Trump at­tacks “slow and bur­den­some” drug ap­proval process, trig­ger­ing a pitch from Am­i­cus CEO

Pres­i­dent Don­ald Trump out­lined his po­lit­i­cal agen­da for 2017 in his ad­dress to a joint ses­sion of Con­gress Tues­day night, aim­ing a broad­side di­rect­ed straight at the “bur­den­some” drug ap­proval process at the FDA. And a promi­nent CEO in the rare dis­ease field stepped in­to Trump’s high pow­ered spot­light to urge a change in the way the agency han­dles or­phan drugs while mak­ing a pitch for a speedy re­ver­sal of a painful set­back hand­ed to him by reg­u­la­tors last year.

In a se­ries of point­ed high­lights dur­ing tonight’s speech, most of Trump’s com­ments on health­care fo­cused on the now stan­dard at­tack on Oba­macare and a de­mand to re­peal and re­place his pre­de­ces­sor’s plans on health in­sur­ance. But he re­served a few mo­ments of heat­ed crit­i­cism re­served specif­i­cal­ly for the FDA and gov­ern­ment re­form.

“We must elim­i­nate the bur­den­some ap­proval process for life-sav­ing drugs so that more lives can be saved,” was in­clud­ed in Trump’s ad­vance list of talk­ing points.

“(O)ur slow and bur­den­some ap­proval process at the FDA keeps too many ad­vances, like the one that saved Megan’s life, from reach­ing those in need,” Trump told his au­di­ence, turn­ing to Am­i­cus CEO John Crow­ley’s daugh­ter Megan in the au­di­ence.

Megan Crow­ley suf­fers from Pompe dis­ease, a rare ail­ment that Am­i­cus has set out to treat with two drugs now in Phase I. Crow­ley found­ed No­vazyme, ac­quired by Gen­zyme, which went on to de­vel­op an en­zyme re­place­ment ther­a­py Megan Crow­ley still us­es.

“If we slash the re­straints, not just at the FDA but in gov­ern­ment,” Trump told Con­gress, “we will be blessed with far more mir­a­cles like Megan.”

Ear­li­er in the speech Trump al­so lashed out once again at the “ar­ti­fi­cial­ly high price of drugs,” which he has vowed to rein in.

That all fits close­ly with the pres­i­dent’s in­sis­tence — out­lined in a re­cent ses­sion with Big Phar­ma ex­ec­u­tives — that the drug ap­proval process needs to be dereg­u­lat­ed, in­sist­ing that slash­ing FDA rules was need­ed in or­der to cut drug prices. But the in­dus­try has re­coiled at the idea that the FDA’s rules gov­ern­ing de­vel­op­ment need to be gut­ted, leav­ing many won­der­ing just how ex­treme a makeover the pres­i­dent has in mind.

Am­i­cus $FOLD rep­re­sents a biotech com­pa­ny which would ben­e­fit sig­nif­i­cant­ly from a sharp change in di­rec­tion at the FDA. Last year the com­pa­ny’s shares were sent in­to a tail­spin af­ter it was forced to mount a new safe­ty study for migala­s­tat, a new drug for Fab­ry dis­ease, af­ter reg­u­la­tors at the FDA de­mand­ed more da­ta in a move that will push back any ap­proval by years. Months be­fore, the Eu­ro­pean Com­mis­sion gave it a green light across the At­lantic.

“(I)t is trou­bling that in re­cent years, rare dis­ease re­search is be­com­ing what the Or­phan Drug Act sought to change: an en­ter­prise too ex­pen­sive to jus­ti­fy in­vest­ment,” the Am­i­cus CEO wrote in a piece for the Ob­serv­er. “It takes now or­phan drugs as much time to com­plete Phase III stud­ies (the stage of drug de­vel­op­ment where ef­fi­ca­cy is es­tab­lished) as it does for non-or­phans. At the same time, the av­er­age clin­i­cal tri­al cost per pa­tient is 14 times high­er for or­phan drugs com­pared to those that are not. The FDA’s reg­u­la­tion of the or­phan de­vel­op­ment process is be­com­ing less flex­i­ble, less ef­fi­cient and less pa­tient-cen­tered.”

“Sim­i­lar­ly,” he added, “my com­pa­ny, Am­i­cus Ther­a­peu­tics, saw the de­vel­op­ment of Galafold, for the treat­ment of Fab­ry dis­ease, slowed by the same process in the U.S., adding mil­lions of dol­lars in cost and de­lay­ing treat­ment for years, de­spite the drug’s ap­proval as a pre­ci­sion med­i­cine in Eu­rope last year. The FDA has de­clared that the on­ly path­way to avail­abil­i­ty for Fab­ry pa­tients in the Unit­ed States is pred­i­cat­ed on yet an­oth­er study. And while we do that, pa­tients wait. All get sick­er. And some will die.”

In­vestors picked up on the pos­si­bil­i­ties, bid­ding Am­i­cus’ shares up 7% Wednes­day morn­ing.

Now the biotech in­dus­try wants to know if a new FDA com­mis­sion­er ap­point­ed by Trump would change the bal­ance of safe­ty and ef­fi­ca­cy da­ta need­ed for an ap­proval, changes which may ben­e­fit in­di­vid­ual com­pa­nies but erode the gold stan­dard long de­mand­ed by the agency.

Crow­ley has al­ready made it clear that he be­lieves he’s ready to start mar­ket­ing the drug in the US “to­day.” His fate this year will in­flu­ence the en­tire in­dus­try, for bet­ter or worse, as Trump and the na­tion’s law­mak­ers go about trans­form­ing the pres­i­dent’s rhetoric in­to po­lit­i­cal re­al­i­ty.


Re­marks As De­liv­ered:

An in­cred­i­ble young woman is with us this evening who should serve as an in­spi­ra­tion to us all.

To­day is Rare Dis­ease day, and join­ing us in the gallery is a Rare Dis­ease Sur­vivor, Megan Crow­ley.  Megan was di­ag­nosed with Pompe Dis­ease, a rare and se­ri­ous ill­ness, when she was 15 months old.  She was not ex­pect­ed to live past five.

On re­ceiv­ing this news, Megan’s dad, John, fought with every­thing he had to save the life of his pre­cious child.  He found­ed a com­pa­ny to look for a cure, and helped de­vel­op the drug that saved Megan’s life.  To­day she is 20 years old — and a sopho­more at Notre Dame.

Megan’s sto­ry is about the un­bound­ed pow­er of a fa­ther’s love for a daugh­ter.

But our slow and bur­den­some ap­proval process at the Food and Drug Ad­min­is­tra­tion keeps too many ad­vances, like the one that saved Megan’s life, from reach­ing those in need.

If we slash the re­straints, not just at the FDA but across our Gov­ern­ment, then we will be blessed with far more mir­a­cles like Megan.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.