12 block­busters: The surg­ing list of $1B-plus drugs rolling out on the mar­ket this year might sur­prise you

What ex­act­ly qual­i­fies as a suc­cess in drug R&D may be dis­cussed and dis­put­ed in many ways, but noth­ing lays an ar­gu­ment to rest quite as de­ci­sive­ly as the re­al pos­si­bil­i­ty of block­buster sta­tus.

The da­ta an­a­lysts at Clar­i­vate An­a­lyt­ics have just as­sem­bled their an­nu­al list of all the drugs that are rolling out on­to the mar­ket this year with a sol­id shot at break­ing the one bil­lion-dol­lar an­nu­al bar­ri­er by 2022, and their ros­ter — 12 like­ly block­busters which I’ve de­tailed be­low — pro­vides some in­ter­est­ing in­sights in­to the state of drug R&D to­day.

Richard Har­ri­son

First, it’s a re­mark­ably eclec­tic col­lec­tion of ther­a­pies, with sev­er­al built on the 46 new ap­provals the FDA post­ed in 2017 — a record 52 if you in­clude some re­mark­able new bi­o­log­ics. At a time when on­col­o­gy cap­tures the largest sin­gle share of the mon­ey be­ing in­vest­ed in the field, on­ly 1 of the 12 is for treat­ing can­cer. Two are for di­a­betes, and the rest are scat­tered across 9 dif­fer­ent dis­ease ar­eas.

“I like the fact that no one thing dom­i­nates,” says Richard Har­ri­son, the CSO at Clar­i­vate. “It tells me that the in­dus­try is look­ing at a lot more in­di­ca­tions.”

Not on­ly is there an ar­ray of dis­eases rep­re­sent­ed on the list, there are some new play­ers mak­ing their ap­pear­ance for the first time — Al­ny­lam and GW Phar­ma­ceu­ti­cals — high­light­ing how years of in­vest­ment in biotech has be­gun to pay off in re­mark­able ways and with new com­mer­cial op­er­a­tions. An­oth­er small play­er, In­di­v­ior, al­so made the list with the first month­ly dose of buprenor­phine.

The drug R&D in­dus­try went through quite a stretch of low pro­duc­tiv­i­ty over the last decade, Har­ri­son notes. But a com­bi­na­tion of fac­tors — bet­ter clin­i­cal strate­gies, bio­mark­ers and so on — have ul­ti­mate­ly con­spired to cre­ate greater ef­fi­cien­cies at dis­cov­er­ing drugs. And that’s why Clar­i­vate is post­ing the longest list of this kind since they start­ed in 2013.

It’s al­ways hard to pre­dict the fu­ture, Har­ri­son added, but he would like to see the surge con­tin­ue in 2019. All the trends he can point to in 2018 all seem to have some durable fea­tures that should last for some time to come.

And with that, here are the 12.

  1. Hem­li­bra (emi­cizum­ab)

De­vel­op­er: Roche
Dis­ease: He­mo­phil­ia
2022 pro­ject­ed sales: $4 bil­lion

The scoop: It’s no sur­prise to find Hem­li­bra at the top of the list. Roche’s land­mark suc­cess for this drug has every­one in the he­mo­phil­ia mar­ket look­ing at a tec­ton­ic shift in mar­ket share. Hem­li­bra helped ease con­cerns about Roche’s abil­i­ty to roll with the loss of patent pro­tec­tion on three big fran­chise drugs. And it’s like­ly to be the biggest longterm suc­cess on this list, by far. That’s not ex­act­ly what ri­vals at Shire or No­vo Nordisk want to hear. But this is a ma­jor ad­vance for pa­tients, and the first big thing to come along in 20 years in he­mo­phil­ia. There may be big things ahead for this drug, but for now Roche is lead­ing the way.


2. Bik­tarvy (bicte­gravir/emtric­itabine/teno­fovir alafe­namide)

De­vel­op­er: Gilead
Dis­ease: HIV
2022 pro­ject­ed sales: $3.71 bil­lion

The scoop: To be sure, Gilead has some en­er­gized com­pe­ti­tion at GSK’s ma­jor­i­ty owned Vi­iV. Nev­er­the­less, Gilead is build­ing on one of the most durable fran­chis­es in drug de­vel­op­ment, as­sured of a block­buster mar­ket for a sin­gle ther­a­py that will make life eas­i­er for many peo­ple liv­ing with HIV. One day, there may be a cure for the virus. But un­til then, no one knows how to work this field bet­ter than Gilead.


3. Ozem­pic (semaglu­tide)

De­vel­op­er: No­vo Nordisk
Dis­ease: Di­a­betes (stud­ied for obe­si­ty)
2022 pro­ject­ed sales: $3.47 bil­lion

The scoop: You have to give No­vo Nordisk top cred­it for run­ning a savvy de­vel­op­ment pro­gram. They are go­ing af­ter Eli Lil­ly’s Trulic­i­ty, and they came up with the da­ta to prove their drug was bet­ter. And they al­so have some re­mark­able stats on weight loss that in­spired a move in­to the clin­ic for obe­si­ty, with the kind of safe­ty da­ta in hand that would make any added des­ig­na­tion on that front a like­ly block­buster all on its own. Di­a­betes is a rugged­ly com­pet­i­tive field, with a few gi­ants dom­i­nat­ed the land­scape. In that re­spect, No­vo con­tin­ues to punch well above its weight.


4. Er­lea­da (apa­lu­tamide)

De­vel­op­er: J&J
Dis­ease: Non-metasta­t­ic prostate can­cer
2022 pro­ject­ed sales: $2 bil­lion

The scoop: Here again you can see how the biotech ecosys­tem is pay­ing div­i­dends for Big Phar­ma. J&J had some nasty set­backs in 2017, but this drug snagged in its Aragon ac­qui­si­tion looks ready to pay off at a crit­i­cal junc­ture. J&J is fac­ing the near-term loss of patent pro­tec­tion on Zyti­ga, which is sold for metasta­t­ic prostate can­cer. The ap­proval in Feb­ru­ary, months ahead of the nor­mal reg­u­la­to­ry sched­ule, al­so un­der­scores the FDA’s will­ing­ness to run out the green light in record time, par­tic­u­lar­ly in on­col­o­gy.


5. Shin­grix

De­vel­op­er: Glax­o­SmithK­line
Dis­ease: Shin­gles
2022 pro­ject­ed sales: $1.37 bil­lion

The scoop: GSK doesn’t make these lists for its phar­ma prod­ucts. But it still has a vi­brant vac­cines group. New CEO Em­ma Walm­s­ley, though, seems fed up with its chron­ic sec­ond place rank­ing in drug de­vel­op­ment, and in­tends to make some things change as the com­pa­ny fo­cus­es on few­er, but big­ger, new drugs. We’ll see.


6. Patisir­an

De­vel­op­er: Al­ny­lam
Dis­ease: Herid­i­tary TTR Amy­loi­do­sis
2022 pro­ject­ed sales: $1.21 bil­lion

The scoop: Al­ny­lam has some com­pe­ti­tion at Io­n­is, but when an­a­lysts start count­ing the dol­lars, vir­tu­al­ly all of them as­sign the li­on’s share in the field to Al­ny­lam. This is lin­ing up as the Cam­bridge, MA-based biotech’s first ap­proval, and it’s a big one. If this pays off as ex­pect­ed, and some of the peak sales es­ti­mates go much high­er, Al­ny­lam can sus­tain its ground­break­ing RNAi plat­form for some time. It’s an im­pres­sive achieve­ment, no mat­ter how you cut it.


7. Epid­i­olex

De­vel­op­er: GW Phar­ma­ceu­ti­cals
Dis­ease: Dravet syn­drome and Lennox-Gas­taut syn­drome
2022 pro­ject­ed sales: $1.19 bil­lion

The scoop: Per­haps it’s not too sur­pris­ing that a cannabis-based ther­a­py can re­duce the rate of seizures for two rare syn­dromes. But GW has im­pressed an­a­lysts with a set of late-stage re­sults that de­ci­sive­ly makes their case. The first PDU­FA date is loom­ing June 27, and the biotech is the odds-on fa­vorite for bring­ing the block­buster.


8. Aimovig (erenum­ab)

De­vel­op­er: Am­gen/No­var­tis
Dis­ease: Mi­graine
2022 pro­ject­ed sales: $1.17 bil­lion

The scoop: Am­gen and No­var­tis don’t have the on­ly CGRP mi­graine drug head­ed to a like­ly ap­proval. The da­ta on these drugs aren’t dra­mat­i­cal­ly dif­fer­ent. Safe­ty looks good. And play­ers rang­ing from Te­va to Eli Lil­ly to lit­tle Alder (and lat­er Al­ler­gen) are all an­gling for their own piece of the pie. But the two heavy­weights do have the first drug un­der re­view at the FDA. Way out front, they are like­ly to be the first to start chang­ing the stan­dard of care in the field. That’s a big deal, and one they promise to make the most of.


9. Lanadelum­ab

De­vel­op­er: Shire
Dis­ease: Hered­i­tary an­gioede­ma
2022 pro­ject­ed sales: $1.15 bil­lion

The scoop: Back in May, when Shire post­ed the da­ta on lanadelum­ab, it was quick­ly ap­par­ent that Shire’s pipeline star had po­ten­tial stel­lar fu­ture ahead of it. The FDA sig­naled their agree­ment with a quick re­view sched­ule. Shire CEO Flem­ming Orn­skov likes to set stretch goals for the com­pa­ny, and he’s done it here as well, pro­ject­ing $2 bil­lion in peak sales. Even if he doesn’t hit that mark, though, it seems like he’d be hard pressed to miss block­buster sta­tus.


10. Elagolix

De­vel­op­er: Ab­b­Vie
Dis­ease: En­dometrio­sis
2022 pro­ject­ed sales: $1.15 bil­lion

The scoop: Ab­b­Vie would seem to have an ap­proval to mar­ket this drug for en­dometrio­sis al­most in the bag, with sol­id da­ta and an ac­cel­er­at­ed time­line at the FDA that seems to al­most al­ways bode well for de­vel­op­ers. More re­cent­ly, Ab­b­Vie added a full slate of pos­i­tive da­ta from two Phase III stud­ies for uter­ine fi­broids, in­di­cat­ing that broad­er and bet­ter things lie ahead for this new fran­chise ther­a­py. Clar­i­vate’s num­ber here is right in line with mar­ket con­sen­sus, but Ge­of­frey Porges is cheer­ing things along with a $1.4 bil­lion pro­jec­tion. The com­pa­ny got this drug in a $575 mil­lion deal it struck in 2010 with Neu­ro­crine $NBIX, which stands to earn a roy­al­ty pay­out on an ap­proval.


11. Steglatro (er­tugliflozin)

De­vel­op­er: Pfiz­er/Mer­ck
Dis­ease: Di­a­betes
2022 pro­ject­ed sales: $1.09 bil­lion

The scoop: Pfiz­er and Mer­ck are late to the SGLT2 par­ty. Sev­er­al years late, to be ex­act. Eli Lil­ly got out front with Jar­diance and a full set of ri­vals fol­lowed in their paths. Nev­er­the­less, di­a­betes is a mas­sive and grow­ing mar­ket, leav­ing a new ar­rival like this still with­in reach of a block­buster re­turn. Steglatro isn’t win­ning awards for in­no­va­tion, but it’s help­ing pa­tients and of­fer­ing some com­pe­ti­tion in an in­tense­ly com­pet­i­tive are­na. That’s worth some re­ward.


12. Sublo­cade (Once-month­ly buprenor­phine)

De­vel­op­er: In­di­v­ior
Dis­ease: Opi­oid de­pen­dence
2022 pro­ject­ed sales: $1.07 bil­lion

The scoop: In­di­v­ior need­ed this drug ap­proval, bad­ly. With gener­ic ri­vals about to crowd in on its stan­dard treat­ment, a month­ly in­jectable of buprenor­phine — us­ing a mild opi­oid — is a wel­come ad­di­tion to the field of eas­ing opi­oid with­draw­al — one of the hottest is­sues of the day. The FDA in­di­cat­ed that it would help, and with the agency lean­ing in fa­vor of the in­dus­try like nev­er be­fore, that’s mon­ey in the bank.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Months af­ter FDA re­jec­tion, Sanofi touts piv­otal win for rare dis­ease drug su­tim­limab as it preps to re­file

One of the pillar drugs of Sanofi’s $11.6 billion pickup of Bioverativ hit a big setback late last year when the FDA sent its application for approval back. Now, as Sanofi gears up to resubmit the drug for review, the drugmaker is touting pivotal data it hopes will help take it over the finish line.

Sanofi’s sutimlimab nailed all three of its primary endpoints in its Phase III CADENZA study for patients with cold agglutinin disease, a rare disorder that can cause severe anemia, without a recent history of blood transfusion, the French drugmaker said Friday. The topline results will be presented at this weekend’s virtual EHA meeting.

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Ver­tex and CRISPR Ther­a­peu­tics un­veil more pos­i­tive gene ther­a­py da­ta, but busul­fan again casts a shad­ow over the field

Less than 12 hours after revealing a flop on its second shot for alpha-1 antitrypsin deficiency, Vertex plowed ahead with another data drop from its partnership with CRISPR Therapeutics. And though the topline proved positive, concerns over conditioning agents continue to linger over the collaboration, as well as the entire gene therapy space.

Presenting data from two trials at the European Hematology Association annual meeting, the pair announced that follow-up data of at least three months for 22 patients with genetic blood disorders indicated a “consistent and sustained” response to the experimental drug CTX001. All 15 patients with transfusion-dependent beta thalassemia did not need further blood transfusions and all seven with severe sickle cell disease were pain free, the biotechs announced.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.