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.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.