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.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

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Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

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George Yancopoulos (L) and Len Schleifer (Regeneron)

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Lon­za look­ing to build on 'd­if­fer­en­ti­at­ed ad­van­tage' in Covid-19, CD­MO mar­ket­place in 2021

It’s not new for Lonza, the Swiss CDMO nearing its quasquicentennial anniversary, to be in the upper echelon of the biotech manufacturing industry.

But 2020 — as it was for many CDMOs — was a special year even by Lonza’s standards. The company inked a deal to produce 1 billion worldwide doses of Moderna’s Covid-19 vaccine and tapped pharma vet Pierre-Alain Ruffieux to lead its operations, moves which have allowed Lonza to make a myriad of other deals that will continue to ramp up its global production capacity.