FDA to re­quire cer­tain IND safe­ty re­ports be sub­mit­ted to FAERS

The FDA on Tues­day is­sued draft guid­ance re­quir­ing spon­sors to sub­mit in­ves­ti­ga­tion­al new drug (IND) safe­ty re­ports for se­ri­ous and un­ex­pect­ed sus­pect­ed ad­verse events to the FDA Ad­verse Event Re­port­ing Sys­tem (FAERS) start­ing 24 months af­ter the guid­ance is fi­nal­ized.

Cur­rent­ly, such re­ports are sub­mit­ted to the FDA in elec­tron­ic com­mon tech­ni­cal doc­u­ment (eCTD) for­mat us­ing PDF files, which the FDA says are “in­ef­fi­cient and la­bor in­ten­sive” to re­view and track.

“Sub­mis­sion of this im­por­tant safe­ty in­for­ma­tion to FAERS as struc­tured da­ta el­e­ments will im­prove the FDA’s abil­i­ty to re­view and track these po­ten­tial safe­ty sig­nals that oc­cur dur­ing the con­duct of clin­i­cal tri­als, and will pro­vide spon­sors with a re­port­ing for­mat that is con­sis­tent with In­ter­na­tion­al Coun­cil for Har­mon­i­sa­tion (ICH) guide­lines and re­port­ing re­quire­ments to oth­er reg­u­la­to­ry agen­cies,” the FDA writes.

The switch to sub­mit­ting IND safe­ty re­ports for se­ri­ous and un­ex­pect­ed sus­pect­ed ad­verse events is part of the FDA’s ef­fort to im­ple­ment the elec­tron­ic sub­mis­sion re­quire­ments of Sec­tion 745A(a) of the Fed­er­al Food, Drug, and Cos­met­ic Act.

While fed­er­al agen­cies are not typ­i­cal­ly able to es­tab­lish new re­quire­ments via guid­ance, Con­gress gave the FDA ex­plic­it au­tho­riza­tion to do so to es­tab­lish the for­mat for elec­tron­ic sub­mis­sions un­der Sec­tion 745A(a).

For now, the FDA says spon­sors will be able to con­tin­ue sub­mit­ting such IND safe­ty re­ports via eCTD, though spon­sors may vol­un­tar­i­ly sub­mit the re­ports to FAERS be­fore the re­quire­ment is in ef­fect.

Once ef­fec­tive, the guid­ance will su­per­sede the FDA’s fi­nal guid­ance Pro­vid­ing Reg­u­la­to­ry Sub­mis­sions in Elec­tron­ic For­mat — Cer­tain Hu­man Phar­ma­ceu­ti­cal Prod­uct Ap­pli­ca­tions and Re­lat­ed Sub­mis­sions Us­ing the eCTD Spec­i­fi­ca­tions for IND safe­ty re­ports that fall with­in the scope of the new guid­ance.

Un­der the pol­i­cy laid out in the guid­ance, spon­sors must sub­mit IND safe­ty re­ports to FAERS via the FDA’s Elec­tron­ic Sub­mis­sion Gate­way (ESG) or Safe­ty Re­port­ing Por­tal (SRP).

The FDA notes that the guid­ance does not ap­ply to IND safe­ty re­ports for any­thing oth­er than se­ri­ous and un­ex­pect­ed sus­pect­ed ad­verse events, such as re­ports de­tail­ing find­ings from oth­er stud­ies, find­ings from an­i­mal or in vit­ro test­ing or of in­creased rate of oc­cur­rence of se­ri­ous sus­pect­ed ad­verse re­ac­tions.

Ad­di­tion­al­ly, the FDA says that sub­mis­sions for non­com­mer­cial INDs will be ex­empt from the elec­tron­ic sub­mis­sion re­quire­ments un­der Sec­tion 745A(a), though the agency en­cour­ages such spon­sors to do so.

Along­side the draft guid­ance, the FDA is­sued a sep­a­rate tech­ni­cal con­for­mance guide meant to sup­ple­ment the draft guid­ance by de­tail­ing the for­mat that should be used for IND safe­ty re­ports sub­mit­ted to FAERS as in­di­vid­ual case safe­ty re­ports (IC­SRs) and IND safe­ty re­ports that should be sub­mit­ted to the FDA in elec­tron­ic com­mon tech­ni­cal doc­u­ment (eCTD) for­mat.


RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

Jim Scholefield via PR Newswire

Mer­ck los­es its chief dig­i­tal of­fi­cer, spot­light­ing tal­ent hunt for the hottest ti­tle in Big Phar­ma

Over the last few years we’ve seen the chief digital officer title become one of the hottest commodities in Big Pharma as global organizations hunt the best talent to sharpen the cutting edge of their tech platforms.

But Merck just discovered how hard it may be to keep them focused on pharma.

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Tim Mayleben (file photo)

Es­pe­ri­on's goldilocks cho­les­terol fight­er wins FDA ap­proval — will its 'tra­di­tion­al' pric­ing ap­proach spur adop­tion?

It’s more effective than decades-old statins but not as good as the injectable PCSK9 — the goldilocks treatment for cholesterol-lowering, bempedoic acid, has secured FDA approval.

Its maker, Esperion Therapeutics, is betting that their pricing strategy — a planned list price of between $10 to $11 a day — will help it skirt the pushback the PCSK9 cholesterol fighters, Repatha and Praluent, got from payers for their high sticker prices.

The sky-high expectations for the pair of PCSK9 drugs that were first approved in 2015 quickly simmered — and despite a 60% price cut, coupled with data showing the therapies also significantly cut cardiovascular risk, sales have not really perked up.

Esperion is convinced that by virtue of being a cheaper oral therapy, bempedoic acid will hit that sweet spot in terms of adoption.

“We’re kind of like the old comfortable shoe,” Esperion’s chief commercial officer Mark Glickman remarked in an interview with Endpoints News ahead of the decision date. “It’s an oral product, once-daily and nontitratable — these are things that just resonate so true with patients and physicians and I think we’ve kind of forgotten about that.”

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James Collins, Broad Institute via Youtube

UP­DAT­ED: A space odyssey for new an­tibi­otics: MIT's ma­chine learn­ing ap­proach

Drug development is complex, expensive and comes with lousy odds of success — but in most cases, if you make it across the finish line brandishing a product with an edge (and play your cards right) it can be a lucrative endeavor.

As it stands, the antibiotic market is cursed — it harbors the stink of multiple bankruptcies, a dearth of innovation, and is consequently barely whetting the voracious appetites of big pharma or venture capitalists. Enter artificial intelligence — the biopharma industry’s cure-all for the pesky process of making a therapeutic, including data mining, drug discovery, optimal drug delivery, and addressable patient population.

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Gilead los­es two more patent chal­lenges on HIV pill, set­ting up court­room fight in Delaware

Gilead sustained two more losses in their efforts to rid themselves of an activist-backed patent lawsuit from the US government over a best-selling HIV pill.

Urged on by activists seeking to divert a portion of Gilead’s revenue to clinics and prevention programs, the Department of Health and Human Services made a claim to some of the patents for the best-selling HIV prevention drug, Truvada, also known as PrEP. Gilead responded by arguing in court that HHS’s patents were invalid.

Today, the US Patent and Trademark Office ruled that Gilead was likely to lose the last two of those challenges as well. The USPTO ruled against Gilead on the first two patents earlier this month.

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Kathy High (file photo)

Gene ther­a­py pi­o­neer Kathy High has left Spark af­ter com­plet­ing $4.3B union with Roche

Kathy High dedicated the past seven years of her life shepherding experimental gene therapies she’s developed at Children’s Hospital of Philadelphia toward the market as president and head of R&D at Spark Therapeutics. Now that the biotech startup is fully absorbed into Roche — with an FDA approval, a $4.3 billion buyout and a promising hemophilia program to boast — she’s ready to move on.

Roche confirmed her departure with Endpoints News and noted “she will take some well-deserved time off and then will begin a new chapter in a sabbatical at a university.”

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Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

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Christos Kyratsous (via LinkedIn)

He built a MERS treat­ment in 6 months and then the best Ebo­la drug. Now Chris­tos Kyrat­sous turns his sights on Covid-19

TARRYTOWN, NY — In 2015, as the Ebola epidemic raged through swaths of West Africa, Kristen Pascal’s roommates sat her down on their couch and staged an intervention.

“Are you sure this is what you want to be doing with your life?” she recalls them asking her.

Special report

Pascal, a research associate for Regeneron, had been coming home at 2 am and leaving at 6 am. At one point, she didn’t see her roommate for a week. For months, that was life in Christos Kyratsous’ lab as the pair led a company-wide race to develop the first drug that could effectively treat Ebola before the outbreak ended. For Pascal, that was worth it.

“I’m ok, I don’t have Ebola,” Pascal told them. “I see that death toll rising and I can’t not do something about it.”

Last August, Regeneron learned they had succeeded: In a large trial across West Africa, their drug, REGN-EB3, was vastly more effective than the standard treatments. It was surprise news for the company, coming just 10 months into a trial they thought would take several years and a major victory in the global fight against a deadly virus that killed over 2,000 in 2019 and can carry a mortality rate of up to 90%.

For Kyratsous and Pascal, though, it brought only fleeting reprieve. Just four months after the NIH informed them REGN-EB3 worked, Kyratsous was back in his office reading the New York Times for updates on a new outbreak on another continent, and wondering alongside Pascal and senior management whether it was time to pull the trigger again.

In late January, as the death toll swelled and the first confirmed cases outside China broke double digits, they made a decision. Soon they were back on the phone with the multiple government agencies and their coronavirus partners at the University of Maryland’s Level 3 bio lab. The question was simple: Can Kyratsous and his team use a process honed over two previous outbreaks, and create a treatment before the newest epidemic ends? Or worse, if, as world health experts fear, it doesn’t vanish but becomes a recurrent virus like the flu?

“Christos likes things immediately,” Matt Frieman, Regeneron’s coronavirus collaborator at the University of Maryland, told Endpoints. “That’s what makes us good collaborators: We push each other to develop things faster and faster.”

Kristen Pascal (Regeneron)

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The first time Regeneron tried to respond to a global outbreak, it was something of a systems test, Kyratsous explains from his office at Regeneron’s Tarrytown headquarters. Kyratsous, newly promoted, has crammed it with photos of his family, sketches of viral vectors and a shark he drew for his 3-year-old son. He speaks rapidly – an idiosyncrasy his press person says has only been aggravated this afternoon by the contents of his “Regeneron Infectious Diseases”-minted espresso glass – and he gesticulates with similar fluidity, tumbling through antibodies, MERS, the novel coronavirus, Ebola-infected monkeys.

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Bank­rupt an­tibi­otics mak­er Ar­a­digm turns to old part­ner/in­vestor for fi­nal $3M fire sale

Grifols once paid Aradigm $26 million for a stake in its inhaled antibiotics. But with Aradigm now in bankruptcy, the Spanish drugmaker is dishing out a final $3.2 million to buy it all.

The fire sale — which comes one year after Aradigm filed for Chapter 11 following a regulatory trifecta for disaster — will see Grifols obtain assets and IP to Apulmiq (formerly Pulmaquin and Linhaliq in Europe), Lipoquin and free ciprofloxacin. In addition to waiving its claims in the bankruptcy case, Grifols also agreed to milestone payments up to $3 million more upon any regulatory approvals.