The new top 10 most ex­pen­sive drugs on the plan­et

Bio­Marin’s de­ci­sion last night to set the whole­sale ac­qui­si­tion cost of its new ul­tra rare dis­ease drug Brineu­ra for CLN2 at $702,000 — with a sig­nif­i­cant­ly low­er net amount — cre­ates a new en­try in the list of the 10 most ex­pen­sive ther­a­pies in the world. With uniQure’s gene ther­a­py Gly­bera un­used and head­ing for the shelves, we al­so de­cid­ed to drop that, leav­ing Hori­zon in the top slot with its WAC price for Hori­zon’s Rav­ic­ti, at *$793,632, ac­cord­ing to the orig­i­nal line­up from Rein­sur­ance Group of Amer­i­ca. Brineu­ra is the new num­ber 2. We’ll al­so note that you could ar­gue for a place on this list for Bio­gen’s Spin­raza, which starts with a $750,000 price for its first year, but then drops by half, falling be­low the new top 10.

 

Whole­sale Ac­qui­si­tion Costs (List Price)

 


1. Rav­ic­ti $793,632

Com­pa­ny: Hori­zon Phar­ma
Cat­e­go­ry: Rare dis­ease – or­phan drug
(glyc­erol phenyl­bu­tyrate)

Ap­proved ini­tial­ly for Hy­pe­r­i­on in 2013 as a treat­ment for urea cy­cle dis­or­ders, Hori­zon saw the po­ten­tial and ac­quired the com­pa­ny for about $1.1 bil­lion, boost­ing its port­fo­lio of rare dis­ease drugs. (*This is the same price cit­ed by AHIP in their pric­ing re­port, which cites WAC and AWP pric­ing da­ta pub­lished in the Fed­er­al Sup­ply Sched­ule and the RED­BOOK, as of Sept. 30, 2015. A spokesman for Hori­zon dis­putes this num­ber, say­ing the WAC is clos­er to $560,000.)


2. Brineu­ra $702,000

Com­pa­ny: Bio­Marin
Cat­e­go­ry: Rare dis­ease – or­phan drug
(cer­liponase al­fa)

The drug treats ul­tra rare cas­es — 20 new di­ag­noses a year — of Neu­ronal Ceroid Lipo­fus­ci­nosis Type 2 dis­ease (CLN2), a form of Bat­ten dis­ease. The dis­ease pro­gress­es rapid­ly, with re­cur­rent seizures and vi­sion loss oc­cur­ring as most vic­tims are quick­ly left un­able to walk by the age of six be­fore even­tu­al­ly killing them. The drug was test­ed in on­ly 22 pa­tients in an ab­bre­vi­at­ed study, with a com­par­i­son on the nat­ur­al his­to­ry of sev­er­al dozen oth­ers.


3. Lu­mizyme $626,400

Com­pa­ny: Sanofi – Gen­zyme
Cat­e­go­ry: Rare dis­ease – or­phan drug
(al­glu­cosi­dase al­fa)

This drug for Pompe’s dis­ease helped make the rare dis­ease field pop­u­lar in bio­phar­ma. Ap­proved in 2010, Sanofi liked the pro­file at Gen­zyme and bought out the com­pa­ny for about $20 bil­lion a year lat­er. The FDA hand­ed out their or­phan drug des­ig­na­tion for this drug in 1997.


4. Carbaglu $585,408

Com­pa­ny: Recor­dati
Cat­e­go­ry: Rare dis­ease – or­phan drug
(car­g­lu­mic acid)

This drug treats rare cas­es of N-acetyl­glu­ta­mate syn­thase de­fi­cien­cy. Al­so ap­proved in 2010, there are on­ly a hand­ful of cas­es of NAGs dis­ease every year. The con­di­tion is char­ac­ter­ized by hy­per­am­mone­mia, en­cephalopa­thy, and res­pi­ra­to­ry al­ka­lo­sis and has fre­quent­ly led to the swift deaths of new­borns.


5. Ac­tim­mune  $572,292

Com­pa­ny: Hori­zon
Cat­e­go­ry: Rare dis­ease – or­phan drug
(In­ter­fer­on gam­ma 1-b)

Ap­proved for se­vere, ma­lig­nant os­teopet­ro­sis and chron­ic gran­u­lo­ma­tous dis­ease, rare ge­net­ic dis­eases, Ac­tim­mune not­ed last last year that the drug failed a Phase III for Friedre­ich’s atax­ia. Hori­zon ob­tained a sep­a­rate or­phan drug des­ig­na­tion for the drug when it de­cid­ed to mount the study.


6. Soliris $542,640

Com­pa­ny: Alex­ion
Cat­e­go­ry: Rare dis­eases – or­phan drug
(eculizum­ab)

This is the drug that built Alex­ion, and its suc­cess has helped in­spire some new ri­vals to see if they can do it one bet­ter in parox­ys­mal noc­tur­nal he­mo­glo­bin­uria and atyp­i­cal he­molyt­ic ure­mic syn­drome. It’s al­so un­der re­view now for re­frac­to­ry gen­er­al­ized myas­the­nia gravis, which earned the com­pa­ny an­oth­er or­phan drug des­ig­na­tion.


7. Al­pro­lix $503,880

Com­pa­ny: Biover­a­tiv
Cat­e­go­ry: Rare dis­ease – or­phan drug
(Co­ag­u­la­tion Fac­tor IX [Re­com­bi­nant], Fc Fu­sion Pro­tein)

This long-act­ing he­mo­phil­ia B med­i­cine was re­cent­ly spun out — along with Eolctate — from Bio­gen and So­bi in­to Biover­a­tiv, which start­ed out life with hun­dreds of mil­lions in cash and plans to build a rare dis­ease drug pipeline. Plen­ty of new drugs are in the works now that could rev­o­lu­tion­ize this field.


8. Idelvion $500,000

Com­pa­ny: CSL Behring
Cat­e­go­ry: Rare dis­ease — or­phan drug
(al­butrepenonacog al­fa)

Ap­proved last year at the FDA, Idelvion is an­oth­er long-act­ing he­mo­phil­ia B drug that is the first in its class to in­clude the blood pro­tein al­bu­min. The drug is de­signed to re­place Fac­tor IX, a nat­u­ral­ly oc­cur­ring clot­ting fac­tor miss­ing in he­mo­phil­ia B pa­tients.


9. Naglazyme $485,747

Com­pa­ny: Bio­marin
Cat­e­go­ry: Rare dis­ease – or­phan drug
(gal­sul­fase)

Naglazyme was orig­i­nal­ly ap­proved back in 2005 for mu­copolysac­cha­ri­do­sis type VI (MPS VI). On­ly a few dozen pa­tients are pre­scribed this en­zyme re­place­ment drug in the US every year.


10. Folo­tyn $450,540

Com­pa­ny: Spec­trum Phar­ma­ceu­ti­cals
Cat­e­go­ry: Rare dis­ease – or­phan drug
(prala­trex­ate)

This drug first be­gan at­tract­ing at­ten­tion for its price back in 2009, when it was priced at $30,000 a month af­ter be­ing ap­proved to treat rare cas­es of pe­riph­er­al T-cell lym­phoma. Al­los, which de­vel­oped the drug, de­fend­ed the price as in line with oth­er drugs for rare can­cers. Spec­trum bought out Al­los in 2012.

Robert Forrester, Verastem

Ve­rastem CEO For­rester steps to the ex­it as the board hunts com­mer­cial-savvy ex­ec for the be­lea­guered biotech

Robert For­rester is step­ping down as CEO of Ve­rastem On­col­o­gy $VSTM just 8 months af­ter the com­pa­ny nabbed an ap­proval for du­velis­ib, a PI3K drug with a sto­ried past — and what ap­pears as not much of a fu­ture.

The biotech put out word this morn­ing that For­rester will take an ad­vi­so­ry role with Ve­rastem while COO Dan Pa­ter­son steps up to take charge of the lead­er­ship team and the board looks around for a new CEO.

In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.

Ken Frazier 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

Who’s next in line to suc­ceed Ken Fra­zier as CEO of the Keytru­da-blessed Mer­ck?

When Merck waved off a looming forced retirement for Ken Frazier last September, the board cited flexibility in CEO transition as a key factor in the decision. Having Frazier — who’s also chairman of the company — around beyond his 65th birthday in 2019 would ensure they install the best person at the best time, they said.

The board has evidently begun that process with a clear preference for internal candidates, sources told Bloomberg. CFO Robert Davis, chief marketing officer Michael Nally, and chief commercial officer Frank Clyburn are all in the running, according to an insider.

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John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

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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A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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