Yes, the FDA did just slap down PTC’s Duchenne MD drug for the third time

What­ev­er last shred of hope re­mained that PTC could turn things around and win a mir­a­cle ap­proval at the FDA for their Duchenne mus­cu­lar dy­s­tro­phy drug ataluren end­ed with a terse state­ment Wednes­day morn­ing not­ing that the agency has re­ject­ed their mar­ket­ing ap­pli­ca­tion.

Ac­cord­ing to PTC, the FDA has de­mand­ed more da­ta from a new clin­i­cal study, along with some CMC in­put along with ad­di­tion­al in­fo that the biotech says it is in the process of pro­vid­ing.

The FDA de­ci­sion marks the lat­est mis­step in a long and rocky road for PTC, which has been bang­ing on the FDA’s doors now for years with­out get­ting much more than re­peat­ed signs to move along. But PTC CEO Stu Peltz still won’t take re­peat­ed no’s for an an­swer.

“We are ex­treme­ly dis­ap­point­ed for the Duchenne com­mu­ni­ty and strong­ly dis­agree with the agency’s con­clu­sions,” said Peltz in a state­ment. “We be­lieve that this de­ci­sion fails to con­sid­er the ben­e­fit-risk of ataluren and the high un­met med­ical need. There­fore, we plan to file a for­mal dis­pute res­o­lu­tion re­quest next week.”

In­stead of see­ing its share price dive on bad news, though, PTC’s stock climbed 4%.

Stu Peltz

Twice the FDA re­fused to even file their ap­pli­ca­tion, slam­ming the biotech for fail­ing to put to­geth­er a cred­i­ble ap­pli­ca­tion af­ter a se­ries of tri­al fail­ures. As a re­sult, bet­ting that the agency might bend in the face of fam­i­ly sup­port for their drug — the way Sarep­ta ben­e­fit­ed de­spite fail­ing to demon­strate ef­fi­ca­cy — the com­pa­ny forced an agency re­view.

The sub­se­quent agency analy­sis of­fered an­oth­er slap-down for PTC, which has been forced to de­tail a se­ries of tri­al fail­ures for their drug. The FDA re­view not­ed:

Ul­ti­mate­ly, no pos­i­tive re­sults from any prospec­tive­ly planned analy­ses that are per­sua­sive have been pro­vid­ed with this ap­pli­ca­tion. The ap­pli­cant has pre­sent­ed on­ly the re­sults from nu­mer­ous post hoc and ex­plorato­ry analy­ses that are in­tend­ed to mit­i­gate two neg­a­tive clin­i­cal tri­als.

In a rare sign of their dis­plea­sure, the agency al­so of­fered their opin­ion that this is one drug that may nev­er be re­deemed by pos­i­tive re­sults from a clin­i­cal tri­al.

Aaron Kessel­heim of Har­vard Med­ical School, who sat on the FDA ex­pert pan­el that looked this drug over, con­clud­ed that PTC’s da­ta were the kind of re­sults that could eas­i­ly be mis­lead­ing, join­ing in a 10 to 1 vote against the mar­ket­ing ap­pli­ca­tion.

It could have gone worse for PTC. The FDA al­lowed the pan­el to con­sid­er a sum­ma­ry ex­e­cu­tion of PTC’s chances, but the pan­el pre­ferred to con­clude that the da­ta were sim­ply in­con­clu­sive thus far — al­low­ing PTC the chance to come back with new da­ta at some point.

Amaz­ing­ly, de­spite the re­peat­ed set­backs on ataluren, the Eu­ro­peans have al­lowed their ac­cel­er­at­ed ap­proval of the drug to stand as PTC is giv­en chance af­ter chance to come up with some­thing fa­vor­able.

PTC, though, is busy mar­ket­ing a Duchenne drug. Af­ter Marathon Phar­ma was gripped in a con­tro­ver­sy fol­low­ing an ap­proval for an old, cheap Eu­ro­pean steroid for the lethal dis­ease, Marathon opt­ed to shut down, hand­ing over their ther­a­py to PTC, which has con­tin­ued to charge even more than Marathon’s planned $84,000 an­nu­al whole­sale price for the larg­er boys in the Duchenne pop­u­la­tion.

The out­rage that greet­ed Marathon, though, has large­ly died down af­ter PTC man­aged to lim­it its pub­lic price to the ball­park dis­count that pay­ers would face.

The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

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David Chang, Allogene CEO (Jeff Rumans)

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Pfiz­er, Mer­ck KGaA ce­ment Baven­cio blad­der can­cer win with OS da­ta — while carv­ing an­oth­er niche in rare can­cer

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Fabrice Chouraqui, Cellarity CEO-partner (LinkedIn)

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Fabrice Chouraqui’s career has taken some big twists along his life journey. He got his PharmD at Université Paris Descartes and jumped into the drug development game for a bit. Then he took a sharp turn and went back to school to get his MBA at Insead before returning to pharma on the commercial side.

Twenty years later, after steadily rising through the ranks and journeying the globe to nab a top job as president of US pharma for the Basel-based Novartis, Chouraqui exited in another career switch. And now he’s headed into a hybrid position as a CEO-partner at Flagship, where he’ll take a shot at leading Cellarity — one of the VC’s latest paradigm-changing companies of the groundbreaking model that aspires to deliver a new platform to the world of drug R&D.

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Paul Hudson, Sanofi CEO (Getty Images)

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