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.

George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

On a roll, Mer­ck blazes through a new seg­ment of the bio­mark­er trail

Merck has notched an approval for using Keytruda to treat a biomarker-based subset of first-line colorectal cancer patients with unresectable or metastatic tumors, as the pharma giant continues to find new niches for its blockbuster PD-1 star.

The OK is significant in a number of ways. Not only does it build on an accelerated approval for all tumors characterized as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR); it also marks the first single treatment for colorectal cancer that doesn’t contain chemotherapy.

Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi preps an­oth­er round of lay­offs as new ex­ecs look to slim down the glob­al phar­ma in an on­go­ing re­or­ga­ni­za­tion — re­port

Sanofi is reportedly once again sharpening up the budget axe as the pharma giant prepares to chop more jobs.

Reuters reports this morning that Sanofi has its sights set on cutting 1,680 jobs in Europe, where the unions have been combating rounds of cutbacks at the Paris-based player. Sanofi itself hasn’t said anything about these cuts, as the company looks to discuss a 3-year plan with staff representatives.

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Covid-19 roundup: Vac­cines will need to beat place­bo by 50% to qual­i­fy for FDA OK; UK tri­al drops Kale­tra

The FDA will set the bar for approving a Covid-19 vaccine at 50% efficacy, the Wall Street Journal reported, meaning any successful candidate will have to reduce the risk of coronavirus disease by at least half compared to placebo.

That requirement is part of guidance that the agency is set to release later today, laying out detailed criteria for vaccine developers — some of whom are eyeing an OK by the end of the year, in line with expectations at Operation Warp Speed.

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