FDA (via Endpoints News)

FDA tar­gets faster re­views for biosim­i­lar sup­ple­ments, draft guid­ance says

The FDA  on Thurs­day pub­lished new draft guid­ance ex­plain­ing how it will speed its re­view of biosim­i­lar or in­ter­change­able ap­pli­ca­tion sup­ple­ments, which can be used to up­date the ini­tial biosim­i­lar ap­proval when it is for few­er than all the ref­er­ence prod­uct’s li­censed con­di­tions of use.

The 9-page draft de­tails how FDA can­not li­cense a biosim­i­lar or in­ter­change­able prod­uct for an in­di­ca­tion pro­tect­ed by or­phan-drug ex­clu­siv­i­ty or pe­di­atric ex­clu­siv­i­ty un­til the ex­pi­ra­tion of that ex­clu­siv­i­ty. Ap­pli­cants al­so may de­cide not to seek li­cen­sure of a pro­posed biosim­i­lar or pro­posed in­ter­change­able prod­uct for con­di­tions of use that are pro­tect­ed by a patent or for oth­er rea­sons.

For in­stance, when Pfiz­er’s In­flec­tra (in­flix­imab-dyyb) and Mer­ck’s Ren­flex­is (in­flix­imab-ab­da) were first ap­proved, FDA ap­proved them for all the same in­di­ca­tions as J&J’s Rem­i­cade (in­flix­imab), ex­cept pe­di­atric ul­cer­a­tive col­i­tis. This in­di­ca­tion was ex­clud­ed be­cause J&J at the time en­joyed an FDA-grant­ed pe­ri­od of or­phan ex­clu­siv­i­ty. Sim­i­lar­ly, when Am­gen won ap­proval for its Hu­mi­ra (adal­i­mum­ab) biosim­i­lar Am­je­vi­ta (adal­i­mum­ab-at­to), the com­pa­ny did not seek ap­proval for sev­er­al in­di­ca­tions be­cause of or­phan ex­clu­siv­i­ty.

But af­ter that ex­clu­siv­i­ty or patent ex­pires, an ap­pli­cant may de­cide to sub­mit a sup­ple­ment to the li­censed 351(k) bi­o­log­ics li­cense ap­pli­ca­tion (BLA) for the pre­vi­ous­ly pro­tect­ed in­di­ca­tion.

And al­though FDA com­mit­ted to re­view­ing and act­ing on orig­i­nal 351(k) BLA sup­ple­ments with clin­i­cal da­ta with­in 10 months of re­ceipt, the agency now says such sup­ple­ments will be re­viewed and act­ed up­on in a 6-month time­frame.

“As not­ed, the Bs­U­FA II Goals Let­ter de­scribes a 10-month goal date for orig­i­nal 351(k) BLA sup­ple­ments with clin­i­cal da­ta. How­ev­er, at this time, FDA be­lieves that a re­view time­frame of 6 months will gen­er­al­ly be ap­pro­pri­ate for a sup­ple­ment to a li­censed 351(k) BLA seek­ing li­cen­sure of the biosim­i­lar or in­ter­change­able prod­uct for an ad­di­tion­al con­di­tion of use that has been pre­vi­ous­ly li­censed for the ref­er­ence prod­uct, as­sum­ing the sup­ple­ment does not raise nov­el re­view is­sues,” the draft says.

If an ap­pli­cant does not want FDA to take ac­tion on a 351(k) BLA or sup­ple­ment be­fore a spec­i­fied date, the ap­pli­cant should re­quest that FDA re­frain from act­ing on the ap­pli­ca­tion, the draft adds.

To make such a re­quest, the draft says that an ap­pli­cant should in­clude the fol­low­ing lan­guage on the cov­er let­ter of their BLA or sup­ple­ment, in bold type­face and promi­nent­ly placed above the body of the cov­er let­ter:

351(k) BLA ac­tion tim­ing re­quest: [Ap­pli­cant Name] re­quests that FDA not take ac­tion on this [ap­pli­ca­tion/sup­ple­ment] be­fore [spec­i­fied date].

The draft al­so ex­plains how the pro­posed la­bel­ing for a biosim­i­lar will de­pend on whether the ap­pli­cant is seek­ing li­cen­sure for all (or few­er) of the con­di­tions of use li­censed for the ref­er­ence prod­uct. And an ap­pli­cant may sub­mit a jus­ti­fi­ca­tion as to why, in the ap­pli­cant’s view, the draft la­bel­ing meets the re­quire­ments for ap­proval, con­sid­er­ing the con­di­tions of use for which the ap­pli­cant is seek­ing li­cen­sure.

FDA al­so says that it rec­om­mends that an ap­pli­cant seek­ing li­cen­sure for a pro­posed in­ter­change­able prod­uct seek li­cen­sure for all of the ref­er­ence prod­uct’s li­censed con­di­tions of use when pos­si­ble.

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.


Zachary Brennan

managing editor, RAPS

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.

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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Deborah Dunsire

The fourth CGRP mi­graine drug is here. Time for Lund­beck to prove it's worth $2B

They may be late, but Lundbeck is now officially in the game for preventing migraine with CGRP drugs.

The FDA has OK’d eptinezumab, the prize in Lundbeck’s $2 billion acquisition of Alder. Like rival offerings from Amgen/Novartis, Eli Lilly and Teva, the antibody blocks the calcitonin gene-related peptide, which is believed to dilate blood vessels in the brain and cause pain.

It will now be sold as Vyepti. The company has yet to announce a price. Amgen and Novartis had set the wholesale acquisition cost of their pioneering Aimovig at $6,900 for a year’s supply before raising it slightly this year; Lilly and Teva had followed suit with Emgality and Ajovy.

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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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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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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)

Click on the image to see the full-sized version

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.