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.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; 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.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

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HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

HHS fin­ish­es off Trump-era rule that would've erased ba­sic FDA regs with­out fre­quent re­views

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.

Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Vi­iV Health­care looks to make long-act­ing HIV pre­ven­tion shot ac­ces­si­ble in low- and mid­dle-in­come coun­tries

The Joint United Nations Programme on HIV and AIDS set a lofty goal back in 2019 to end the HIV epidemic by 2030. But according to the World Health Organization, infection rates are not falling rapidly enough to meet that target.

GSK’s ViiV Healthcare thinks it can help change that.

On Friday, ViiV announced that it’s in talks with the UN-backed Medicines Patent Pool (MPP) for patent rights to its cabotegravir long-acting HIV injectable for pre-exposure prophylaxis (PrEP) in low- and middle-income countries.