EMA and FDA his­tor­i­cal­ly agree on just about every new drug ap­proval, but is that slow­ly chang­ing?

The EMA and FDA con­cur more than 90% of the time in their de­ci­sions to ap­prove new drugs, ac­cord­ing to a new study from EMA and FDA of­fi­cials that looked at 107 ap­pli­ca­tions from 2014 to 2016.

In just eight of the 107 ap­pli­ca­tions, the FDA ini­tial­ly de­clined to ap­prove a new drug or bi­o­log­ic while the EMA ap­proved it, al­though in all eight of those cas­es, the FDA end­ed up ap­prov­ing that drug or bi­o­log­ic. And in one case (Take­da’s Nin­laro (ix­a­zomib) for mul­ti­ple myelo­ma), the FDA ap­proved the treat­ment and the EMA ini­tial­ly did not, but lat­er did.

“Over­all, tak­ing ac­count of the re­sub­mit­ted and re­ex­am­ined ap­pli­ca­tions, the EMA and the FDA had fi­nal dis­cor­dant mar­ket­ing au­tho­riza­tion de­ci­sions for two drugs: cori­fol­litropin al­fa and ataluren,” the study notes, as both were ap­proved by the EMA and not the FDA.

More re­cent­ly, how­ev­er, the EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use (CHMP) adopt­ed neg­a­tive opin­ions for two drugs in 2018 that were ap­proved by FDA in 2017, and one sick­le cell drug in 2019 that was al­so pre­vi­ous­ly ap­proved by FDA. In ad­di­tion, CHMP raised ques­tions about Mit­subishi Tan­abe Phar­ma’s treat­ment for amy­otroph­ic lat­er­al scle­ro­sis, which with­drew its ap­pli­ca­tion this year, and which was ap­proved by FDA in 2017.

“Di­ver­gence in ap­proval de­ci­sions, type of ap­proval, and ap­proved in­di­ca­tion were pri­mar­i­ly due to dif­fer­ences in agen­cies’ con­clu­sions about ef­fi­ca­cy based on re­view of the same da­ta or dif­fer­ing clin­i­cal da­ta sub­mit­ted to sup­port the ap­pli­ca­tion,” the study pub­lished in Clin­i­cal Phar­ma­col­o­gy & Ther­a­peu­tics found.

In the more re­cent case of the sick­le cell drug, the FDA said its ap­proval was based on a tri­al show­ing that pa­tients treat­ed with En­dari (glu­t­a­mine) ex­pe­ri­enced few­er hos­pi­tal vis­its for sick­le cell crises, on av­er­age, when com­pared to place­bo. But the EMA’s CHMP said it “con­sid­ered that the main study did not show that [glu­t­a­mine] was ef­fec­tive at re­duc­ing the num­ber of sick­le cell crises or hos­pi­tal vis­its.”

The study al­so notes how the FDA more com­mon­ly grant­ed ac­cel­er­at­ed ap­provals (12/25 in on­col­o­gy and 5/8 in hema­tol­ogy) than the EMA grant­ed con­di­tion­al mar­ket­ing au­tho­riza­tion or au­tho­riza­tion un­der ex­cep­tion­al cir­cum­stances (7/25 in on­col­o­gy and 2/8 in hema­tol­ogy).

But sub­mis­sions in these ar­eas of­ten oc­curred lat­er to the EMA than the FDA, and of­ten in­clud­ed ad­di­tion­al clin­i­cal tri­als or more ma­ture da­ta from the same clin­i­cal tri­al than were sub­mit­ted to the FDA. “In those in­stances, the EMA was more like­ly than the FDA to grant stan­dard ap­proval (where­as the FDA is­sued ac­cel­er­at­ed ap­proval) or a broad­er in­di­ca­tion,” the study said.

The study al­so found the EMA had a high­er rate of first-cy­cle ap­provals than the FDA, and the re­searchers “ob­served re­mark­able sim­i­lar­i­ty in the ba­sic sci­en­tif­ic and da­ta in­ter­pre­ta­tion is­sues raised by the FDA and the EMA dur­ing re­views of the same ap­pli­ca­tions. Specif­i­cal­ly, most of the FDA’s sec­ond cy­cle ap­provals (i.e., ap­provals af­ter re­sub­mis­sion of the ap­pli­ca­tions) were based on sub­mis­sion by the spon­sor of the same ad­di­tion­al da­ta that EMA had re­ceived dur­ing its ini­tial re­view ei­ther from the start or fol­low­ing re­quest af­ter clock‐stops.”

In their dis­cus­sion of the re­sults, the study au­thors al­so note the study’s lim­i­ta­tions, such as on­ly us­ing two years’ worth of da­ta. But over­all, the two agen­cies are com­mu­ni­cat­ing and work­ing to­geth­er more close­ly than in years past.

“The high rate of con­ver­gence in the au­tho­ri­sa­tion of new med­i­cines at EMA and the FDA is the re­sult of ex­pand­ed in­vest­ment in di­a­logue and co­op­er­a­tion since 2003 and has fos­tered align­ment be­tween the EU and the US with re­spect to de­ci­sions on mar­ket­ing au­tho­ri­sa­tions, while both agen­cies eval­u­ate ap­pli­ca­tions in­de­pen­dent­ly of each oth­er,” said Zaide Frias, head of the EMA’s hu­man med­i­cines eval­u­a­tion di­vi­sion.

So­cial im­age: Shut­ter­stock, AP


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.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

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Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Ben Zimmer, Priovant CEO

Roivant un­veils lat­est spin­out as Pfiz­er en­trusts JAK1/TYK2 to Pri­o­vant

In November, Pfizer disclosed it’s spun out the Phase II dual JAK1/TYK2 inhibitor to a startup formed in collaboration with an unnamed, experienced partner.

We now know who the partner is. And as Pfizer and Roivant officially take the wraps off Priovant Therapeutics, the companies reveal that they have started two registrational trials of the drug, brepocitinib, as part of a broader plan to develop a big, first-in-class franchise spanning multiple orphan and specialty autoimmune diseases.

No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP Images)

Up­dat­ing the Covid-19 vac­cine: FDA of­fers a qual­i­fied thumbs-up ahead of ad­comm

The FDA’s adcomm of outside vaccine experts will meet tomorrow to discuss how to protect the US from a likely coming wave of Omicron cases in the fall and winter, and whether to deploy vaccines that specifically target the Omicron variant.

While the data so far are limited, the FDA sounded an upbeat tone in the briefing documents on Pfizer/BioNTech’s candidates, released this weekend ahead of the VRBPAC meeting.

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.