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.”

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

Editor’s note: This is a live story and will be updated.

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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