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.

Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary. The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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FDA ex­pert pan­el unan­i­mous­ly rec­om­mends ap­proval for Hori­zon Ther­a­peu­tics eye drug

An FDA advisory committee noted with concern a small safety database but unanimously endorsed a Horizon Therapeutics drug for a rare eye autoimmune disease that can blind patients: teprotumumab for thyroid eye disease (TED).

“It was a pretty easy vote,” said Erica Brittain, an NIH biostatistician and one of the 12 panelists on FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee.

Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny — just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.

After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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Arie Belldegrun at UKBIO 2019. Shai Dolev for Endpoints News

Kite Phar­ma's ex-CEO con­tra­dicts founder as CAR-T patent tri­al heats up, with con­flict­ing val­u­a­tions

Two days after Kite Pharma founder Arie Belldegrun told a federal courtroom that a meeting he had with a Memorial Sloan Kettering executive wasn’t about licensing their immunotherapy patent, Kite’s ex-CEO Aya Jakobovits said it was.

The admission came Tuesday during cross-examination in a patent infringement case that features two of the biggest cancer biotechs and some of the most well-known names in American medicine.

Jakobovits initially said she was not in attendance, didn’t know it was going to happen and didn’t know what took place, according to Law360. But then the plaintiff’s lawyer handed her a document – whose contents were not publicly revealed – and asked again if she learned after-the-fact that the meeting involved a potential patent license.

“Yes,” Jakobovits eventually said.

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On the heels of promis­ing MCL da­ta, Kite hus­tles its 2nd CAR-T to the FDA as the next big race in the field draws to the fin­ish line

Three days after Gilead’s Kite subsidiary showed off stellar data on their number 2 CAR-T KTE-X19 at ASH, the executive team has pivoted straight to the FDA with a BLA filing and a shot at a near-term approval.

In a small, 74-patient Phase II trial reported out at the beginning of the week, investigators tracked a 93% response rate with two out of three mantle cell lymphoma patients experiencing a complete response.

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