Once re­ject­ed, FDA now rolls out a short red car­pet for Am­i­cus’ mi­gala­s­tat

John Crow­ley

Am­i­cus CEO $FOLD John Crow­ley must have made a very fa­vor­able im­pres­sion on the FDA as he lob­bied se­nior of­fi­cials to over­turn their re­jec­tion of his ex­per­i­men­tal Fab­ry drug mi­gala­s­tat.

Not on­ly did reg­u­la­tors just ac­cept the fil­ing for a new re­view, the agency is giv­ing it pri­or­i­ty sta­tus, cut­ting the re­think to 6 months and flag­ging a clear in­di­ca­tion that it’s an odds-on fa­vorite for an of­fi­cial mar­ket­ing OK .

Cut­ting 4 months off the re­view process, the FDA will have its new de­ci­sion out by Au­gust 13.

An­a­lysts are al­ready count­ing the near-term rev­enue that Am­i­cus stands to earn. Joseph Schwartz at Leerink not­ed:

With the PDU­FA goal date of Aug 13, 2018, US rev­enue could con­tribute to FY2018 rev­enue guid­ance of $75M-$85M an­nounced last month. We con­tin­ue to see strong growth dri­vers for Am­i­cus this yr. as the com­pa­ny con­tin­ues to ad­vance its mi­gala­s­tat (Galafold; Fab­ry dis­ease) and ATB200/AT2221 (Pompe dis­ease) pro­grams through the clin­ic.

Crow­ley had em­ployed some du­bi­ous po­si­tions as he fought against the re­jec­tion, which came with a de­mand from the FDA for a new Phase III study of gas­troin­testi­nal symp­toms. Ini­tial­ly, the CEO had told in­vestors it would take 2 years to come up with the da­ta. But he told Janet Wood­cock and oth­ers that the ac­tu­al time­line would be 5 to 7 years, which made the de­vel­op­ment pro­gram im­prac­ti­cal.

Scott Got­tlieb

This was one of three drug pro­grams that the FDA re­versed it­self on in the months af­ter Scott Got­tlieb took over as com­mis­sion­er at the FDA, with Eli Lil­ly and Ther­a­peu­tic­sMD — chaired by ex-HHS sec­re­tary Tom­my Thomp­son — get­ting a do-over. The agency has nev­er ex­plained the re­jec­tions, nev­er ex­plained why it de­cid­ed to re­con­sid­er their de­ci­sion, or why it would of­fer Am­i­cus a quick de­ci­sion now.

But they nev­er do, re­quired by the rules to stay qui­et. Got­tlieb had ini­tial­ly said he would press for the redact­ed pub­li­ca­tion of com­plete re­sponse let­ters like these, but he re­cent­ly shrugged that off.

“Con­grat­u­la­tions, again!” Crow­ley wrote in a note to Got­tlieb at­tached to his case for a re­view. “As dis­cussed, the hopes and well be­ing of so many liv­ing with rare, dev­as­tat­ing dis­eases rests now with your great lead­er­ship and wis­dom. Thanks for your at­ten­tion to the at­tached.”

Got­tlieb’s staff told me that the com­mis­sion­er nev­er saw that pack­et, redi­rect­ing it to oth­er se­nior of­fi­cials at the FDA. Of­fi­cials, though, de­clined to ex­plain what Crow­ley meant by the word “again,” or if Got­tlieb had dis­cussed the is­sue with the CEO.

That note, along with Crow­ley’s as­ser­tions on the time­line to re­spond, were ob­tained un­der FOIA by the Pro­ject on Gov­ern­ment Over­sight.

Got­tlieb has open­ly ad­vo­cat­ed for a more ef­fi­cient re­view process, par­tic­u­lar­ly where rare dis­ease are con­cerned. That al­so squares with Pres­i­dent Don­ald Trump’s vow to speed up the reg­u­la­to­ry process. And af­ter start­ing out with a high rat­ing from the in­dus­try, biotech ex­ecs are now cheer­ing him on as one of the best things that’s hap­pened at the FDA in years.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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David Hung (file photo)

Mas­ter deal­mak­er David Hung re­tools a SPAC sedan in­to a fi­nanc­ing mus­cle ve­hi­cle that leaves his can­cer start­up with $850M and a place on Wall Street

It’s only right that one of the industry’s top dealmakers just completed one of the biggest SPAC-related deals in the pipeline.

David Hung, of Medivation fame, has completed a back flip into the market, merging with EcoR1 Capital’s SPAC Panacea and landing neatly on Wall Street with an $NUVB stock ticker after filling out the blank check in his name. In addition to the $144 million held in the SPAC — provided none of the investors opt out — Hung is getting ahold of $500 million more being chipped in by a slate of institutional investors who feel that Hung could have the keys to another Medivation-style success.

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Roche finds a home for a new, $500M man­u­fac­tur­ing lo­gis­tics hub, promis­ing 500 jobs

Roche is pouring $500 million into its Canadian headquarters in Mississauga, Ontario to set up a new hub that will coordinate logistics for its global supply chain.

Over the 5-year investment, the Swiss pharma giant expects to add 200 jobs over next year and another 300 by the end of 2023.

Introduced as a $190 million global pharmaceutical development site in 2011, the campus currently houses Roche’s Canadian commercial unit as well as product development, global procurement and pharma informatics. The new expansion will see it organize manufacturing across 13 plants and 11 sites, according to FiercePharma.

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Pfizer CEO Albert Bourla (Drew Angerer/Getty Images)

Pfiz­er is on the verge of claim­ing a multi­bil­lion-dol­lar first-mover ad­van­tage with their Covid-19 vac­cine — an­a­lyst

From the beginning, Pfizer CEO Albert Bourla eschewed government funding for his Covid-19 vaccine work with BioNTech, willing to take all the $2 billion-plus risk of a lightning-fast development campaign in exchange for all the rewards that could fall its way with success. And now that the pharma giant has seized a solid lead in the race to the market, those rewards loom large.

SVB Leerink’s Geoff Porges has been running the numbers on Pfizer’s vaccine, the mRNA BNT162b2 program that the German biotech partnered on. And he sees a $3.5 billion peak in windfall revenue next year alone. Even after the pandemic is brought to heel, though, Porges sees a continuing blockbuster role for this vaccine as people around the world look to guard against a new, thoroughly endemic virus that will pose a permanent threat.

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UP­DAT­ED: CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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UP­DAT­ED: Brazil­ian vol­un­teer in As­traZeneca Covid-19 vac­cine tri­al has died in the place­bo arm — re­ports

A volunteer in AstraZeneca’s Covid-19 vaccine trial in Brazil has died, Brazilian health authorities said Wednesday, triggering fresh alarms over the future of the Oxford program. But later reports noted that the death was in the placebo group and AstraZeneca issued word that there were no concerns about continuing the study.

The Brazilian health agency Anvisa said it had received data from an investigation into the issue, per a Reuters report. The report was then updated citing a Brazilian newspaper with unnamed sources saying that the volunteer was in the placebo arm of the trial.