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.

Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.

Sen. Elizabeth Warren (D-MA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

Sen. Eliz­a­beth War­ren urges FTC to 'scru­ti­nize' two phar­ma buy­outs

Sen. Elizabeth Warren (D-MA) is calling on senior Federal Trade Commission officials to “closely scrutinize” two proposed pharma mergers.

Warren expressed concern over “rampant consolidation in the pharmaceutical industry,” in particular Amgen’s $28 billion plans to take over Horizon Therapeutics, and Indivior’s proposed acquisition of Opiant for $145 million upfront, in a letter to FTC Chair Lina Khan and Commissioners Alvaro Bedoya and Rebecca Kelly Slaughter earlier this week.