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.

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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Federal Trade Commission commissioner Rohit Chopra testifies on Capitol Hill (AP Photo/Susan Walsh)

FTC clears Bris­tol-My­ers’ $74B deal to buy Cel­gene — but Dems sig­nal a po­ten­tial hard shift against Big Phar­ma M&A

Bristol-Myers Squibb’s record $74 billion takeover of Celgene is a done deal. And it will all be over — except for the lingering complaints from die-hard Celgene investors — on Wednesday.

Like much else that’s going on in Washington these days, the vote among the 5 FTC commissioners split along party lines, with the 3 Republicans voting to clear the way and the 2 Democrats steamed over what they see as a major M&A move that will lessen competition and innovation. And that split has big implications for the M&A side of the business if the Dems take the White House in 2020.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

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What does $62B buy you these days? A lot, says Take­da ex­ecs as the phar­ma play­er promis­es a block­buster R&D fu­ture

First comes the $62 billion buyout. Then comes the asset auction and reorganization to pay down debt. Now comes the detailed pledge of a bigger, brighter future in drug development.

That’s where Takeda finds itself on R&D day today, about 11 months after closing on their Shire acquisition. R&D chief Andy Plump is joining CEO Christophe Weber and other top members of the team to outline a new set of priorities in the greatly expanded pipeline at Takeda, which has jumped into the top ranks of the world’s pharma giants in the wake of the Shire deal.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.

Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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