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Lessons in Cat­e­go­ry Cre­ation: In Con­ver­sa­tion with Bob­by Aza­mi­an, CEO Tar­sus Phar­ma­ceu­ti­cals
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4. United Therapeutics to take pulmonary hypertension drug to FDA for approval
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Drew Armstrong
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We've got an update to today's story on uniQure's interactions with the FDA, with comments from a senior FDA official who shared more of the agency's thinking about why it told the biotech it would have to run a placebo-controlled trial, and what that would mean for the company.

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Drew Armstrong
Executive Editor, Endpoints News
@ArmstrongDrew
1
by Max Bayer

HHS Sec­re­tary Robert F. Kennedy Jr. named two new mem­bers to a key CDC vac­cine pan­el less than three weeks be­fore it’s set to con­vene.

Sean Down­ing and An­geli­na Farel­la were ap­point­ed to the Ad­vi­so­ry Com­mit­tee on Im­mu­niza­tion Prac­tices in an an­nounce­ment Fri­day. Their join­ing comes as the group is set to dis­cuss Covid-19 vac­cine in­juries and long Covid at a meet­ing lat­er this month.

Down­ing is a Sara­so­ta, FL-based physi­cian de­scribed by HHS as hav­ing more than two decades of ex­pe­ri­ence in pri­ma­ry care. He opened a concierge pri­ma­ry care prac­tice in 2019 and has pro­vid­ed med­ical ser­vices to a Flori­da non­prof­it, Sec­ond Heart Homes, that pro­vides hous­ing and wrap-around care to un­housed peo­ple.

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Matt Kapusta, uniQure CEO
2
by Max Gelman

Will Hunt­ing­ton’s dis­ease pa­tients be will­ing to risk a 12-hour sham brain surgery if it means a gene ther­a­py for their fa­tal con­di­tion might ul­ti­mate­ly be ap­proved?

That’s the im­plied ques­tion at the heart of uniQure’s dis­pute with the FDA, as an in­creas­ing num­ber of rare dis­ease com­pa­nies have come un­der height­ened — and of­ten con­tra­dic­to­ry — reg­u­la­to­ry scruti­ny. Un­der FDA Com­mis­sion­er Mar­ty Makary, the agency has trum­pet­ed ef­forts to ac­cel­er­ate rare dis­ease drug ap­provals, while at the same time re­ject­ing such treat­ments and in­sist­ing that they be test­ed in clin­i­cal tri­als that may not be fea­si­ble.

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Paul Chaplin, Bavarian Nordic CEO
3
by Anna Brown

Fol­low­ing the col­lapse of a $3 bil­lion bid for Bavar­i­an Nordic by pri­vate eq­ui­ty firms in No­vem­ber, CEO Paul Chap­lin is step­ping down af­ter more than three decades at the com­pa­ny due to "per­son­al rea­sons."

Chap­lin, who has been CEO of the Dan­ish vac­cine mak­er since 2014, will con­tin­ue in his role un­til the end of this year, be­fore re­lo­cat­ing to Aus­tralia with his fam­i­ly or un­til a suc­ces­sor is ap­point­ed. The board has start­ed its hunt for a new CEO, a Mon­day press re­lease states.

Bavar­i­an’s stock was down about 2.84% Mon­day on the Copen­hagen stock ex­change.

In No­vem­ber, the com­pa­ny’s chair­man Luc De­bruyne stepped down, fol­low­ing the break­down of Nordic Cap­i­tal and Per­mi­ra’s po­ten­tial $3 bil­lion takeover of Bavar­i­an. The firms pulled out af­ter the bid failed to muster up enough sup­port from the vac­cine mak­er's share­hold­ers.

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4
by Kyle LaHucik

Unit­ed Ther­a­peu­tics said its pul­monary ar­te­r­i­al hy­per­ten­sion drug ra­linepag suc­ceed­ed in a Phase 3 study, set­ting the drug­mak­er up for an FDA ap­proval re­quest this year and a po­ten­tial launch in 2027.

Pul­monary ar­te­r­i­al hy­per­ten­sion is a form of high blood pres­sure