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

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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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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J&J ad­comm live blog: J&J faces ques­tions on old­er adults, asymp­to­matic in­fec­tion, long-term im­mu­ni­ty

The FDA adcom has advanced to the free-for-all question stage of the hearing, and, as they did for Moderna and Pfizer, committee members are raising questions about the lingerings surrounding the vaccine.

In J&J’s case, one of those unknowns is a group of participants who appeared to respond worse to the vaccine: Those over 60 with commodities. In that group, the vaccine was only 42% effective at stopping disease starting 28 days after vaccination.