The top 10 or­phan drugs in the late-stage pipeline

Or­phan drugs have come in­to the spot­light, scar­ing up head­lines in­volv­ing big prices and small pa­tient pop­u­la­tions. Some com­pa­nies have been ac­cused of prof­i­teer­ing by ma­nip­u­lat­ing the FDA’s rules on this — hel­lo, Marathon — but or­phan ther­a­pies re­main a very close­ly watched are­na with some ma­jor new prospects com­ing through the late-stage pipeline. And some of these drugs are of­fer­ing pa­tients re­al hope for the first time.

Every year, Eval­u­ate crunch­es the num­bers on the top prospects in the pipeline, and the com­pa­ny’s ed­i­to­r­i­al arm EP Van­tage in­cludes it in their an­nu­al re­port on or­phan drugs. I’ve picked up their list and re­arranged it by Eval­u­ate’s sales es­ti­mates for 2022.

A word of cau­tion. Sales es­ti­mates are easy to make and hard to de­liv­er on, so it’s al­ways im­por­tant to take these num­bers with a grain of salt. Al­so, some of these drugs still face big ques­tions as they come un­der FDA re­view. And the FDA re­view process it­self may well change rad­i­cal­ly, es­pe­cial­ly for or­phan drugs, as Pres­i­dent Trump de­mands faster re­sponse times on this front.

It’s still ear­ly to be count­ing chick­ens, but these eggs de­serve care­ful at­ten­tion.

Tesaro: ni­ra­parib, PARP/can­cer
2022 sales: $1.8B

Two years made a big dif­fer­ence for Tesaro $TSRO. In 2015, Eval­u­ate fore­cast­ed this drug would bring in $499 mil­lion in 2020. Now un­der re­view as the biotech ex­plores a broad la­bel, an­a­lysts have been hik­ing their pro­jec­tions, which keeps this com­pa­ny on most an­a­lysts’ lists of like­ly takeover tar­gets. Ovar­i­an can­cer qual­i­fied this drug for or­phan sta­tus.

Kite Phar­ma: ax­i­ab­ta­gene ciloleu­cel, CAR-T/can­cer
2022 sales: $1.7B

Kite’s new name for KTE-C19, its top CAR-T in the pipeline, is a tongue twister, but af­ter the biotech un­veiled 6-month da­ta on Tues­day, we should all learn how to pro­nounce it. The first clear ev­i­dence of dura­bil­i­ty leaves Kite $KITE in an im­pres­sive po­si­tion as it looks to beat out No­var­tis with the first gen­er­a­tion of CAR-Ts. It should be quite a ri­val­ry.

Roche: emi­cizum­ab (ACE910)/ he­mo­phil­ia
2022 sales: $1.5B

Maybe we shouldn’t get too far ahead of our­selves on this one. Yes, Roche $RHH­BY has very high ex­pec­ta­tions for this drug. But it’s al­so been plagued by safe­ty ques­tions, which have yet to be ful­ly aired by reg­u­la­tors. A top prospect? You bet. But don’t count on the mon­ey yet.

As­traZeneca: treme­li­mum­ab/CT­LA-4 check­point in­hibitor
2022 sales:  $1.3B

Usu­al­ly men­tioned right along­side dur­val­um­ab, As­traZeneca’s $AZN oth­er check­point, the treme­li­mum­ab com­bo emerges as an ab­solute­ly cen­tral fea­ture in dis­tin­guish­ing the phar­ma gi­ant as it tries to leapfrog some very ad­vanced play­ers like Mer­ck, Bris­tol-My­ers and Squibb. This one is do or die for As­traZeneca, which won or­phan sta­tus for ma­lig­nant mesothe­lioma.

Shire: lanadelum­ab (SHP643)/hered­i­tary an­gioede­ma 
2022 sales:  $1.1B

This was the jew­el in the crown for Shire {SH­PG} when it paid $5.9 bil­lion for Dyax back in late 2015. The drug blocks en­zyme plas­ma kallikrein to treat hered­i­tary an­gioede­ma, and Shire ex­ecs have al­ready in­di­cat­ed that they be­lieve it can reach $2 bil­lion in an­nu­al sales. And last sum­mer this drug fig­ured promi­nent­ly among the top three pro­grams fea­tured by CEO Flem­ming Orn­skov. It has a break­through drug des­ig­na­tion and a shot at glo­ry.

Te­va: SD-809/tar­dive dysk­i­ne­sia
2022 sales:  $1B

Te­va $TE­VA al­ready has had to deal with a se­ri­ous set­back on this drug when the FDA re­ject­ed its ap­pli­ca­tion for Hunt­ing­ton’s chorea last year. But it’s still shoot­ing for a block­buster with a new dri­ve on to get this ap­proved this year for tar­dive dysk­i­ne­sia. A seem­ing­ly per­ma­nent­ly trou­bled com­pa­ny, Te­va needs this win bad­ly. Te­va’s big pro­gram for this drug puts it in a head-to-head show­down with Neu­ro­crine $NBIX, which not­ed re­cent­ly that it filed for an ap­proval on the ri­val drug val­be­nazine. And some an­a­lysts would put Neu­ro­crine’s drug well ahead of Te­va’s on this one.

blue­bird bio: Lenti­Glo­bin/gene ther­a­py
2022 sales:  $969M

Blue­bird $BLUE helped make gene ther­a­py a hot field again, and its strug­gles with vary­ing de­grees of im­pact on pa­tients has helped raise fresh ques­tions about where it’s head­ed on be­ta tha­lassemia, where in­ves­ti­ga­tors are now pur­su­ing a Phase III tri­al. Po­ten­tial? You bet. But there are still plen­ty of unan­swered ques­tions.

As­traZeneca: acal­abru­ti­nib (ACP-196) /BTK in­hibitor 
2022 sales:  $968M

As­traZeneca won’t mind be­ing the on­ly com­pa­ny to make two ap­pear­ances on this top 10 list. This drug came its way when it ac­quired a ma­jor­i­ty in­ter­est in Ac­er­ta in 2015, as Pas­cal So­ri­ot sought to ex­e­cute a ma­jor turn­around in the phar­ma gi­ant’s prospects. With gener­ic com­pe­ti­tion eat­ing away at rev­enue, As­traZeneca needs all the help it can get from its on­col­o­gy pipeline. This drug is in late-stage stud­ies for B-cell blood can­cers.

No­var­tis: CTL019/CAR-T
2022 sales: $917M

This is the “oth­er” CAR-T you prob­a­bly heard about. Af­ter No­var­tis ripped up its cell ther­a­py unit last year, the phar­ma gi­ant went to some trou­ble to try and re­as­sure ob­servers that it was still on track to file for an ap­proval in ear­ly 2017. But an­a­lysts are dis­count­ing its im­pact com­pared to Kite, which re­mains the leader in the field, re­gard­less of who gets to the FDA first.

Ab­b­Vie: veli­parib/PARP in­hibitor
2022 sales: $854M

PARPs are all the rage these days, es­pe­cial­ly since Pfiz­er picked up ta­la­zoparib in its $14 bil­lion Medi­va­tion buy­out. Ab­b­Vie, though, re­mains well be­hind what is be­com­ing a fair­ly crowd­ed field. That leaves them on the list for now, but just bare­ly, as all eyes re­main on Tesaro.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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