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.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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CEO Stephen Yoder (Pieris)

Pieris fi­nal­ly vaults FDA hold on next-gen sol­id tu­mor hunter, clear­ing the path for mid-stage tri­al

Finally freed from the restraints of a partial FDA clinical hold on its lead HER2-positive solid tumor candidate, Pieris Pharmaceuticals is now racing toward Phase II.

The FDA slapped a partial hold on Pieris’ PRS-343 back in July, restricting the biotech from enrolling new patients in a Phase I trial. While Pieris was allowed to continue dosing patients who were already enrolled, the agency requested they conduct an additional “in-use and compatibility study” before recruiting any more.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

News brief­ing: Ve­rastem CMO ex­its two weeks af­ter join­ing com­pa­ny; Ther­mo Fish­er inks $550M M&A deal

Two weeks after joining Verastem Oncology as chief medical officer, Frank Neumann is leaving the company for another job.

Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

FDA hits the brakes on His­to­gen's knee car­ti­lage ther­a­py, ask­ing for more in­fo on man­u­fac­tur­ing process

A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Andrew Allen, Gritstone CEO (Gritstone via website)

Grit­stone con­tin­ues Covid-19 push with deal to de­vel­op 'self-am­pli­fy­ing RNA' vac­cines, as shares con­tin­ue bal­loon­ing

Gritstone Oncology has had a big week, and it’s only Wednesday.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.

Artist rendering of the Assembly Square site in Somerville, MA (BioMed Realty)

Bio­Med Re­al­ty snaps up in­no­va­tion cam­pus site with­in earshot of pricey and bustling Boston biotech hub

On the short list of the premier biotech hubs in the world, the Boston area has transformed into a home for innovation — and ridiculously high rent. Now, a real estate firm is seeking tenants for a major site in neighboring Somerville with more than enough elbow room.

Snapped up by BioMed Realty, the land — which consists of an existing 162,000 square-foot office building and a 7.5 acre site — will serve as an “innovation space” for a variety of research, technology and life science tenants, the real estate company said in a press release. Financial terms weren’t disclosed.

Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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