So­bi's high-pri­or­i­ty rare dis­ease drug runs in­to a wall at EMA

The EMA has re­buffed Swe­den-based So­bi’s pitch for ema­palum­ab, shoot­ing down high hopes for a $568 mil­lion drug.

So­bi said it will re­quest a re-ex­am­i­na­tion by the reg­u­la­tors, which will kick off a new re­view process that should pro­duce a de­ci­sion by the end of the year. The an­ti­body is de­signed to treat pri­ma­ry he­mo­phago­cyt­ic lym­pho­his­ti­o­cy­to­sis in chil­dren un­der 18 years old by tar­get­ing in­ter­fer­on gam­ma (IFNγ).

The drug, al­ready ap­proved in the US as Gam­i­fant, is one of the stars of So­bi’s pipeline af­ter it re­struc­tured its op­er­a­tions to fo­cus on hema­tol­ogy and im­munol­o­gy.

The com­pa­ny first li­censed it from Swiss biotech Novim­mune in 2018, pay­ing $50 mil­lion and promis­ing $400 mil­lion in mile­stones be­fore de­cid­ing to ac­quire the whole thing — com­plete with in­tel­lec­tu­al prop­er­ty rights, the team work­ing on it and a pri­or­i­ty re­view vouch­er. Un­der the new deal, mile­stones add up to $518 mil­lion.

“Dur­ing the last years our team has gained a lot of ex­pe­ri­ence in this rather com­plex dis­ease area,” CEO Gui­do Oelk­ers said in a state­ment, as over 100 pa­tients have been treat­ed in the US.

He didn’t elab­o­rate on what might have gone wrong in the ap­pli­ca­tion. Fran­co Lo­catel­li, the prin­ci­pal in­ves­ti­ga­tor in the EU, was quot­ed say­ing he was “sig­nif­i­cant­ly sur­prised” about the EMA rec­om­men­da­tion against ap­proval.

“I had the priv­i­lege to ob­serve that this mon­o­clon­al an­ti­body, tar­get­ing the main cy­tokine in­volved in the dis­ease patho­phys­i­ol­o­gy, was well tol­er­at­ed and ef­fec­tive in a large pro­por­tion of the pa­tients, rep­re­sent­ing a mod­el of pre­ci­sion med­i­cine,” he said. “While US chil­dren have since al­most 2 years the pos­si­bil­i­ty to be treat­ed with this nov­el, safe, high­ly ef­fec­tive and tar­get­ed ther­a­py, the EMA de­ci­sion paves the way for mi­gra­to­ry health flows to­wards non-Eu­ro­pean Cen­ters that can grant this treat­ment.”

In the re­lease So­bi al­so re­it­er­at­ed its es­ti­mat­ed peak sales tar­get of over $500 mil­lion — which “re­mains un­changed re­gard­less of an ap­proval in Eu­rope.”

Sales of Gam­i­fant in the first six months of 2020 were $26.7 mil­lion. In 2019, the first year of launch, to­tal sales were $61.3 mil­lion.

By So­bi’s es­ti­mate, the most im­por­tant mar­kets based on num­ber of pa­tients for HLH — a rare but se­vere dis­ease that can re­sult in fever, en­large­ment of the liv­er and spleen, en­larged lymph nodes, rash­es and hos­pi­tal­iza­tions — are Chi­na, fol­lowed by the US, Eu­rope and Japan.

It’s al­so ini­ti­at­ing clin­i­cal stud­ies for pre-emp­tive treat­ment of pa­tients with risk fac­tors of HSCT acute graft fail­ure.

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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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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Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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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.

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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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Joaquin Duato, J&J CEO (Photo by Charles Sykes/Invision/AP)

New J&J CEO Joaquin Du­a­to promis­es an ag­gres­sive M&A hunt in quest to grow phar­ma sales

Joaquin Duato stepped away from the sideline and directly into the spotlight on Tuesday, delivering his first quarterly review for J&J as its newly-tapped CEO after an 11-year run in senior posts. And he had some mixed financial news to deliver today while laying claim to a string of blockbuster drugs in the making and outlining an appetite for small and medium-sized M&A deals.

Duato also didn’t exactly shun large buyouts when asked about the future of the company’s medtech business — where they look to be in either the top or number 2 position in every segment they’re in — even though the bar for getting those deals done is so much higher.

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Amgen's Twitter campaign #DearAsthma inspired thousands of people to express struggles and frustrations with the disease

Am­gen’s #Dear­Asth­ma spon­sored tweet lands big on game day, spark­ing thou­sands to re­spond

Amgen wanted to know how people with asthma really felt about daily life with the disease. So it bought a promoted tweet on Twitter noting the not-so-simple realities of life with asthma and ended the post with a #DearAsthma hashtag, a megaphone emoji and a re-tweet button.

That was just over one week ago and the responses haven’t stopped. More than 7,000 posts so far on Twitter replied to #DearAsthma to detail struggles of daily life, expressing humor, frustration and sometimes anger. More than a few f-bombs have been typed or gif-ed in reply to communicate just how much many people “hate” the disease.

Pfiz­er, Bris­tol My­ers dom­i­nate top 10 pre­dic­tions for the best-sell­ing drugs of 2022

The annual exercise where analysts try and predict which drugs will become blockbusters and make the most money tends to highlight the biggest trends in biopharma R&D. 2022 is no exception.

The team at Evaluate Vantage published its predictions for the top 10 selling drugs for the year — expecting tens of billions of dollars in sales and highlighting an industry-wide focus on certain diseases and indications.

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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.