Once stiff-armed at the FDA, new man­age­ment of­fers Am­i­cus a warm em­brace for Galafold -- priced at $315,000

It turns out the FDA wasn’t done Fri­day with the his­toric ap­proval for Al­ny­lam’s world’s-first RNAi drug. Af­ter the mar­ket close, Am­i­cus Ther­a­peu­tics $FOLD re­vealed that the agency had al­so come through with an OK for Galafold (mi­gala­s­tat), a con­tro­ver­sial new med that had ini­tial­ly been firm­ly re­ject­ed by the agency.

On sec­ond glance, which came with an ac­cel­er­at­ed pri­or­i­ty re­view and some red-car­pet treat­ment at the agency, the FDA de­ter­mined that Galafold had every­thing they were look­ing for in a drug for Fab­ry dis­ease and an amenable galac­tosi­dase al­pha gene vari­ant based on in vit­ro as­say da­ta.

Up to half of the 3,000 or so US pa­tients with Fab­ry’s dis­ease could qual­i­fy for this drug, which treats a rare en­zyme de­fi­cien­cy. And they will be con­sid­er­ing a drug that comes with a price of $315,000 a year, ac­cord­ing to a Reuters re­port on Mon­day morn­ing. That may look high, but it’s well with­in the usu­al norm for six-fig­ure prices as­so­ci­at­ed with ther­a­pies aimed at a rare dis­ease. Am­i­cus is al­so out­lin­ing plans to keep fu­ture price hikes in line with in­fla­tion.

The FDA’s ini­tial po­si­tion on Galafold was that it had no rea­son to be ap­proved at this stage, look­ing for a new Phase III to jus­ti­fy a mar­ket­ing OK. Now it came through with a green light sev­er­al days ahead of the ac­cel­er­at­ed PDU­FA date.

John Crow­ley

Click on the im­age to see the full-sized ver­sion


Am­i­cus CEO John Crow­ley em­ployed some du­bi­ous ar­gu­ments as he fought ve­he­ment­ly against the re­jec­tion and a de­mand for a new Phase III study of gas­troin­testi­nal symp­toms. Ini­tial­ly, the CEO had pub­licly told in­vestors it would take 2 years to come up with the added da­ta, which is about av­er­age for the task. But he pri­vate­ly told Janet Wood­cock and oth­ers that the ac­tu­al time­line would be 5 to 7 years, which made the de­vel­op­ment pro­gram im­prac­ti­cal.

Crow­ley not on­ly lob­bied the new­ly elect­ed Pres­i­dent Don­ald Trump, he al­so im­plored the new­ly-se­lect­ed FDA di­rec­tor Scott Got­tlieb about the un­ten­able de­mands in a let­ter. Got­tlieb’s staff lat­er said the chief nev­er saw those ar­gu­ments, send­ing it along to the right of­fi­cials.

This was one of three drug pro­grams that the FDA re­versed it­self on in the months af­ter 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 all three won their ap­provals, though Lil­ly was left with a green light on a low dose that may have few tak­ers.

There wasn’t a hint of any past ob­jec­tions in the FDA ap­proval re­lease Fri­day evening.

“Thus far, treat­ment of Fab­ry dis­ease has in­volved re­plac­ing the miss­ing en­zyme that caus­es the par­tic­u­lar type of fat buildup in this dis­ease. Galafold dif­fers from en­zyme re­place­ment in that it in­creas­es the ac­tiv­i­ty of the body’s de­fi­cient en­zyme,” said Julie Beitz, M.D., di­rec­tor of the Of­fice of Drug Eval­u­a­tion III in FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search.

And you can be sure that at least a few reg­u­la­tors are pray­ing this dra­mat­ic about-face won’t come back to haunt them lat­er.

Nick Galakatos, Blackstone global head of life sciences

Nick Galakatos and the Black­stone team now have a record $4.6B to in­vest in bio­phar­ma, with a big fo­cus on push­ing com­pa­nies over the top

Nick Galakatos and his team at Blackstone Life Sciences have seen their biggest opportunities swell up in mostly established players who don’t have all the money they need to accomplish everything on the to-do list. And right now, with the industry booming, that’s a long list with some hefty needs.

The Blackstone team has neatly tied up the largest private fund ever raised in life sciences for making big dreams come true in biopharma. Late Thursday, Blackstone put out word that they had closed their highly anticipated fund with the projected $4.6 billion all in.

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Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Top biotech an­a­lyst projects a gloomy out­look for Pfiz­er's JAK port­fo­lio

Many in the pharma world are hoping — better yet, expecting — JAK inhibitors to provide one of the next big boons for the industry. Few have invested as heavily in this area as Pfizer, which boasts a portfolio including Xeljanz and at least five mid-to-late stage candidates in the pipeline.

But a top Wall Street analyst is pumping the brakes on just how much good fortune is in store for the Big Pharma.

Gilead boasts of pos­i­tive remde­sivir da­ta on mor­tal­i­ty — but their analy­sis pro­vokes the skep­tics

Gilead is surging again off data that suggest its antiviral remdesivir might improve survival.

The new data come from an analysis Gilead conducted comparing the death rate and recovery time of patients in one of its remdesivir trials to a group of 800 patients “with similar baseline characteristics and disease severity” who received only standard-of-care around the same time. The result, they said, suggested that patients who received remdesivir had a 62% better chance at surviving than those who did not.

Hal Barron, GSK

Win or lose on the mar­ket­ing OK, the FDA just gunned down GSK’s bright hopes for their BC­MA ther­a­py

The FDA’s ODAC — the Oncologic Drugs Advisory Committee — has a well-known bias in favor of adding new cancer drugs to the market, even if efficacy is at best marginal and serious safety issues demand careful management.

Doctors want as many arrows in their quiver as they can get. And when patients are dying after failing multiple drugs, why not give it a go one more time?

GlaxoSmithKline, though, is about to test out how their new BCMA antibody drug conjugate belantamab mafodotin can do after being mauled in an in-house FDA review, ahead of the Tuesday expert panel discussion. Even if the agency goes ahead with an expected green light, this drug will likely be constrained to a small niche — icing any plans they may have for making waves in oncology anytime soon.

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Covid-19 roundup: BioN­Tech go­ing head-to-head with Mod­er­na as PhI­II mR­NA launch looms; Tri­al on Shin­zo Abe’s once-fa­vorite an­tivi­ral is in­con­clu­sive

It’s a race to the Phase III finish line now for the 2 leading mRNA vaccines in the pipeline for Covid-19.

BioNTech chief Ugur Sahin told the Wall Street Journal that his company will start Phase III testing of their vaccine later this month, setting them up to lateral the data to regulators before the end of this year.

That puts them essentially on the exact same schedule as Moderna is dedicated to. The Massachusetts rival to BioNTech also expects to launch Phase III this month. Lots of rumors have circulated about delays and conflict among the scientists advancing the Moderna jab, but the biotech has consistently stuck to its plan to start a late-stage pivotal this month.

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Andrew Kruegel, Kures president and co-founder (Columbia Tech Ventures via Vimeo)

Af­ter psilo­cy­bin and ke­t­a­mine, a new biotech comes along de­vel­op­ing a drug Scott Got­tlieb fought

Andrew Kruegel was six years into his chemistry work at Columbia University, when, one day in August 2016, he learned he might have only 30 days before the government made him destroy his research.

Kruegel had been studying kratom, a leaf long used in Southeast Asia as a stimulant or for pain. It had opioid-like properties, he found, but seemed to offer pain relief without the addictive potential or respiratory side effects of traditional opioids — a riddle that might help illuminate how human opioid receptors work.

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The home run count: The $100M+ mega-round boom in biotech in­spired a $7.3B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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