FDA so­lic­its in­dus­try in­put for OND pol­i­cy pri­or­i­ties

A Fri­day FDA no­tice is so­lic­it­ing pol­i­cy sug­ges­tions from the in­dus­try that re­view staff at the FDA’s Of­fice of New Drugs (OND) can im­ple­ment in the near fu­ture.

To hear from in­dus­try on “spe­cif­ic, ac­tion­able pol­i­cy sug­ges­tions that could be im­ple­ment­ed in the near-term,” the FDA is so­lic­it­ing pre­sen­ta­tions for dis­cus­sions at a pub­lic meet­ing set via the Fed­er­al Reg­is­ter no­tice as well as invit­ing com­ments on the top­ics that the no­tice dis­cuss­es.

The meet­ing is set for 7 No­vem­ber, with the goal of so­lic­it­ing sug­ges­tions on read­i­ly and broad­ly ap­plic­a­ble poli­cies across var­i­ous ther­a­peu­tic ar­eas or dis­eases. This dif­fers from the in­put in­dus­try can of­fer for guid­ances and frame­works on high-pol­i­cy ini­tia­tives, such as to in­cor­po­rate re­al-world ev­i­dence and the sci­ence of pa­tient in­put in­to reg­u­la­to­ry de­ci­sion-mak­ing.

The fo­cus of the no­tice is cen­tered on di­rect­ing the at­ten­tion to five top­ics that are of par­tic­u­lar in­ter­est for the sug­ges­tions to aid OND in fo­cus­ing on pol­i­cy pri­or­i­ties while the FDA- and Cen­ter for Drug Eval­u­a­tion and Re­search (CDER)-wide high-pol­i­cy ini­tia­tives are un­der­way.

The FDA so­lic­its in­put about where OND can fa­cil­i­tate ad­di­tion­al guid­ance or sci­en­tif­ic dis­cus­sion in the near fu­ture to im­prove clar­i­ty and pro­mote ef­fec­tive drug de­vel­op­ment “for var­i­ous clin­i­cal ar­eas linked by a shared ther­a­peu­tic con­text,” such as drugs that are in­tend­ed for the treat­ment of se­ri­ous, life-threat­en­ing rare dis­eases. Spe­cif­ic sug­ges­tions can al­so be about where OND can un­der­take guid­ance or pol­i­cy de­vel­op­ment sup­port­ing drug de­vel­op­ment for dis­eases that are cur­rent­ly un­a­menable to tar­get­ed ther­a­pies. In­dus­try can share views on guid­ance, ben­e­fits, draw­backs to ex­tend­ing nov­el tri­al de­signs to ad­di­tion­al ther­a­peu­tic ar­eas and how to pro­mote ef­fec­tive drug de­vel­op­ment pro­grams when such ap­proach­es cause un­cer­tain­ty-re­lat­ed ten­sion.

The re­main­ing top­ic of par­tic­u­lar in­ter­est to the agency re­lates to the FDA guid­ances that are not dis­ease- or in­di­ca­tion-spe­cif­ic. The FDA wants to hear from those who may “be­lieve that OND re­view di­vi­sions are im­ple­ment­ing these guid­ances in dif­fer­ent ways.” It seeks rec­om­men­da­tions for top­ics where, “This may re­flect a need for guid­ance re­vi­sion or ad­di­tion­al pol­i­cy de­vel­op­ment.”

“Con­sis­tent with FDA’s broad­er ini­tia­tives and mod­ern­iza­tion ef­forts, the clin­i­cal and sci­en­tif­ic lead­ers of OND are seek­ing sug­ges­tions on where we can pro­vide reg­u­la­to­ry clar­i­ty to pro­mote in­no­v­a­tive and ef­fec­tive drug de­vel­op­ment across mul­ti­ple ther­a­peu­tic ar­eas,” the agency states in the no­tice. The move comes amid the mod­ern­iza­tion of OND, a FY 2019 pri­or­i­ty, and CDER.

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.

So­cial im­age: FDA, AP Im­ages

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.

DB­V's peanut pre­ven­tion patch ap­proach­es key stage of ap­proval process

Almost a year and a half after DBV Technologies pulled its peanut allergy immunotherapy patch from FDA review, the biotech will get their day in court. The FDA has scheduled an advisory committee hearing for May 15.

In the two-horse race to develop the first immunotherapy for peanut allergy, DBV had the early lead, filing an NDA for their patch in 2018. But on December 20 of that year, the company withdrew their application after, they said, meeting with regulators and determining they had not submitted “sufficient detail regarding data on manufacturing procedures and quality controls.” Aimmune filed their BLA 3 days later and won approval as the first immunotherapy for peanuts this month.

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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eF­FEC­TOR co-founder Siegfried Re­ich jumps to Turn­ing Point; An­to­nio Gual­ber­to starts post-Ku­ra ca­reer at Ei­sai sub­sidiary H3

→ Seasoned drug hunter Siegfried Reich has left eFFECTOR, the biotech he co-founded to discover selective translation regulators, to take up the CSO role at Turning Point Therapeutics.

“I have followed the progress Turning Point has made, and was drawn to its focus on drug discovery and its work to advance the pipeline,” Reich told Endpoints News. “I was also impressed by the depth of its management team and board, many of whom I have worked with before.”

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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Say good­bye to Toca­gen, strug­gling brain can­cer biotech to re­verse merge with Forte Bio­sciences

Five months after a huge Phase III failure triggered mass layoffs at the company, Tocagen will sign itself out of existence.

The biotech, once focused on brain cancer, announced it has signed a reverse merger agreement with Forte Biosciences, a biotech tackling atopic dermatitis and other inflammatory skin diseases. Tocagen’s stock shot up 85% on the news, although that only translated to a 41-cent bump for a company that saw the last of its value go poof in September. The new company will trade under the ticker $FBRX.

JJ Bienaimé (BioMarin via YouTube)

Speedy re­view and no ad­comm for Bio­Mar­in's pi­o­neer­ing he­mo­phil­ia gene ther­a­py

BioMarin’s keenly anticipated hemophilia A gene therapy — which CEO JJ Bienaimé envisions pricing at an eye-watering $2 million to $3 million a shot — has secured red carpet treatment at the FDA.

The therapy, fondly called valrox, has won priority review — and there the agency has indicated there are no plans for an advisory committee meeting. The US regulator’s final decision is expected by August 21. If approved, it will be the pioneering gene therapy for hemophilia.

“While the company…indicated that FDA does not currently plan on convening an advisory committee meeting to discuss the merits of the application, we caution that the possibility of such still remains as the agency digs into its review, which could be interpreted negatively by the Street,” BMO Capital Market’s George Farmer wrote.

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