President Trump (AP Images)

FDA takes the lead on defin­ing es­sen­tial un­der Trump's 'Buy Amer­i­can' ex­ec­u­tive or­der — as phar­ma warns of sup­ply chain dis­rup­tion

Pres­i­dent Don­ald Trump has signed an ex­ec­u­tive or­der de­tail­ing how the fed­er­al gov­ern­ment should help on-shore drug man­u­fac­tur­ing — and the FDA will play a cen­tral role.

The agency now has three months to draw up the list of “es­sen­tial med­i­cines, med­ical coun­ter­mea­sures, and their crit­i­cal in­puts” that the US must have avail­able at all times. Var­i­ous de­part­ments and agen­cies are then di­rect­ed to buy these drugs and their in­gre­di­ents from Amer­i­can man­u­fac­tur­ers.

While lim­it­ed to fed­er­al con­tracts and not pri­vate ones, the ex­ec­u­tive or­der threat­ens to dis­rupt the glob­al sup­ply chain, phar­ma groups warn.

“The ad­min­is­tra­tion is forc­ing bio­phar­ma­ceu­ti­cal com­pa­nies to shift their crit­i­cal at­ten­tion and re­sources away from COVID-19 work to fo­cus on mak­ing sub­stan­tial changes to their busi­ness mod­els nec­es­sary to com­ply with this and oth­er re­cent ex­ec­u­tive or­ders,” PhRMA said in a state­ment. “In­creas­ing U.S. man­u­fac­tur­ing of med­i­cines is a laud­able goal, but it can­not hap­pen overnight and should not come at the ex­pense of in­no­va­tion or Amer­i­cans’ ac­cess to the med­i­cines they need.”

Gil Roth, pres­i­dent of the Phar­ma & Bio­phar­ma Out­sourc­ing As­so­ci­a­tion, said it’s un­clear how ben­e­fi­cial or detri­men­tal the or­der will be to the CD­MOs he rep­re­sents. The sec­tor is strong, he not­ed, and too many ques­tions re­main as to whether new poli­cies will tru­ly ad­dress the bot­tle­becks faced by the in­dus­try.

“Our mem­bers, both on the API and dosage form sides, want a lev­el play­ing field, not a com­mand econ­o­my, and we hope the fed­er­al gov­ern­ment will take in­to ac­count this sec­tor’s ex­ist­ing do­mes­tic ca­pac­i­ty for in­no­v­a­tive and gener­ic drugs be­fore im­ple­ment­ing more man­dates and fund­ing new ca­pac­i­ty at tax­pay­ers’ ex­pense,” he wrote in a state­ment.

Ex­perts have es­ti­mat­ed that as much 90% of the ac­tive phar­ma­ceu­ti­cal in­gre­di­ents in gener­ic drugs sold on US soil is made abroad, al­though there are no of­fi­cial fig­ures.

Un­der the new White House di­rec­tive, gov­ern­ment agen­cies will have to start sub­mit­ting an­nu­al re­ports of where they source their prod­ucts. De­spite the sweep­ing “Buy Amer­i­can” head­line, waivers ap­ply in spe­cif­ic cas­es: when it’s against the pub­lic in­ter­est, the drugs are not pro­duced in the US at a large enough scale, the do­mes­tic prod­ucts would in­crease the cost by more than 25%, or there’s a na­tion­al emer­gency.

Stephen Hahn

FDA com­mis­sion­er Stephen Hahn has sev­er­al more tasks on his list.

Work­ing with HHS Sec­re­tary Alex Azar, he’s charged with ac­cel­er­at­ing FDA ap­proval for do­mes­tic pro­duc­ers of es­sen­tial meds, “in­clud­ing those need­ed for in­fec­tious dis­ease and CBRN threat pre­pared­ness and re­sponse.” In ad­di­tion, he’s al­so ex­pect­ed to is­sue guid­ances for de­vel­op­ing ad­vanced man­u­fac­tur­ing tech­niques, con­duct more site in­spec­tions over­seas and refuse drugs from non-com­pli­ant fa­cil­i­ties.

It re­mains to be seen how the new on-shoring ini­tia­tive will af­fect drug prices, an­oth­er key is­sue Trump is cam­paign­ing on.

For its part, the White House in­sists that low­er­ing the reg­u­la­to­ry bar­ri­er to do­mes­tic phar­ma­ceu­ti­cal man­u­fac­tur­ing (and per­haps rais­ing the bar for for­eign play­ers) and in­vest­ing in new tech would keep drug prices low.

But John and Lau­ra Arnold, who have backed a non-prof­it gener­ic drug man­u­fac­tur­er, are adopt­ing a wait-and-see stance.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to discuss their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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