President Donald Trump and FDA Commissioner Stephen Hahn (AP Images)

FDA is­sues fi­nal rule al­low­ing im­por­ta­tion of drugs from Cana­da — but al­so keeps the pow­er to re­voke it

Just over a month away from the pres­i­den­tial elec­tion, the FDA has is­sued a fi­nal reg­u­la­tion ful­fill­ing Pres­i­dent Trump’s promise to let states im­port cer­tain pre­scrip­tion drugs from Cana­da.

On Thurs­day, Trump told a crowd in North Car­oli­na that the new rule goes in­to ef­fect “to­day.” But the pub­lished reg­u­la­tion states that it won’t take ef­fect for 60 days. And even then, it could be a while be­fore cheap­er drugs make it across the bor­der.

Ac­cord­ing to the rule, states, ter­ri­to­ries, tribes, phar­ma­cists and whole­salers would need to set up new pro­grams and ap­ply to im­port the drugs, which would have to meet sev­er­al spec­i­fi­ca­tions. The prod­ucts would need to be re­la­beled and test­ed for “au­then­tic­i­ty and degra­da­tion,” ac­cord­ing to the FDA. And the pro­grams must demon­strate “sig­nif­i­cant cost re­duc­tions” to the con­sumer.

“If you put Cana­di­an drugs on a dogsled and point­ed it in the di­rec­tion of Flori­da, the dogs would ar­rive long be­fore any drugs through this reg­u­la­to­ry pro­pos­al,” for­mer HHS of­fi­cial Chris Meekins told the Wash­ing­ton Post back in De­cem­ber.

Trump signed an ex­ec­u­tive or­der in Ju­ly di­rect­ing the FDA to com­plete the “rule­mak­ing process” to al­low the im­por­ta­tion of Cana­di­an pre­scrip­tion drugs. Un­der a law es­tab­lished in 2003, such im­por­ta­tion is on­ly al­lowed if cer­ti­fied by the HHS sec­re­tary. Alex Azar has be­come the first sec­re­tary to for­mal­ly do so.

Drugs that have been ap­proved by Cana­da’s Health Prod­ucts and Food Branch and meet the con­di­tions of an FDA-is­sued NDA or AN­DA will be el­i­gi­ble. In a press brief­ing on Fri­day, HHS chief of staff Bri­an Har­ri­son said that safe­ty has been “at the fore­front of our ef­forts.”

“We’ve cre­at­ed smart safe op­por­tu­ni­ties for Amer­i­cans to se­cure low­er cost of drugs through im­por­ta­tion as no pres­i­dent has ever done be­fore,” Har­ri­son said in the brief­ing.

But oth­ers are skep­ti­cal that the reg­u­la­tion will be ef­fec­tive in low­er­ing drug costs. In a New Eng­land Jour­nal of Med­i­cine Per­spec­tive piece pub­lished in May, Wash­ing­ton Uni­ver­si­ty as­so­ciate law pro­fes­sor Rachel Sachs and Uni­ver­si­ty of Michi­gan law pro­fes­sor Nicholas Bagley sug­gest­ed the Trump ad­min­is­tra­tion could be do­ing more to make drugs af­ford­able.

“Far from a bold ini­tia­tive to help peo­ple af­ford their pre­scrip­tion drugs, the pro­pos­al seems de­signed to al­low the Trump ad­min­is­tra­tion to claim that it is tak­ing ac­tion, even as it op­pos­es con­gres­sion­al leg­is­la­tion that would sharply curb some drug prices,” the piece states.

Bagley and Sachs al­so point­ed out that the ini­tial pro­pos­al lacked guid­ance on how states can prove the im­port­ed drugs will re­duce costs. Cana­di­an reg­u­la­tors could change their rules to dis­cour­age ex­por­ta­tion, or sav­ings could ben­e­fit the whole­salers or the health care providers pre­scrib­ing the drugs, rather than pa­tients, they said. “Im­por­ta­tion might still save mon­ey, at least for some drugs, but a raw com­par­i­son of prices in the Unit­ed States and Cana­da pro­vides very thin sup­port for that con­clu­sion.”

Alexan­der Gaffney of Politi­co’s Agen­cy­IQ not­ed that the fi­nal­ized reg­u­la­tion in­cludes lan­guage which could al­low the FDA to re­voke im­por­ta­tion pro­grams.

“This will be a game-chang­er for Amer­i­can se­niors,” Trump tout­ed Thurs­day in a speech pre­sent­ing his Amer­i­ca First health­care plan. “And by al­low­ing you to do this through Cana­da, we’re do­ing it very, very quick­ly. So it goes very fast.”

The pres­i­dent al­so promised $200 pre­scrip­tion drug coupons to Medicare ben­e­fi­cia­ries, which he said will come from $6.6 bil­lion in sav­ings that do not cur­rent­ly ex­ist — or from phar­ma’s cof­fers.

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: FDA anoints Gilead­'s remde­sivir as the Covid-19 treat­ment win­ner, hand­ing down full ap­proval — de­spite some deep skep­ti­cism

Seven months into the Covid-19 pandemic, the race to develop a treatment for the disease that’s proved to be the biggest health crisis in a century has an officially designated winner: Gilead. And they’re picking up the prize — worth billions in peak sales — despite a major study that concluded the drug was no help in reducing the number of people who die from the virus.

The FDA handed down a thumbs-up for remdesivir, the company announced Thursday afternoon, as the drug becomes the first fully approved treatment for Covid-19 in the US. Remdesivir, to be marketed as Veklury, will come with a label for treatment in adults and children older than 12 in Covid-19 cases that require hospitalization.

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Stephen Hoge, Moderna president (Moderna)

On morn­ing of FDA Covid-19 ad­comm, Mod­er­na com­pletes PhI­II en­roll­ment, putting them neck-and-neck with Pfiz­er

Weeks away from a potential EUA application, Moderna announced they have completed enrollment in their 30,000-person Phase III Covid-19 vaccine trial, with over a third of volunteers non-white and a quarter over the age of 65.

The announcement caps what has been the most closely-watched recruitment race in the history of drug development, as Pfizer and Moderna rushed to get enough volunteers to prove whether or not experimental vaccines could actually protect people from contracting Covid-19. Pfizer reached that mark on Sept. 15. Moderna said around the same time that they would slow down enrollment to ensure they enrolled enough participants from minority and at-risk groups.

Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.