Bio­Marin scores a quick FDA OK for its lat­est rare dis­ease en­try — flagged at $702K WAC

Jean Jacques Bi­en­aimé

The FDA has pro­vid­ed a wide­ly ex­pect­ed green light for Bio­Marin’s Brineu­ra (cer­liponase al­fa), set­ting up the roll­out for one of the most ex­pen­sive new drugs to hit the mar­ket.

Bio­Marin $BM­RN at­tract­ed at­ten­tion for its lat­est rare dis­ease drug for its ef­fi­ca­cy in treat­ing 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. And just days ago the Eu­ro­pean Med­i­cines Agency’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use pro­vid­ed their thumbs up as well.

The drug was test­ed in on­ly 22 pa­tients in an ab­bre­vi­at­ed study, with a com­par­i­son on the nat­ur­al his­to­ry of sev­er­al dozen oth­ers. And that’s a mod­el that will help de­fine more such stud­ies in the fu­ture.

Bio­Marin CEO JJ Bi­en­ame not­ed in a call with an­a­lysts Thurs­day evening  that the ap­proval came “af­ter just 3 years and 8 months in the clin­ic,” with max­i­mum flex­i­bil­i­ty from the FDA for an ul­tra rare dis­ease based on one small clin­i­cal study, with a his­tor­i­cal com­par­i­son, and no ex­pert pan­el re­view. And they won a pri­or­i­ty re­view vouch­er, which could eas­i­ly be worth $125 mil­lion or more, based on re­cent sales.

The drug is de­signed to slow loss of am­bu­la­tion among pa­tients, as the da­ta in­di­cate.

The cost: $27,000 every two weeks, or $702,000 list; an av­er­age $486,000 per year af­ter Med­ic­aid dis­counts are fac­tored. That will be the sec­ond most ex­pen­sive ther­a­py avail­able in the US, right af­ter Hori­zon’s Rav­ic­ti, at $793,632 whole­sale, ac­cord­ing to a Rein­sur­ance Group of Amer­i­ca list based on WAC prices. Spin­raza from Bio­gen is now be­ing sold for $750,000 for the first year, which then drops to half that price.

Piper Jaf­fray an­a­lyst Joshua Schim­mer has al­ready not­ed that the com­pa­ny warned that its list price on this drug will fall on the high side, putting it on our lat­est list of the world’s 10 prici­est drugs, which you can see here. That list is dom­i­nat­ed by or­phan drugs de­vel­oped for small pa­tient pop­u­la­tions.

Bio­Marin’s stock, which had es­sen­tial­ly baked this ap­proval in­to the price, was up on­ly slight­ly at the end of the day.

“The FDA is com­mit­ted to ap­prov­ing new and in­no­v­a­tive ther­a­pies for pa­tients with rare dis­eases, par­tic­u­lar­ly where there are no ap­proved treat­ment op­tions,” said Julie Beitz, di­rec­tor of the Of­fice of Drug Eval­u­a­tion III in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “Ap­prov­ing the first drug for the treat­ment of this form of Bat­ten dis­ease is an im­por­tant ad­vance for pa­tients suf­fer­ing with this con­di­tion.”

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

No IND Re­quired for Cell and Gene Ther­a­py Stud­ies with Aus­tralia’s Ac­cred­it­ed CRO Avance Clin­i­cal

Avance Clinical is the specialist Australian CRO, with CGT accreditation, for international biotechs that leverages Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend.

Learn more about Avance ClinicReady here.
Contact us about your next study.
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The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Joan Perelló, Sanifit CEO

Joan Perel­ló set out 17 years ago to de­vel­op a drug. And to­day he's be­ing re­ward­ed with a $424M biotech buy­out

Joan Perelló beat all the odds with his little Spanish biotech startup Sanifit.

Working on the far perimeter of the big US/European drug development scene, he took a drug born out of his PhD work and got enough seed cash to get started. That’s one near miracle. In the second near miracle he gathered a previously unheard of venture raise in Spain — helping build an industry ecosystem from scratch — to pursue a successful search for solid human data for his drug, SNF472. And while gathering a virtual team of developers from Europe and the US, the CEO/co-founder steered it into the late-stage arena.

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