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.

UP­DAT­ED: A small, ob­scure biotech just won big with their IPO. In this mar­ket. Are you kid­ding me?

How could a small, largely unknown biotech that emerged from stealth mode just months ago with early-stage cancer programs jump onto Wall Street in the middle of a Category 6 financial hurricane and sail through with a $165 million IPO?

And what does that mean for the rest of the industry waiting to see just how much damage global lockdowns will wreak on clinical development?

The biotech is a company called Zentalis. The crew there nabbed an $85 million crossover round late last year — notably waiting 5 years before waving the numbers around to attract attention, according to my read of a FierceBiotech story. Perceptive joined in, but the syndicate was not in general the kind of marquee affair that gets tongues wagging.

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Gilead CEO Daniel O'Day attends a meeting with the President and other biopharma leaders at the White House on March 2, 2020 (AP Photo)

Ramp­ing up glob­al pro­duc­tion of remde­sivir, Gilead CEO Dan O’Day de­tails plans to dis­trib­ute 1.5M dos­es to fight Covid-19 — for free

Gilead is still some days away from turning the card on its first round of data on remdesivir’s ability to fight severe cases of Covid-19, but the big biotech is ramping up an emergency supply of a million courses of therapy as it starts free distribution of the drug to tens of thousands of patients under their compassionate use and expanded access program as well as clinical trials.

In his latest open letter posted over the weekend, Gilead CEO Dan O’Day outlined how the company has been successful in cutting production time on remdesivir while repurposing some of their own facilities and turning to contract manufacturers to build a near-term supply of 1.5 million doses. They are still working on efficacy and dosing, but that supply could cover 140,000 courses of treatment. That supply, he added, would be more widely available following a potential approval.

Bob Nelsen at the Milken Institute Global Conference on April 29, 2019 in Beverly Hills, California. (Photo by Michael Kovac/Getty Images)

ARCH chief Bob Nelsen has $1.5B to prove 2 sim­ple points: ‘We’re in the most in­no­v­a­tive time ever’ and in­vestors are stay­ing

ARCH co-founder and managing director Bob Nelsen has a well known yen for the home run swing, betting big on potentially transformative meds and tech and the biotech teams he helps bring together. He thrives and bleeds on the cutting edge. And now Nelsen and the ARCH group have debuted 2 big funds to prove that this is the time for the best of biotech to shine — deadly pandemic be damned.

Two new funds, ARCH Venture Fund X and ARCH Venture Fund X Overage, gathered a combined $1.46 billion. And that’s a record. ARCH Venture Fund IX and ARCH Venture Fund IX Overage closed in 2016 with a combined $1.1 billion. ARCH Venture Fund VIII and ARCH Venture Fund VIII Overage closed in 2014 with a combined $560 million.

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Have a new drug that promis­es to fight Covid-19? The FDA promis­es fast ac­tion but some de­vel­op­ers aren't hap­py

After providing an emergency approval to use malaria drugs against coronavirus with little actual evidence of their efficacy or safety in that setting, the FDA has already proven that it has set aside the gold standard when it comes to the pandemic. And now regulators have spelled out a new approach to speeding development that promises immediate responses in no uncertain terms — promising a program offering the ultimate high-speed pathway to Covid-19 drug approvals.

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Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Aaron Royston, venBio

In­vest­ing in the time of coro­n­avirus: the good, the bad and the hope­ful, as biotech VC firms close funds worth $3B

Apart from disrupting biopharma R&D and regulatory timelines, the coronavirus pandemic has inevitably ravaged financial markets and eroded investor risk appetite. Investing in the time of coronavirus feels reckless, but if biotech venture funds are any indication, the time is ripe.

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Drug dis­cov­ery in the age of coro­n­avirus

Developing new drugs is incredibly hard. That’s why, despite superhuman efforts from the industry, we’re still looking at 12-18 months minimum before we can realistically hope for a vaccine for Covid-19, and probably months before there’s a proven viable drug treatment.

But our increasing ability to begin to industrialize the drug discovery and development process through an engineering approach means that we have more hope for speeding up this process than ever before — and not just to defeat coronavirus, but to benefit the development of all new medicines in the future.