EC ap­proves Bio­Marin drug as first treat­ment for chil­dren with com­mon form of dwarfism; Servi­er sN­DA wins ap­proval

The Eu­ro­pean Com­mis­sion just ap­proved its first drug for use in chil­dren with achon­dropla­sia, a com­mon form of dwarfism.

Bio­Marin re­ceived mar­ket­ing au­tho­riza­tion to­day for Vox­zo­go, al­so known as vosori­tide. The drug is a once-dai­ly in­jec­tion to treat achon­dropla­sia in chil­dren from the age of 2 un­til growth plates are closed, which oc­curs af­ter pu­ber­ty.

An es­ti­mat­ed 11,000 chil­dren across Eu­rope, Mid­dle East, and Africa are af­fect­ed by achon­dropla­sia — and a third of this pop­u­la­tion are in coun­tries au­tho­rized un­der the EMA li­cense. The French Na­tion­al Agency for Med­i­cines and Health Prod­ucts Safe­ty al­so grant­ed Vox­zo­go a “Tem­po­rary Au­tho­riza­tion for Use” to al­low im­me­di­ate ac­cess.

The list price in France un­der that tem­po­rary au­tho­riza­tion is about $840 per vial across all ages and weights. An­nu­al­ly, that would be just over $300,000, as­sum­ing a pa­tient takes a dose every day.

FDA ap­proves sN­DA for Servi­er’s Tib­so­vo

French biotech Servi­er an­nounced to­day that they won ap­proval from the FDA for an­oth­er in­di­ca­tion of their on­col­o­gy drug Tib­so­vo.

The new in­di­ca­tion is for the treat­ment of adult pa­tients with pre­vi­ous­ly treat­ed, lo­cal­ly ad­vanced or metasta­t­ic cholan­gio­car­ci­no­ma with an IDH1 mu­ta­tion, which is a rare type of can­cer in the bile ducts in the liv­er. Tib­so­vo has pre­vi­ous­ly been ap­proved to treat AML.

The ap­proval is a win for Servi­er, which spent $1.8 bil­lion late last year to ac­quire Tib­so­vo from Agios, along with the rest of their on­col­o­gy pipeline.

Servi­er came un­der fire ear­li­er this year af­ter they were fined $3.2 mil­lion and con­vict­ed on manslaugh­ter, in­vol­un­tary wound­ing and ag­gra­vat­ed de­cep­tion charges in a French court. This hap­pened af­ter French pros­e­cu­tors as­so­ci­at­ed Servi­er’s di­a­betes drug Me­di­a­tor with up to 2,000 deaths in the 33 years the drug was on the mar­ket in France.

In­sil­i­co Med­i­cine and 4B Tech­nolo­gies An­nounce Strate­gic Col­lab­o­ra­tion

Chi­nese AI start­up In­sil­i­co and 4B Tech­nolo­gies an­nounced to­day that they have agreed to a strate­gic col­lab­o­ra­tion. The goal? To de­vel­op small mol­e­cule ther­a­pies for ALS and oth­er neu­ro­log­i­cal dis­eases.

Ac­cord­ing to 4B Tech­nolo­gies CEO Xi­aom­ing Guan, the tra­di­tion­al R&D process is time-con­sum­ing and ex­pen­sive. As such, col­lab­o­rat­ing with a com­pa­ny that has an em­pha­sis on AI would al­low 4B to ex­pe­dite its R&D process.

This is not In­sil­i­co’s on­ly re­cent deal. Just a few weeks ago, they an­nounced a deal with BioNo­va to joint­ly de­vel­op small mol­e­cule in­hibitors. This is just two months af­ter In­sil­i­co CEO Alex Zha­voronkov pulled in a mas­sive $255 mil­lion Se­ries C round back in June.

In­novent and Bolt Bio­ther­a­peu­tics to col­lab­o­rate on an­ti-can­cer can­di­dates

In­novent Bi­o­log­ics and Bolt Bio­ther­a­peu­tics an­nounced that they are col­lab­o­rat­ing on three new im­mune-stim­u­lat­ing an­ti­body con­ju­gate can­di­dates. The on­col­o­gy tar­gets re­main undis­closed.

The deal will see In­novent tak­ing re­spon­si­bil­i­ty for all R&D costs through clin­i­cal proof-of-con­cept — and uti­liz­ing Bolt’s ISAC plat­form to help cre­ate the can­di­dates. Up­on re­view, the com­pa­nies can ex­er­cise li­cens­ing op­tions for con­tin­u­ing de­vel­op­ment and ex­clu­sive com­mer­cial­iza­tion rights.

Ac­cord­ing to the agree­ment, In­novent has the rights to all three can­di­dates in Chi­na, Hong Kong, Macau and Tai­wan. Bolt will re­ceive $5 mil­lion in cash up­front from In­novent and a pos­si­ble fu­ture eq­ui­ty in­vest­ment of up to $10 mil­lion. Both com­pa­nies have ad­di­tion­al mile­stone pay­ments and roy­al­ties based on de­vel­op­ment and com­mer­cial­iza­tion in dif­fer­ent mar­kets.

Bolt, based out of Cal­i­for­nia, al­so part­nered with Gen­mab in June to de­vel­op up to three new an­ti­body-based con­ju­gates in on­col­o­gy.

Bio-Thera and Hik­ma to co-com­mer­cial­ize mon­o­clon­al an­ti­body

Chi­nese bio­phar­ma firm Bio-Thera So­lu­tions and British phar­ma­ceu­ti­cal com­pa­ny Hik­ma Phar­ma­ceu­ti­cals have en­tered in­to a li­cense agree­ment to com­mer­cial­ize a biosim­i­lar to Janssen’s Ste­lara, an im­muno­sup­pres­sant ap­proved in the US for Crohn’s dis­ease.

The biosim­i­lar can­di­date is cur­rent­ly in a glob­al Phase III clin­i­cal tri­al and un­der the agree­ment, Bio-Thera will keep re­spon­si­bil­i­ty for the de­vel­op­ment, man­u­fac­tur­ing, and sup­ply of the po­ten­tial biosim­i­lar. Hik­ma will have ex­clu­sive rights to com­mer­cial­ize it in the US, and first-right-to-ne­go­ti­ate to add Eu­rope, ex­clud­ing the nine mem­bers of the Com­mon­wealth of In­de­pen­dent States.

Bio-Thera is el­i­gi­ble for an up­front pay­ment of $20 mil­lion, as well as fur­ther de­vel­op­ment and com­mer­cial mile­stones of up to $130 mil­lion.

Alexander Lefterov/Endpoints News

A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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Bahija Jallal, Immunocore CEO

BREAK­ING: FDA hur­ries up a quick ap­proval for the world's first TCR -- af­ter a 14-year R&D trek

Over the 14 years since Immunocore was spun out of MediGene in a quest to develop a gamechanging cancer med, the biotech has raised record sums and undergone a major shakeup on a long roller coaster ride of valuations for investors. But they survived and thrived and today they’re popping the champagne corks to celebrate an FDA approval of their first TCR drug.

Immunocore flagged the FDA’s green light for tebentafusp Wednesday morning by highlighting a series of firsts.

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Dominic Borie, Kyverna Therapeutics CEO

Well-con­nect­ed, Gilead-backed biotech gets an­oth­er stack of cash to pur­sue CAR-Ts for au­toim­mune dis­ease

Almost exactly two years after its debut at the 2020 JP Morgan confab — and on the heels of a new partnership with the gene editing experts at Intellia — a Gilead-backed, autoimmune disease-focused startup has returned to the well with a clearer outline of just what it plans to do with its CAR-T platform.

Kyverna brought in $85 million in its oversubscribed Series B, the company announced Wednesday. Northpond Ventures led the round, and Westlake Village BioPartners, Vida Ventures, Gilead and Intellia all contributed as well.

Days af­ter Gilead yanks PI3K drug, In­cyte with­draws NDA for its own PI3K — say­ing con­fir­ma­to­ry tri­als would take too long

The FDA’s intensifying scrutiny on accelerated approvals isn’t just putting pressure on drugmakers with marketed products. It is also subtly reshaping the regulatory dynamics.

Case in point: Incyte announced late Tuesday that it has made the “business decision” to withdraw an NDA for parsaclisib, its oral PI3Kδ inhibitor, after deciding that running the confirmatory studies the agency was asking for to support an accelerated approval wouldn’t be worth it.

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Dan O'Day (Getty Images)

In a set­back, FDA or­ders Gilead to hit the brakes on their late-stage, $5B can­cer play

Gilead’s $5 billion drug magrolimab has run into a serious setback.

The FDA ordered Gilead to halt enrollment on their studies of the drug in combination with azacitidine after investigators reports revealed an “apparent imbalance” in the suspected unexpected serious adverse reactions between study arms. And the halt is raising questions about Gilead’s plans for a quick pitch to regulators.

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Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Florida Gov. Ron DeSantis (AP Photo/Wilfredo Lee, File)

Opin­ion: Flori­da is so mAb crazy, Ron De­San­tis wants to use mAbs that don't work

Florida Gov. Ron DeSantis is trying so hard to politicize the FDA and demonize the federal government that he entered into an alternate universe on Monday evening in describing a recent FDA action to restrict the use of two monoclonal antibody, or mAb, treatments for Covid-19 that don’t work against Omicron.

Without further ado, let’s break down his statement from last night, line by line, adjective by adjective.

Not cheap­er by the dozen: Bris­tol My­ers be­comes the 12th phar­ma com­pa­ny to re­strict 340B sales

Bristol Myers Squibb recently joined 11 of its peer pharma companies in limiting how many contract pharmacies can access certain drugs discounted by a federal program known as 340B.

Bristol Myers is just the latest in a series of high-profile pharma companies moving in their own direction as the Biden administration’s Health Resources and Services Administration struggles to rein in the drug discount program for the neediest Americans.