High­light­ing a ma­jor shift at the FDA, reg­u­la­tors put Am­i­cus' once-spurned drug on fast track

In gen­er­al, the FDA’s fast track des­ig­na­tions don’t make news. The ma­jor­i­ty of new drugs which gain ap­proval these days get some sort of spe­cial sta­tus — to the point that it isn’t so spe­cial any­more.

But when you’re Am­i­cus Ther­a­peu­tics $FOLD, and you’re bounc­ing back from a sting­ing set­back for the lead drug mi­gala­s­tat, it’s a spe­cial mo­ment to an­nounce that the agency has put your drug for Fab­ry dis­ease on the fast track. Reg­u­la­tors are es­sen­tial­ly promis­ing to make things hap­pen fast as Am­i­cus lines up a new fil­ing be­fore the end of this year, though it’s not a guar­an­tee of any kind.

It’s al­so note­wor­thy for the in­dus­try, as the change of sta­tus high­lights a new regime at the FDA which has vowed to step up drug ap­provals — with­out dam­ag­ing the gold stan­dard on drug ap­provals.

FDA com­mis­sion­er Scott Got­tlieb is draw­ing a new line in the sand.

John Crow­ley, Am­i­cus

“As we ex­e­cute our in­ter­na­tion­al launch and con­tin­ue pur­su­ing glob­al reg­u­la­to­ry ap­provals for mi­gala­s­tat, it is our vi­sion to bring this im­por­tant treat­ment to even more peo­ple in more ge­o­gra­phies who may ben­e­fit,” said CEO John Crow­ley in a state­ment. “We look for­ward to sub­mit­ting our NDA and col­lab­o­rat­ing with the U.S. FDA through­out the reg­u­la­to­ry process.”

The FDA was in any­thing but a col­lab­o­ra­tive mood when it slammed Am­i­cus with a ma­jor de­lay last fall, de­mand­ing that it con­duct a Phase III study. But then came a new Trump ad­min­is­tra­tion and a new FDA com­mis­sion­er. And Crow­ley — who had talked di­rect­ly with Trump about the sit­u­a­tion, gain­ing his sup­port — was giv­en the green light to re­file over the sum­mer.

To­day, he al­so has the FDA’s com­mit­ment to speed things along.

Sig­nif­i­cant­ly, Am­i­cus isn’t the on­ly drug de­vel­op­er to ben­e­fit from the change of the pow­er group in Wash­ing­ton DC. Phar­ma gi­ant Eli Lil­ly al­so ex­e­cut­ed a swift turn­around af­ter bit­ter­ly crit­i­ciz­ing the FDA’s re­jec­tion of baric­i­tinib on safe­ty rea­sons. Now, in­stead of be­ing forced back in­to the clin­ic, Lil­ly has the FDA’s ap­proval to re­file as well. And both these drugs loom large for their re­spec­tive com­pa­nies.

The FDA doesn’t do any­thing like com­ment pub­licly about its stance on any drug, un­til and un­less they ac­tu­al­ly ap­prove it. But Am­i­cus and Lil­ly are prime ex­hibits in the case that the Trump/Got­tlieb ap­proach to drug de­vel­op­ment won’t be quite as ready to throw the red flag.

The im­pli­ca­tions for bio­phar­ma are huge.

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II 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.

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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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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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 help@endpointsnews.com 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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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

UP­DAT­ED: 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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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.