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.

Jake Van Naarden, Josh Bilenker, Nisha Nanda (Credit: Loxo, Aisling Capital)

Josh Bilenker and his Loxo crew are tak­ing the reins on on­col­o­gy R&D at Eli Lil­ly, culling the weak and map­ping a new path

Josh Bilenker, Jake Van Naarden and Nisha Nanda came out of Eli Lilly’s $8 billion Loxo Oncology buyout with a bundle of cash and plenty of choices on what they could do next. Start a new company, go public. Live on the beach in 5-star luxury. Contemplate the stars — in their own observatory.

So what are they doing?

They formed a new executive team that is taking over the management of Eli Lilly’s hundreds-strong oncology R&D group — essentially using Loxo as a base for a bold new experiment in Big Pharma R&D in an attempt to create a true biotech environment with the deep pockets of a top-15 industry player. They’ve recruited David Hyman from Memorial Sloan Kettering to join the team as chief medical officer. And the mandate includes culling out the oncology pipeline, highlighting their star prospects and going after new programs wherever they can find the best prospects.

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One of Wall Street’s most high-pro­file hedge funds push­es Alex­ion's CEO to the auc­tion block — and he's not budg­ing

Fresh off buying Barnes & Noble and prodding AT&T with some heavy-handed criticism after picking up a $3.2 billion stake in the company, the activist — and supremely high profile — hedge fund Elliott Management has stepped up with some M&A advice for Alexion’s management team.
And the execs on the team $ALXN are giving them a polite — but very firm — stiff arm Friday morning.
In a release out early Friday, the big biotech said that the Elliott team had been in touch to encourage them to sell the company. But that’s not on the agenda.

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Samantha Budd Haeberlein. Biogen via YouTube

UP­DAT­ED: Skep­tics pounce as Bio­gen de­tails pos­i­tive sub­group analy­sis on ad­u­canum­ab — and both sides are dig­ging in

“Exhilarating.” “A major advance.” “A milestone achievement.” If one had just tuned into the panel comments on Biogen’s presentation at CTAD, it would seem that the biotech had an impressive, disease-modifying Alzheimer’s drug in aducanumab.

But off the stage, reactions to their admittedly complicated dataset and the biotech’s explanation for resurrecting a drug that failed its futility analysis were a lot more mixed, with analysts continuing to question whether the evidence is substantial enough to warrant an FDA approval and raising new doubts on the safety side.

In an investor call later in the day, execs noted that they are not planning another study and stood by their intention, publicized in October to much surprise, to submit regulatory filings based on what they have.

“We don’t file willy nilly,” said Al Sandrock, head of R&D. “We only go to filing when we believe that there is a benefit-risk argument based on science, based on data. And if you look at our history, we haven’t done filings right and left without good reason.”

Biogen had a theory going into the Clinical Trials on Alzheimer’s Disease meeting.

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Un­lock the full End­points ex­pe­ri­ence for your com­pa­ny — and sup­port our mis­sion of in­de­pen­dent bio­phar­ma re­port­ing

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We offer two premium plans — Enterprise for companies ($1,000/year, unlimited people), and Insider for individuals ($200/year). This month of December will be the last chance to enroll at the original rates — which have remained flat since we launched them in 2017.

Jasper Ther­a­peu­tics launch­es out of Stan­ford with new ap­proach to stem cell treat­ment

The first girl in the trial came in with chronic diarrhea and the immune system of an untreated HIV patient. Born with a rare genetic disease that impeded her ability to make B and T cells, she had once been given a stem cell transplant but it didn’t take.  Back in the hospital, she was injected with a new experimental antibody and then given a new stem cell transplant. Soon, she gained weight. The diarrhea stopped.

Ex-Cel­gene ex­ec Ter­rie Cur­ran puts her Phath­om team in place; Car­away taps Mar­tin Williams as CEO

→ Gastrointestinal disease-focused Phathom Pharmaceuticals has shaken up its leadership team. The company has tapped former Celgene exec Terrie Curran as CEO, succeeding David Socks, who is transitioning to interim CFO. Curran was president of Celgene’s global inflammation and immunology franchise — helping with the sale of Otezla for $13.4 billion to Amgen — and has held a previous stint at Merck. In addition to Curran, the company also welcomed former Omeros CMO Eckhard Leifke as CMO, ex-Celgene exec Joseph Hand as chief administrative officer, and former general counsel for Cyclerion Therapeutics Larry Miller as general counsel. They also replaced Chris Slavinsky on the board with Takeda exec Asit Parikh.

UCB buffs up in block­buster pso­ri­a­sis race as bimek­izum­ab beats Hu­mi­ra in head-to-head

Just weeks after boasting head-to-head victories over first placebo and then J&J’s IL-23 contender Stelara in clearing psoriasis, the results are in for UCB’s last Phase III trial, in which bimekizumab went up against the world’s best-selling drug.

Only topline results are provided for today’s readout of the BE SURE study, so we won’t find out just how superior bimekizumab proved against Humira on the co-primary endpoints — standard scores known as PASI90 and IGA measuring the impact and severity of the disease — until a scientific conference in 2020.

With EMA re­view in progress, No­var­tis un­veils more pos­i­tive da­ta on asth­ma ther­a­py

Having made its marketing pitch to the EU regulator, Novartis on Thursday unveiled positive pivotal study data supporting the use of its inhaled asthma treatment.

The therapy, QMF149, consists of the long-acting beta-agonist, or LABA, called indacaterol acetate and the corticosteroid mometasone furoate. In the 2,216-patient, 52-week PALLADIUM study, asthma patients either received a medium or high dose of the Novartis therapy (150/160 μg; 150/320 μg) or mometasone furoate (MF) alone.

Black Di­a­mond rais­es an­oth­er $85M to bring new onco­gene ap­proach in­to clin­ic

Nearly a year after raising $85 million from investors, Black Diamond Therapeutics has raised another $85 million to help push its allosteric therapies into the clinic in the next few months. The Series C round was led by Boxer Capital.

Black Diamond launched last December as the first company to come out of Versant’s Ridgeline discovery engine in Basel. They had two former developers of the cancer drug Tarceva as co-founders in David Epstein and Elizabeth Buck and a relatively new approach to oncology. Within a month of their full launch, they also had $105 million in backing.