast agentsser­vic­ing and main­te­nance of med­ical de­vicesa pi­lot project for ac­tive sur­veil­lance of med­ical de­vices and a man­ag­er’s amend­ment that pro­vides a num­ber of tech­ni­cal changes and strength­ens the la­bel­ing re­quire­ments for over-the-counter hear­ing aids un­der the reau­tho­riza­tion bill.

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 for more in­for­ma­tion.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

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Take­da to pull key hy­poparathy­roidism drug from the mar­ket en­tire­ly by end of 2024 af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024.

The decision to not re-commercialize Natpara will be a blow to not only the 2,400 people who were awaiting supplies of their reliable injection since 2019, but also the additional nearly 400 people who were accessing the drugs via the company’s Special Use Program as Takeda sought to resolve these manufacturing issues over the past five years.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Astel­las, Pan­th­er­na add or­gan to mR­NA tie-up; Rock­et launch­es sale of six fig­ures worth of stock

Astellas and Pantherna have expanded their November 2021 pact surrounding the latter’s mRNA platform to include a new target organ, the duo announced Tuesday morning, though they did not specify what that target is.

German biotech Pantherna is home to two platform technologies — one that designs mRNAs for non-vaccine therapies and another that designs LNPs. Astellas and Pantherna’s deal appears to mainly revolve around the first platform, which Astellas said it is using to research direct reprogramming, or turning cells from one kind into another without an intermediate stem cell phase.