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.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists

President Donald Trump, who seems intent on announcing a COVID-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

Anthony Fauci (AP Images)

A press of­fi­cer at An­tho­ny Fau­ci’s NI­AID was un­masked as a hard-right Covid troll. He just re­tired to­day

William B Crews had been a public affairs specialist at the NIH’s National Institute of Allergy and Infectious Diseases.

That ended today when he informed the agency of his decision to retire, after he was identified as the managing editor at RedState, a prominent Trump loyalist website.

Crews’ RedState duties are performed under the alias streiff. While enjoying the benefits of pseudonymity, he disparaged and worked against NIAID with an incendiary level of rhetoric in the midst of a pandemic.

Donald Trump, AP

Covid-19 roundup: Trump sug­gests Pfiz­er vac­cine could be first ap­proved; VBI Vac­cines inks de­vel­op­ment deal with Cana­da

President Donald Trump commented Monday morning that Pfizer’s Covid-19 vaccine candidate could be the first to win approval by regulators.

During an interview on a Fox News’ morning show, the president said Pfizer was doing “very well” when asked which candidate could be approved, according to a Reuters report. He added that J&J could follow up afterward, saying “they’ll probably be a little later.”

Is­raeli biotech rais­es $57M to go where cur­rent BRAF in­hibitors can't, with back­ing from No­var­tis, SR One

For the blockbuster potential of Novartis’ Tafinlar and Pfizer’s Braftovi, all the BRAF inhibitors on the market so far only target V600 mutations — which accounts for roughly 50% of patients.

Israeli biotech Novellus now has $57 million to develop a drug that they say can help the other 50% who have everything else.

The Series C will fund a Phase II trial for PLX-8394, a “paradox breaker” that could block RAF without activating MAPK signaling. In a Phase I trial, a patient with a BRAF fusion saw their tumor go away after taking the drug, allowing Novellus to hit the ground running.