New drug ap­pli­ca­tions pile up with de­lays as there's no easy fix for the in­spec­tion back­log

The FDA’s back­log of drug man­u­fac­tur­ing fa­cil­i­ty in­spec­tions con­tin­ues to be a sore spot for the agency, as FDA said Mon­day that a to­tal of 52 new drug (but no bi­o­log­ics) ap­pli­ca­tions still re­main de­layed due to the back­log from the pan­dem­ic.

And while the agency has main­tained that it won’t is­sue a CRL for a de­layed in­spec­tion, the num­ber of de­layed ap­pli­ca­tions is ris­ing as FDA said in May that 48 new drug ap­pli­ca­tions have been de­layed due to FDA’s in­abil­i­ty to con­duct pre-ap­proval, pre-mar­ket, or pre-li­cense in­spec­tions as of last March.

The agency’s in­abil­i­ty to work over­seas has al­so hurt its out­put well in­to 2021, with just 37 for­eign drug in­spec­tions be­tween April and Sep­tem­ber 2021. Pri­or to the pan­dem­ic, the FDA typ­i­cal­ly con­duct­ed about 1,000 for­eign drug in­spec­tions per year.

“FDA be­gan the new fis­cal year with on­go­ing trav­el re­stric­tions and oth­er un­cer­tain­ties con­tin­u­ing to im­pact over­sight op­er­a­tions. FDA is con­tin­u­ing to com­plete mis­sion-crit­i­cal work, pri­or­i­tize oth­er high­er-tiered in­spec­tion­al needs (e.g., for-cause in­spec­tions), and car­ry out sur­veil­lance in­spec­tions us­ing risk-based ap­proach­es for eval­u­at­ing pub­lic health im­pact,” FDA said in its new re­port.

The agency has sought to shift to more re­mote as­sess­ments of fa­cil­i­ties, but the le­gal de­f­i­n­i­tion of an in­spec­tion is one that is con­duct­ed on site.

“As we have done through­out the pan­dem­ic, FDA will use every op­tion avail­able to meet our reg­u­la­to­ry re­spon­si­bil­i­ties and pro­tect the pub­lic health, in­clud­ing con­tin­ued col­lab­o­ra­tion with state, lo­cal, trib­al, ter­ri­to­r­i­al, and for­eign reg­u­la­to­ry part­ners,” the agency added.

De­spite the back­log, there were a few bright spots in the re­port too.

In ear­ly 2021, the FDA es­ti­mat­ed that more than 15,000 sur­veil­lance in­spec­tions through­out the US had been post­poned thanks to the Covid-19 pan­dem­ic. The agency pre­dict­ed that it would com­plete 14% of those in­spec­tions, in­clud­ing 26% of the re­main­ing hu­man and an­i­mal med­ical do­mes­tic sur­veil­lance in­spec­tions. The agency ex­ceed­ed that num­ber two-fold, it re­port­ed, a com­ple­tion rat­ing of 134%.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Vlad Coric, Biohaven CEO

Vlad Coric charts course for new Bio­haven with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Ying Huang, Legend CEO

Lentivi­ral vec­tor ramp-up: J&J and Leg­end to in­vest $500M in New Jer­sey man­u­fac­tur­ing to sup­port Carvyk­ti

In response to a question on manufacturing scale at Legend Biotech’s R&D day yesterday, the company’s top exec said its partnership with Johnson & Johnson will be doubling its investment in its New Jersey manufacturing center and will be investing a total of $500 million.

With an eye on their BCMA-directed CAR-T therapy Carvykti (cilta-cel), approved in February as a fifth-line treatment for multiple myeloma, Legend CEO Ying Huang said that the ramp-up in production and the decision to manufacture its own lentiviral vectors — currently in shortage worldwide — means they won’t have to deal with that shortage.

Kite Phar­ma gets FDA to sign off on new Cal­i­for­nia-based vec­tor man­u­fac­tur­ing fa­cil­i­ty

Kite Pharma just got FDA approval to kick off operations at a new manufacturing campus.

The cancer-focused, CAR-T cell therapy player made the announcement Monday, saying that the federal regulatory agency gave the green light to Kite’s 100,000 square-foot, retroviral vector manufacturing facility in Oceanside, CA.

Kite’s global head of technical operations Chris McDonald tells Endpoints News that the facility has been in the works for about four years, after Kite teamed up with its parent company Gilead. Gilead acquired Kite Pharma for just shy of $12 billion in 2017.

Take­da to pull key hy­poparathy­roidism drug from the mar­ket en­tire­ly by end of 2024 af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024, but the entire inventory will be available until depleted or expired, a company spokesperson said via email.

Christophe Bourdon, Leo Pharma CEO

Leo Phar­ma looks 'be­yond the skin' in atopic der­mati­tis aware­ness cam­paign

As Leo Pharma aims to take on heavyweight champ Dupixent in atopic dermatitis, the company is launching “AD Days Around the World,” an awareness campaign documenting real patient stories across Europe.

The project, unveiled on Monday, spotlights four patients: Marjolaine, Laura, Julia and África from France, Italy, Germany and Spain, respectively, in short video clips on the challenges of living with AD, the most common form of eczema.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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