Aim­ing for more copy­cats of each brand­ed drug, FDA ex­pands gener­ic drug pri­or­i­ty re­views

Talk of bring­ing down the price of phar­ma­ceu­ti­cals of­ten hinges on gener­ic com­pe­ti­tion, and the US is see­ing ap­provals of new gener­ic drugs faster and more con­sis­tent­ly than ever — a trend like­ly to con­tin­ue.

The progress comes as FDA com­mis­sion­er Scott Got­tlieb on Thurs­day in­di­cat­ed that the agency will ex­pand which ab­bre­vi­at­ed new drug ap­pli­ca­tions (AN­DA) will see pri­or­i­ty re­views.

“Ear­li­er this year we made changes to how we pri­or­i­tize the agency’s gener­ic drug sub­mis­sions. The goal was to pri­or­i­tize the re­view of gener­ic ap­pli­ca­tions un­til the FDA has ap­proved three gener­ic ver­sions of each par­tic­u­lar drug,” Got­tlieb said in a state­ment. “To­day we’re ex­pand­ing this com­pe­ti­tion-fo­cused pol­i­cy to pri­or­i­tize any ap­pli­ca­tion that can meet the FDA’s ap­proval stan­dards at the point when the 180-day ex­clu­siv­i­ty pe­ri­od ex­pires on a first gener­ic en­trant to a brand­ed med­i­cine.”

The shift could ac­cel­er­ate gener­ic com­peti­tors to mar­ket more quick­ly and help bring down costs, and comes a day af­ter the Fed­er­al Trade Com­mis­sion held a work­shop on drug com­pe­ti­tion.


2017 saw the high­est num­ber of AN­DA ap­provals in one fis­cal year ever, and that fol­lowed a sim­i­lar­ly record-break­ing 2016.

But those record years of ap­provals do not seem to be slow­ing. A look at the FDA’s lat­est gener­ic drug re­view dash­board re­veals the num­ber of AN­DAs pend­ing re­view at the agency has still in­creased over the last 10 months.

As of 1 Jan­u­ary 2017, FDA’s gener­ic drug re­view dash­board not­ed that 2,358 ap­pli­ca­tions are still with FDA and ei­ther pend­ing fil­ing re­view, filed with no re­view com­mu­ni­ca­tions or have had at least one re­view com­mu­ni­ca­tion is­sued.

But there are now 140 more ap­pli­ca­tions pend­ing with FDA than there were to start the year, ac­cord­ing to the lat­est re­view dash­board as of 1 Oc­to­ber 2017. How­ev­er, the num­ber of ap­pli­ca­tions with in­dus­try (ei­ther pend­ing a re­sponse or re­ceiv­ing ten­ta­tive ap­proval) has fall­en over the same pe­ri­od by 116 ap­pli­ca­tions.

Robert Pol­lock

On whether there might be a new back­log form­ing at the agency, for­mer FDA deputy di­rec­tor of the Of­fice of Gener­ic Drugs Robert Pol­lock, who is now a se­nior ad­vis­er with Lach­man Con­sul­tants, told Fo­cus: “As long as the num­ber of re­ceipts is greater than the num­ber of ap­provals [the num­ber of AN­DAs with FDA] won’t change.”

“I do ex­pect the num­ber of ap­provals to rise mod­est­ly over the next two years and then if it does not dra­mat­i­cal­ly im­prove af­ter that, then some­thing is dras­ti­cal­ly wrong,” Pol­lock said.

Part of that in­crease of ap­pli­ca­tions await­ing FDA’s ac­tion is due to an uptick in re­quests dur­ing re­view for fur­ther in­for­ma­tion or clar­i­fi­ca­tions, though the num­ber of AN­DAs pend­ing fil­ing re­view de­clined by 40 ap­pli­ca­tions over those 10 months and the num­ber of AN­DAs filed with no re­view com­mu­ni­ca­tion fell by 100 AN­DAs in the same pe­ri­od.

As FDA hit its AN­DA back­log re­duc­tion in 2016 un­der the first Gener­ic Drug User Fee Amend­ments (GDU­FA), how the agency stays the course or shifts its work­load un­der the sec­ond it­er­a­tion of GDU­FA will be im­por­tant to track, es­pe­cial­ly with more pri­or­i­ty re­views for AN­DAs com­ing.

Im­age: Scott Got­tlieb in front of Con­gress ear­li­er this year AP Im­ages

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.


Zachary Brennan

managing editor, RAPS

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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Step­ping on Roche's toes, Mer­ck cuts in­to SCLC niche with third-line Keytru­da OK

In the in­creas­ing­ly crowd­ed check­point race, small cell lung can­cer has been a rare area where Roche, a sec­ond run­ner-up, has a lead over the en­trenched lead­ers Mer­ck and Bris­tol-My­ers Squibb. But Mer­ck is fi­nal­ly mak­ing some head­way in that di­rec­tion with the lat­est ap­proval for its PD-1 star.

The lat­est green light en­dors­es Keytru­da in the third-line treat­ment of metasta­t­ic SCLC, where it would be giv­en to pa­tients whose dis­ease ei­ther don’t re­spond to or re­lapse af­ter chemother­a­py, which would have fol­lowed at least one pri­or line of ther­a­py.

Image: Shutterstock

Gene ther­a­py R&D deals turn red hot as Big Phar­ma steps up to play

This September will mark the 20th anniversary of the death of Jesse Gelsinger, a young man suffering from X-linked genetic disease of the liver. He was killed in a gene therapy study conducted by Penn’s James Wilson, and the entire field endured a lengthy deep freeze as the field grappled with the safety issues inherent in the work.

Some thought gene therapy R&D would never survive. But it did. And this year marked a landmark approval for Zolgensma, a new gene therapy for spinal muscular atrophy Novartis priced at $2.1 million.

“Gene therapy is the hottest item on the block now. But there was a time when we first got into this trial, where there wasn’t a person in the world who believed that gene therapy would work. We have to remember that,” noted gene therapy investigator Jerry Mendell told SMA News Today.

We’re still right on the pioneering frontier when it comes to getting approvals for gene therapies and launching marketing campaigns with the European green light for bluebird's leading program last Friday underscoring the nascent nature of the field. But gene therapy R&D is booming, and has been for several years now.

The rapid growth of gene therapy clinical development is well known, but we decided to put some numbers on it, to quantify what’s going on. DealForma chief Chris Dokomajilar took a lot over the past 10 years, as the number of deals, R&D partnerships and buyouts steadily gained steam, spiking last year and on track to maintain the surge in 2019.

The upfronts and totals for the dollars on deals so far in 2019 is already close to the 2018 mark, underscoring a new phase of negotiations as the major players step up to gain a piece of the late-stage and commercial action.

Once again, we’re looking at an “overnight” biotech success story, decades in the making.

At some point, that may start to brake the numbers we’re seeing. But for now, as rivals line up to compete for frontline prominence across a range of diseases, the arrows are all pointed north.

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Albert Bourla appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

UP­DAT­ED: Pfiz­er CEO Al­bert Bourla is back in the M&A game, but why is he pay­ing $11.4B for Ar­ray?

Pfiz­er $PFE has cut short its time on the side­lines of bio­phar­ma M&A.

Mon­day morn­ing the phar­ma gi­ant un­veiled an $11.4 bil­lion deal to ac­quire Ar­ray Bio­Phar­ma, beef­ing up its on­col­o­gy work and adding a new re­search hub in Boul­der, Col­orado to its glob­al op­er­a­tions.

At $48 a share, Ar­ray $AR­RY in­vestors will be get­ting a 62% pre­mi­um off the Fri­day close of $29.59.

Pfiz­er, which has strug­gled to gain all the up­side promised in past buy­outs like Medi­va­tion, high­light­ed the ac­qui­si­tion of 2 ap­proved drugs in the deal — Braftovi (en­co­rafenib) and Mek­tovi (binime­tinib).

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Right back at you, Pfiz­er: BeiGene and a Pfiz­er spin­out launch a new­co to de­vel­op a MEK/BRAF in­hibitor that could ri­val $11.4B com­bo

A day af­ter Pfiz­er bought Ar­ray and its ap­proved can­cer com­bo, BeiGene and Pfiz­er spin­out Spring­Works have part­nered in launch­ing a new biotech that has an eye on the very same mar­ket the phar­ma gi­ant just paid bil­lions for. And they’re plan­ning on us­ing an ex-Pfiz­er drug to do it.

In a nut­shell, Chi­na’s BeiGene is toss­ing in a pre­clin­i­cal BRAF in­hibitor — BGB-3245, which cov­ers both V600 and non-V600 BRAF mu­ta­tions — for a big stake in a new, joint­ly con­trolled biotech called Map­Kure with Bain-backed Spring­Works.

Sanofi aligns it­self with Google to stream­line drug de­vel­op­ment

Tech­nol­o­gy is bleed­ing in­to health­care, and big phar­ma is rid­ing the wave. Sanofi $SNY ap­point­ed its first chief dig­i­tal of­fi­cer this Feb­ru­ary, fol­low­ing the foot­steps of its peers. By May, the French drug­mak­er and some of its big phar­ma com­pa­tri­ots joined forces with Google par­ent Al­pha­bet’s Ver­i­ly unit to aug­ment clin­i­cal tri­al re­search. On Tues­day, the Parisian com­pa­ny tied up with Google to ac­cess its cloud com­put­ing and ar­ti­fi­cial in­tel­li­gence tech to spur the de­vel­op­ment of new ther­a­pies.

Af­ter watch­ing its share price soar on a Bloomberg re­port and heat­ed ru­mors, Bio­haven stock takes a bil­lion-dol­lar bath

Back in April, Biohaven Pharmaceutical became one hot biotech stock $BHVN based on a report in Bloomberg that some “potential bidders” had been kicking the tires at the biotech, which has a lead drug for migraines. Then the rumor mill really started to smoke when execs canceled a presentation at an investor conference a little more than a week ago.

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UP­DAT­ED: Roche fields first ap­proval for Ro­z­lytrek in the run-up to a show­down with Bay­er, Pfiz­er

While it’s wait­ing to hear back from FDA reg­u­la­tors, Roche is be­gin­ning the vic­to­ry lap for en­trec­tinib in Japan.

Roche is giv­ing Bay­er a run for their mon­ey with this tu­mor-ag­nos­tic drug, which tar­gets NTRK gene fu­sions. Now dubbed Ro­z­lytrek, it’s sanc­tioned to treat adult and pe­di­atric pa­tients in Japan with neu­rotroph­ic ty­ro­sine re­cep­tor ki­nase fu­sion-pos­i­tive, ad­vanced re­cur­rent sol­id tu­mors.

A uni­corn stalks Wall Street in search of IPO cash; CASI Phar­ma in-li­cens­es CD19 ther­a­py from Chi­na’s Ju­ven­tas

→ A herd of up­start biotechs will look to Wall Street for some ma­jor wind­falls this week as a burst of new of­fer­ings con­tin­ues to feed cash in­to the R&D sys­tem. To­day we learned that Bridge­Bio will look to raise in the neigh­bor­hood of $225 mil­lion by of­fer­ing 15 mil­lion shares for $14 to $16 each. And they have a string of joint bookrun­ners: J.P. Mor­gan, Gold­man Sachs, Jef­feries, SVB Leerink, KKR, Piper Jaf­fray, Mizuho Se­cu­ri­ties, BMO Cap­i­tal Mar­kets and Ray­mond James. If suc­cess­ful, Bridge­Bio will emerge with a mar­ket cap of around $1.7 bil­lion. There are 5 biotechs look­ing to IPO this week, in­clud­ing Akero and Pre­vail.