The list of the top 10 most ex­pen­sive drugs on the plan­et will soon have a new open­ing

The world’s most ex­pen­sive drug — and first gene ther­a­py — will soon be­come ex­tinct.

UniQure an­nounced this morn­ing that it has de­cid­ed not to seek the reau­tho­riza­tion au­thor­i­ty that would have been re­quired to keep this drug on the mar­ket past Oc­to­ber. But af­ter by­pass­ing the FDA as a lost cause and see­ing on­ly one re­port­ed use of the gene ther­a­py in Eu­rope, which pro­vid­ed a con­di­tion­al ap­proval, the com­pa­ny would pre­fer to stick with its new­ly re­fo­cused pipeline plan.

Matthew Ka­pus­ta

Matthew Ka­pus­ta, chief ex­ec­u­tive of­fi­cer of uniQure, said:

“Gly­bera’s us­age has been ex­treme­ly lim­it­ed and we do not en­vi­sion pa­tient de­mand in­creas­ing ma­te­ri­al­ly in the years ahead.”

MIT Tech­nol­o­gy Re­view re­vealed last year that the drug was be­ing kept on the shelf, as pay­ers re­quired a mas­sive ef­fort to get an ap­proval for use and ques­tions lin­gered about its longterm ef­fi­ca­cy.

list of the world’s most ex­pen­sive drugs re­cent­ly ap­peared, from Rein­sur­ance Group of Amer­i­ca, and we thought that this would be a good time to show you how, and why, a swelling group of pricey or­phan ther­a­pies has been hit­ting the mar­ket.

In a re­cent in­ves­ti­ga­tion, Kaiser Health News con­clud­ed that the law on or­phan drugs — which of­fers a num­ber of in­cen­tives, in­clud­ing sev­en years of mar­ket­ing ex­clu­siv­i­ty — had in­spired a long list of pricey new ther­a­pies. Here are the most ex­pen­sive ones, ac­cord­ing to an­nu­al list prices. There are undis­closed dis­counts avail­able.

1. Gly­bera – $1,210,000

Com­pa­ny: uniQure
Cat­e­go­ry: Rare dis­ease — EU on­ly
(ali­po­gene tipar­vovec)

The world’s first gene ther­a­py is the prici­est — and al­so one of the least used — ap­proved drug on the plan­et. OK’d on­ly in Eu­rope for lipopro­tein li­pase de­fi­cien­cy, pay­ers don’t want to cov­er this one. UniQure has had to re­struc­ture the com­pa­ny in re­cent months, tai­lor­ing R&D to fo­cus on the com­pet­i­tive he­mo­phil­ia B area, Parkin­son’s and a col­lab­o­ra­tion with Bris­tol-My­ers Squibb.

2. 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.

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 2099, 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.

John Hood [file photo]

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CEO Pascal Soriot via Getty Images

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