FDA Ad­Com backs Gilead­'s move to ex­pand De­scovy la­bel — but stops short of en­dors­ing broad PrEP use

Gilead is one step clos­er to un­lock­ing a po­ten­tial­ly block­buster new use of its two-drug HIV reg­i­men De­scovy — though prob­a­bly not all of it.

In a meet­ing late Wednes­day, the FDA’s An­timi­cro­bial Drugs Ad­vi­so­ry Com­mit­tee vot­ed 16 to 2 en­dors­ing De­scovy as a pre-ex­po­sure pro­phy­lax­is, or PrEP, in men and trans­gen­der women who have sex with men. On the ques­tion of ex­pand­ing the in­di­ca­tion to in­clude cis­gen­der women, though, a slight ma­jor­i­ty of ex­perts (10 ver­sus 8) vot­ed against it, cit­ing a lack of da­ta.

Di­ana Brainard

The rec­om­men­da­tions were large­ly in line with the agency’s in-house re­view, which point­ed out that an ap­proval cov­er­ing cis­gen­der women would re­quire a ques­tion­able ex­trap­o­la­tion of PK da­ta. While Gilead did pro­vide proof of sys­temic drug con­cen­tra­tions, the mea­sure­ments were large­ly un­help­ful in quan­tifi­able and in­ter­pret­ing whether there are ad­e­quate drug con­cen­tra­tions in cer­vi­co­v­agi­nal tis­sues.

In a state­ment, Di­ana Brainard — SVP of HIV and emerg­ing virus­es — praised the Ad­Com for a “thought­ful re­view and dis­cus­sion of the da­ta.” Though the com­pa­ny did not com­ment di­rect­ly on ex­pand­ing De­scovy’s use to cis­gen­der women, it had this to add:

Gilead rec­og­nizes the val­ue that De­scovy could bring to HIV pre­ven­tion ef­forts for the broad­est pos­si­ble at-risk pop­u­la­tion in the Unit­ed States and will con­tin­ue to work with FDA as the agency com­pletes its re­view of the ap­pli­ca­tion.

In terms of seiz­ing a big piece of the mar­ket, though, miss­ing out on the cis­gen­der women la­bel might not be that big of a deal af­ter all. Cis­gen­der women form less than 7% of PrEP use, ac­cord­ing to Jef­feries an­a­lyst Michael Yee.

SVB Leerink’s Ge­of­frey Porges not­ed just a few days ago that MSMs form the most im­por­tant pop­u­la­tion for De­scovy’s add-on OK, though “for Gilead’s pur­pos­es it is use­ful to have a broad la­bel with all rel­e­vant at-risk pop­u­la­tions in­clud­ed, and get­ting such an en­dorse­ment from the Ad­Com would give their De­scovy launch ad­di­tion­al mo­men­tum.”

His peak sales es­ti­mate for De­scovy stands at $3.8 bil­lion.

Ad­di­tion­al mo­men­tum mat­ters here be­cause De­scovy will like­ly be go­ing up against gener­ics of Gilead’s own Tru­va­da, with launch­es sched­uled for next Sep­tem­ber. De­scovy’s ad­van­tages over Tru­va­da — sub­sti­tut­ing TAF, or teno­fovir alafe­namide, for TDF (teno­fovir diso­prox­il fu­marate) in com­bi­na­tion with emtric­itabine — could ap­pear slight un­der the best of cir­cum­stances. It re­mains to be seen whether pay­ers will be will­ing to cov­er a new brand­ed drug over a gener­ic for safe­ty is­sues such as bone min­er­al den­si­ty.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

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FDA to Sarep­ta: Your wide­ly an­tic­i­pat­ed fol­lowup to Ex­ondys 51 is not get­ting an ac­cel­er­at­ed OK for Duchenne MD

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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UP­DAT­ED: No­var­tis spin­off Nabri­va fi­nal­ly scores its first an­tibi­ot­ic ap­proval

In May, Nabriva Therapeutics suffered a setback after the FDA rejected its antibiotic for complicated urinary tract infections — the Novartis spinoff has now had some better luck with the US agency, which on Monday approved its other drug for community-acquired bacterial pneumonia.

The drug, lefamulin, has been developed as an intravenous and oral formulation and been tested in two late-stage clinical trials. The semi-synthetic compound, whose dosing can be switched between the two formulations, is engineered to inhibit the synthesis of bacterial protein by binding to a part of the bacterial ribosome.

Saqib Islam. CheckRare via YouTube

Spring­Works seeks $115M to push Pfiz­er drugs across fin­ish line while Sat­suma sells mi­graine play in $86M IPO

SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.

Mi­nor­i­ty racial groups con­tin­ue to be dis­mal­ly rep­re­sent­ed in can­cer tri­als — study

Data reveal that different racial and ethnic groups — by nature and/or nurture — can respond differently in terms of pharmacokinetics, efficacy, or safety to therapeutics, but this disparity is not necessarily accounted for in clinical trials. A fresh analysis of the last decade of US cancer drug approvals suggests the trend continues, cementing previous research that suggests oncology trials are woefully under-representative of the racial makeup of the real world.

Van­da shares slide af­ter FDA spurns their big end­point and re­jects a pitch on jet lag re­lief

Back in the spring of last year, Vanda Pharmaceuticals $VNDA served up a hot stew of mixed data for a slate of endpoints related to what they called clear evidence that their melatonin sleep drug Hetlioz (tasimelteon) could help millions of travelers suffering from jet lag.

Never mind that they couldn’t get a planned 90 people in the study, settling for 25 instead; Vanda CEO Mihael H. Polymeropoulos said they were building on a body of data to prove it would help jet-lagged patients looking for added sleep benefits. And that, they added, would be worth a major upgrade from the agency as they sought to tackle a big market.