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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

Anthony Fauci, AP Images

First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

Mallinck­rodt, once the na­tion’s largest oxy­codone pro­duc­er, an­nounces ten­ta­tive $1.6B set­tle­ment

Three years after it first paid out fines for its role in the US opioid abuse epidemic, Mallinckrodt has announced an agreement-in-principle that will see the company pay out $1.6 billion and place its generics unit in bankruptcy.

The tentative deal would settle hundreds of lawsuits from state and local governments over Mallinckrodt’s role in the epidemic, while also helping address the company’s increasingly mountainous debt. Although Purdue Pharma has drawn the bulk of both public and legal acrimony for opioid sales, documents made public earlier this year showed that Mallinckrodt subsidiary SpecGx, along with the generic subsidiaries of Teva and Endo Pharmaceuticals, accounted for the vast majority of the 76 billion opioid pills distributed from 2006 to 2012. Mallinckrodt was at the top of that list.