FDA or­ders a rare re­treat on Am­i­cus’ mi­gala­s­tat, drop­ping PhI­II de­mand and sig­nal­ing a ma­jor shift for drug de­vel­op­ers

Megan Crow­ley is rec­og­nized by Pres­i­dent Don­ald Trump dur­ing his first ad­dress to a joint ses­sion of Con­gress on Feb­ru­ary 28, 2017. At 15 months old, Crow­ley was di­ag­nosed with Pompe Dis­ease and not ex­pect­ed to live more than a few short years. AP Im­ages


Am­i­cus Ther­a­peu­tics $FOLD an­nounced ear­ly Tues­day that the FDA has dropped its de­mand for a new Phase III study of its Fab­ry dis­ease drug mi­gala­s­tat af­ter it was stiff-armed by un­sat­is­fied reg­u­la­tors last fall — then un­der a dif­fer­ent ad­min­is­tra­tion with a dif­fer­ent FDA com­mis­sion­er.

In­stead of pur­su­ing a new and ex­pen­sive late-stage study, Am­i­cus CEO John Crow­ley tells me the com­pa­ny has the green light to file again with the da­ta at hand. And based on dis­cus­sions with the FDA, he adds, the com­pa­ny is shoot­ing to file in the 4th quar­ter, with an ac­cel­er­at­ed ap­proval that could ar­rive in 2018, po­ten­tial­ly slic­ing years off the process.

“These are the days it’s good to be a biotech CEO,” says Crow­ley, who has been on a roller coast­er ride at the FDA over the past year af­ter be­ing forced to walk back his own plans last No­vem­ber.

John Crow­ley

There is no guar­an­tee of an ap­proval here, but the agency’s sud­den volte-face clear­ly sig­nals a new tone that vast­ly im­proves the biotech’s shot at an OK — at least in so far as we hear it from Crow­ley. And that could have a ma­jor in­flu­ence on oth­er de­vel­op­ers tack­ling the same task.

In­vestors cheered on the sud­den change in for­tunes at Am­i­cus, bid­ding up shares by 43% in pre-mar­ket trad­ing Tues­day.

Crow­ley has nev­er made a se­cret of the fact that he felt the drug — ap­proved in Eu­rope last year — was ready to go in the US af­ter it com­plet­ed the piv­otal pro­gram. The FDA, though, re­ject­ed his ap­pli­ca­tion, say­ing reg­u­la­tors want­ed more safe­ty da­ta than the Eu­ro­peans need­ed.

That re­jec­tion be­came a cause cele­bre of sorts in DC, as Pres­i­dent Don­ald Trump turned to Crow­ley’s daugh­ter, Megan, who suf­fers from Pompe dis­ease, and called out the FDA for the “slow and bur­den­some” process in­volved in new drug ap­provals dur­ing an ad­dress to Con­gress.

“Our slow and bur­den­some ap­proval process at the Food and Drug Ad­min­is­tra­tion keeps too many ad­vances, like the one that saved Megan’s life, from reach­ing those in need,” Trump said at the time. “If we slash the re­straints, not just at the FDA, but across our gov­ern­ment, then we will be blessed with far more mir­a­cles like Megan.”

Crow­ley picked up on that change in think­ing at the White House in a blog post at the time in which he said: “The FDA’s reg­u­la­tion of the or­phan de­vel­op­ment process is be­com­ing less flex­i­ble, less ef­fi­cient and less pa­tient-cen­tered.”

I asked Crow­ley what had changed be­tween the set­back last fall and the new, soft green light that the FDA has switched to.

First and fore­most, Crow­ley hit the theme that the com­pa­ny had made a con­vinc­ing case with what was avail­able. That in­cludes new da­ta and analy­sis that was made pos­si­ble af­ter the re­jec­tion. The com­pa­ny start­ed from the be­gin­ning to mar­shal the ev­i­dence need­ed to meet the agency’s gold stan­dard for an OK. That, he says, was ab­solute­ly key, ul­ti­mate­ly win­ning the day.

In ad­di­tion, he says there’s al­so a new frame­work for drug re­views that’s been gath­er­ing steam at the agency.

“I do think, can­did­ly, that there is an emerg­ing frame­work in rare dis­eases,” he says, point­ing to new ap­provals for Bio­marin on Bat­ten dis­ease as well as a nov­el la­bel ex­ten­sion for Ver­tex based on their in vit­ro as­say.

Scott Got­tlieb ap­pears be­fore a US Sen­ate sub­com­mit­tee, June 20, 2017 AP Im­ages

You can bet, though, that there will be plen­ty of sen­ti­ment sug­gest­ing that a change of ad­min­is­tra­tions and a new FDA com­mis­sion­er in Scott Got­tlieb made a telling dif­fer­ence for an abrupt about-face that is ex­tra­or­di­nar­i­ly rare to see at the FDA.

What­ev­er the cause, though, in­stead of prepar­ing to gath­er new da­ta that wouldn’t be avail­able un­til 2019, Crow­ley is back to mak­ing launch plans in the US. That will prob­a­bly re­quire a com­mer­cial group of about 50, he says, com­pared to the 80 need­ed to ad­dress a more com­plex and chal­leng­ing pay­er en­vi­ron­ment in Eu­rope.

Biotech has large­ly cheered on new FDA com­mis­sion­er Scott Got­tlieb af­ter he won con­fir­ma­tion by the Sen­ate. He promised to turn to new tech­nolo­gies to help ac­cel­er­ate new ap­provals, but vowed to stick with the in­dus­try gold stan­dard that has gov­erned reg­u­la­tors thoughts on safe­ty and ef­fi­ca­cy date.

So did the FDA just soft­en its stan­dards or wake up to the fact that the drug should nev­er have been re­ject­ed in the first place?

Let’s start the dis­cus­sion.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.