For 3rd time, the FDA and its new com­mis­sion­er dropped safe­ty is­sues and tri­al de­mands, clear­ing the path for snubbed ther­a­py

Ther­a­peu­tic­sMD $TXMD was slammed on May 8 when the FDA for­mal­ly re­ject­ed its ap­pli­ca­tion for a vagi­nal pain med­i­cine dubbed TX-004HR. Reg­u­la­tors want­ed more safe­ty da­ta, which would have re­quired a new study and years more work.

Then, one day lat­er, Scott Got­tlieb won con­fir­ma­tion as the new com­mis­sion­er of the FDA, and that may have made a huge dif­fer­ence in the biotech’s for­tunes.

This morn­ing Ther­a­peu­tic­sMD, which had said it would ap­peal the de­ci­sion, says the FDA has re­lin­quished its de­mands and will al­low them to re­sub­mit their ap­pli­ca­tion in a mat­ter of weeks. In­stead of a pre-ap­proval study, reg­u­la­tors say they will now be sat­is­fied with a post-ap­proval tri­al.

The biotech’s stock rock­et­ed up 116%.

Once rare, these sud­den about-faces by the FDA have be­come al­most rou­tine this year, with Ther­a­peu­tic­sMD lin­ing up as one of three com­pa­nies to win a huge re­prieve — sig­nal­ing a much more tol­er­ant at­ti­tude at the FDA in light of Pres­i­dent Don­ald Trump’s man­date to push more drug ap­provals faster as a way of bring­ing down phar­ma costs in the US.

All three had ap­pli­ca­tions re­ject­ed be­fore Got­tlieb’s ar­rival.

Robert Finizio

Am­i­cus Ther­a­peu­tics got this trend start­ed in Ju­ly — two months af­ter Got­tlieb took the head of­fice — when the FDA dropped its de­mand for a safe­ty study of its Fab­ry dis­ease drug mi­gala­s­tat, which bat­tered the biotech’s shares. In a fol­low-up, the FDA put the drug on its fast track pro­gram to speed up the new re­view.

Four months af­ter Got­tlieb showed up at his new of­fice at the FDA, Eli Lil­ly an­nounced that the FDA had re­versed course on its block­buster ap­pli­ca­tion for baric­i­tinib, al­so re­ject­ed on safe­ty grounds. As Lil­ly told me at the time:

The FDA has grant­ed us the op­por­tu­ni­ty to file our re­sub­mis­sion pack­age with­out a new clin­i­cal study.

Ther­a­peu­tic­sMD makes three, sig­nal­ing that the agency’s old stan­dards on safe­ty have clear­ly been down­grad­ed. While the agency has not been re­luc­tant to spurn new drug ap­pli­ca­tions, a new day has dawned on where the agency draws the line on safe­ty sig­nals. And that has ma­jor im­pli­ca­tions for drug de­vel­op­ers har­ried by ques­tions of safe­ty is­sues.

I asked Got­tlieb about the agency’s safe­ty stan­dards and a re­sponse to this sto­ry, but me­dia con­tacts at the FDA did not im­me­di­ate­ly re­spond.

While Am­i­cus and Eli Lil­ly both made cas­es for lead­ing ther­a­pies in their pipelines, Ther­a­peu­tic­sMD has been seek­ing an ap­proval for a me-too drug that will go up against oth­er well es­tab­lished ther­a­pies. Reg­u­la­tors had ob­ject­ed to the 12 weeks of safe­ty da­ta pro­vid­ed in the ap­pli­ca­tion, say­ing they want­ed 12 months of re­sults. But now they have been over­ruled.

An­oth­er con­sid­er­a­tion: If any of these drugs now be­ing helped to­ward the mar­ket back­fire and hurt pa­tients in un­ex­pect­ed ways, the FDA and Got­tlieb will have plen­ty of ex­plain­ing to do.

“We ap­pre­ci­ate the hard work and col­lab­o­ra­tive ef­fort by the FDA in mov­ing TX-004HR one step clos­er to ap­proval,” said Ther­a­peu­tic­sMD CEO Robert Finizio. “We are ex­treme­ly pleased with the FDA’s po­si­tion that an ad­di­tion­al pre-ap­proval safe­ty study is no longer nec­es­sary for the re­sub­mis­sion of the NDA for TX-004HR.”

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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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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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.