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: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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US month­ly costs for biosim­i­lars 'sub­stan­tial­ly high­er' than Ger­many or Switzer­land, JA­MA re­search finds

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

Kirk Myers is shown in a still image from a new film series showcasing the efforts of HIV advocates funded by Gilead.

Gilead spot­lights HIV projects and the com­mu­ni­ty lead­ers dri­ving them in new mi­ni-doc­u­men­tary films

Gilead is going behind the scenes of some of the HIV initiatives it funds through grants in a new film series narrated by the people helming the projects.

The first four films and leaders come from across the US — Arianna Lint in Florida and Puerto Rico, Cleve Jones in San Francisco, June Gipson in Mississippi and Kirk Myers in Texas. Their HIV-focused efforts range from addressing unmet needs of the transgender community to delivering social services and high-quality health care in underserved communities.

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EMA pulls an opi­oid from the 1950s used to treat dry cough

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Bay­er starts work on $43M+ ex­pan­sion of OTC man­u­fac­tur­ing site in Penn­syl­va­nia

German pharma giant Bayer will be looking to make a significant investment into one of its US plants that produces over-the-counter drugs.

Bayer announced that it will spend $43.6 million to expand its facility in Myerstown, PA, a small town east of Harrisburg. Bayer plans to increase the site by 70,000 square feet and will have room for the installation of eight packaging lines and an area to install rooftop solar panels. The project is expected to be completed by 2025 and will add around 50 to 75 jobs.