No­var­tis' PI3K drug, al­ready ap­proved in breast can­cer, nabs a speedy OK to treat ul­tra-rare con­di­tion

Half a year af­ter No­var­tis show­cased the po­ten­tial of its PI3K in­hibitor alpelis­ib among a group of young pa­tients with an ul­tra-rare con­di­tion, the FDA has green­light­ed the drug’s use based sole­ly on re­al-world ev­i­dence.

Brand­ed Vi­joice, it is the first and on­ly treat­ment aimed at the root cause of PIK3CA-re­lat­ed over­growth spec­trum (PROS), ac­cord­ing to No­var­tis, a dis­ease es­ti­mat­ed to af­flict 14 peo­ple per mil­lion.

The un­der­ly­ing mu­ta­tions in the PIK3CA are just what alpelis­ib tar­gets. The same drug is al­ready mar­ket­ed as Piqray to treat a sub­set of ad­vanced or metasta­t­ic breast can­cer that’s HR-pos­i­tive, HER2-neg­a­tive and PIK3CA-mu­tat­ed. No­var­tis re­port­ed $390 mil­lion in sales for 2021.

With PROS, pa­tients’ body parts can grow too large or have an un­usu­al shape, and they may have blood ves­sel ab­nor­mal­i­ties.

“PROS con­di­tions can be de­bil­i­tat­ing and dis­abling and can re­sult in dis­rup­tions to every­day ac­tiv­i­ties,” said Kris­ten Davis, ex­ec­u­tive di­rec­tor of CLOVES Syn­drome Com­mu­ni­ty, in a pre­pared state­ment. “Un­til to­day, of­ten the on­ly treat­ment op­tions for pa­tients were sur­gi­cal or in­ter­ven­tion­al ra­di­ol­o­gy pro­ce­dures.”

Through a com­pas­sion­ate use pro­gram, a num­ber of PROS pa­tients re­ceived the drug. No­var­tis then ran a re­al-world study dubbed EPIK-P1 re­view­ing the med­ical charts of 57 pa­tients, in­clud­ing 39 pe­di­atric and 18 adult pa­tients, across five coun­tries. It found that those treat­ed with Vi­joice saw a re­duc­tion in the size of PROS le­sions as well as im­prove­ments in re­lat­ed signs and symp­toms.

Since Vi­joice was OK’d un­der the ac­cel­er­at­ed ap­proval path­way, No­var­tis will be re­quired to run a con­fir­ma­to­ry tri­al.

The phar­ma gi­ant said it will con­duct two ad­di­tion­al Phase II stud­ies: EPIK-P2 to as­sess safe­ty, ef­fi­ca­cy and phar­ma­co­ki­net­ics, and EPIK-P3 to look in­to long-term safe­ty and ef­fi­ca­cy for pa­tients who were in EPIK-P1.

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.”

Co-CEOs Chintu and Chirag Patel (Amneal)

Look out, Neu­las­ta: A 5th biosim­i­lar is com­ing

As Neulasta sales slip, Amgen has yet another biosimilar to look out for: Amneal Pharmaceuticals and Kashiv Biosciences’ Flynetra.

Flynetra became the fifth approved biosimilar to Neulasta on Friday, snagging a win in neutropenia, a condition common among chemotherapy patients where neutrophils, a type of white blood cell that fights infection, are too low.

As of last summer, the list price of Neulasta was more than $6,400 per dose. It’s designed to be taken in a single dose per chemotherapy cycle. Amneal declined to reveal how much it intends to charge for Flynetra in an email to Endpoints News. 

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.

Sen. Bill Cassidy (R-LA) (J. Scott Applewhite/AP Images)

Sen­ate un­veils its ver­sion of ac­cel­er­at­ed ap­proval re­forms as bi­par­ti­san duo calls on FDA and PTO to work to­geth­er

The Senate is joining its House counterparts and advancing accelerated approval pathway reforms to the FDA user fee legislation that must be signed by President Joe Biden before the end of September or else the FDA will have to start laying off its staff.

While Sen. Richard Burr (R-NC) warned yesterday that the user fee deals could be delayed by the infant formula crisis, the newly introduced bill on Friday shows how the Senate is aligning with its House counterparts on similar accelerated approval reforms.

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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.