FDA OKs CSL’s new HAE drug, al­ready em­broiled in a block­buster fight with Shire

CSL Behring’s Bern, Switzer­land man­u­fac­tur­ing cam­pus


CSL Behring has picked up an FDA OK to start mar­ket­ing its new drug for rare cas­es of hered­i­tary an­gioede­ma, which is al­ready em­broiled in a court fight trig­gered by its HAE ri­vals at Shire. And in do­ing so, the FDA has now crossed the line for all its new drug ap­provals of 2016, still just shy of the halfway mark to the year.

The agency’s ap­proval for the C1 es­terase in­hibitor Hae­gar­da came through Thurs­day evening, two months af­ter Shire fired a pre­emp­tive le­gal shot in US Dis­trict Court in Delaware claim­ing that CSL’s drug vi­o­lates its new patent for its own C1 drug Cin­ryze. Shire — which saw its shares $SH­PG drop 1.3% Fri­day morn­ing — al­so mar­kets Fi­razyr and Kalbitor for HAE, a fran­chise that CSL hopes to carve up with a drug that can be self-ad­min­is­tered.

It comes with a clear en­dorse­ment from the FDA.

“The ap­proval of Hae­gar­da pro­vides a new treat­ment op­tion for ado­les­cents and adults with hered­i­tary an­gioede­ma,” said Pe­ter Marks, di­rec­tor of FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search. “The sub­cu­ta­neous for­mu­la­tion al­lows pa­tients to ad­min­is­ter the prod­uct at home to help pre­vent at­tacks.”

The drug was green light­ed by reg­u­la­tors af­ter in­ves­ti­ga­tors put it through a piv­otal tri­al with 70 pa­tients, demon­strat­ing its su­pe­ri­or­i­ty over a place­bo in pre­vent­ing at­tacks. It will now go in­to a small mar­ket with some 6,000 to 10,000 pa­tients in the US.

Flem­ming Orn­skov

CSL is an ex­pe­ri­enced play­er in the rare dis­ease field, which Shire CEO Flem­ming Orn­skov has col­ored in as its own pri­ma­ry fo­cus. CSL mar­kets Idelvion with a $500,000 whole­sale price. Idelvion — one of the top 10 most ex­pen­sive drugs on the plan­et — is a long-act­ing he­mo­phil­ia B drug that is the first in its class to in­clude the blood pro­tein al­bu­min.

Shire is fight­ing for time as it hus­tles along an FDA ap­pli­ca­tion for its new HAE drug lanadelum­ab af­ter bag­ging stel­lar Phase III da­ta just last month. Shire’s drug — ob­tained in a $6.5 bil­lion deal to ac­quire Dyax — has al­so been des­ig­nat­ed a break­through drug. Some an­a­lysts be­lieve it could be worth $2 bil­lion in peak sales, un­less CSL man­ages to make the most of its first mover ad­van­tage.

CSL’s drug re­duced HAE at­tacks by 89% and 95% while Shire’s new drug came in right along­side with an 87% re­duc­tion. These are not head-to-head re­sults, though, re­flect­ing dif­fer­ent ways to mea­sure ef­fi­ca­cy. Those fine points can make a big dif­fer­ence.

Law360 not­ed that Shire filed a law­suit against CSL the same day it ob­tained the patent for Cin­ryze.

These patent fights have be­come the or­der of the day in bio­phar­ma. Am­gen has been par­tic­u­lar­ly ag­gres­sive in the courts, push­ing an ad­van­tage it has over Re­gen­eron and Sanofi in a scuf­fle over its PC­SK9 drug patents. And Juno just had its case against Kite thrown out af­ter the judge de­cid­ed that the court didn’t have ju­ris­dic­tion af­ter de­ter­min­ing there was no way to guar­an­tee that Kite’s CAR-T would soon be ap­proved, even though it faces a loom­ing PDU­FA date.

Now at the halfway mark in the year, the FDA’s ac­tion marks the 23rd new drug ap­proved so far in 2017, 1 more than for all of 2016.

Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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The FDA will hus­tle up an ex­pe­dit­ed re­view for As­traZeneca’s next shot at a block­buster can­cer drug fran­chise

AstraZeneca paid a hefty price to partner with Daiichi Sankyo on their experimental antibody drug conjugate for HER2 positive breast cancer. And they’ve been rewarded with a fast ride through the FDA, with a straight shot at creating another blockbuster oncology franchise.

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Sean Parker, AP

Sean Park­er helps cre­ate a CRISPRed cell ther­a­py 2.0 play — and he’s got a high-pro­file set of lead­ers on the team

You can rack up one more high-profile debut effort in the wave of activity forming around cell therapy 2.0. It’s another appealing Bay Area group that’s attracted some of the top hands in the business to a multi-year effort to create a breakthrough. And they have $85 million in hand to make that first big step to the clinic.

Today it’s Ken Drazan and the team at South San Francisco-based ArsenalBio that are coming from behind the curtain for a public bow, backed by billionaire Sean Parker and a collection of investors that includes Beth Seidenberg’s new venture investment operation based in LA.
Drazan — a J&J Innovation vet with a long record of entrepreneurial endeavors — exited the stage in 2018 when his last mission ended as he stepped aside as president of Grail. It wasn’t long, though, before he was helping out with a business plan for ArsenalBio that revolved around the work of a large group of interconnected scientists supported by the Parker Institute for Cancer Immunology.

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CSL ac­cus­es ri­val Pharm­ing of par­tic­i­pat­ing in a scheme to rip off IP on HAE while re­cruit­ing se­nior R&D staffer

Pharming has landed in the middle of a legal donnybrook after recruiting a senior executive from a rival R&D team at CSL. The Australian pharma giant slapped Pharming with a lawsuit alleging that the Dutch biotech’s new employee, Joseph Chiao, looted a large cache of proprietary documents as he hit the exit. And they want it all back.
Federal Judge Juan Sanchez in the Eastern District Pennsylvania court issued an injunction on Tuesday prohibiting Chiao from doing any work on HAE or primary immune deficiency in his new job and demanding that he return any material from CSL that he may have in his possession. And he wants Pharming to tell its employees not to ask for any information on the forbidden topics.
For its part, Pharming fired off an indignant response this morning denying any involvement in extracting any kind of IP from CSL, adding that it’s cooperating in the internal probe that CSL has underway.

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Eli Lil­ly’s first PhI­II show­down for their $1.6B can­cer drug just flopped — what now?

When Eli Lilly plunked down $1.6 billion in cash to acquire Armo Biosciences a little more than a year ago, the stars seemed aligned in its favor. The jewel in the crown they were buying was pegilodecakin, which had cleared the proof-of-concept stage and was already in a Phase III trial for pancreatic cancer.

And that study just failed.

Lilly reported this morning that their cancer drug flopped on overall survival when added to FOLFOX (folinic acid, 5-FU, oxaliplatin), compared to FOLFOX alone among patients suffering from advanced pancreatic cancer.

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