Pan­dem­ic trav­el re­stric­tions force FDA to de­lay a BLA re­view — in­volv­ing a site in­spec­tion in Ko­rea

In an un­usu­al move, the FDA is ap­par­ent­ly de­fer­ring ac­tion on a BLA be­cause it was un­able to con­duct a man­u­fac­tur­ing site in­spec­tion on time.

Spec­trum Phar­ma­ceu­ti­cals’ ap­pli­ca­tion for eflape­gras­tim is now in lim­bo as it works with the FDA to ex­pe­dite its trip to the Han­mi plant in South Ko­rea, where the drug sub­stance is man­u­fac­tured. Giv­en pan­dem­ic trav­el re­stric­tions, reg­u­la­tors were un­able to in­spect the site dur­ing the cur­rent re­view cy­cle, ac­cord­ing to a state­ment put out Mon­day morn­ing — two days af­ter the orig­i­nal PDU­FA date.

“Spec­trum has con­firmed with the FDA that this is not a Com­plete Re­sponse Let­ter,” it added in a re­lease.

As Covid-19 was rear­ing its head in the US — long be­fore the FDA was em­broiled in heat­ed de­bates about po­lit­i­cal in­ter­fer­ence in its drug and vac­cine re­view process — com­mis­sion­er Stephen Hahn had warned that with many staffers work­ing on Covid-19 ac­tiv­i­ties, the agency may slow down its re­view of oth­er drugs. But al­most six months lat­er, this ap­pears to be the first time it had to miss a dead­line due to the pan­dem­ic.

Al­ready chris­tened Rolon­tis, Spec­trum’s drug is a long-act­ing gran­u­lo­cyte colony-stim­u­lat­ing fac­tor (G-CSF) de­signed to boost a type of white blood cells in can­cer pa­tients re­ceiv­ing myelo­sup­pres­sive drugs. That makes it a ri­val to Am­gen’s Neu­las­ta, a $4 bil­lion fran­chise that has come un­der biosim­i­lar threats.

The two Phase III tri­als that went in­to the BLA test­ed eflape­gras­tim in pa­tients with ear­ly-stage breast can­cer who ex­pe­ri­enced neu­trope­nia due to chemother­a­py, sug­gest­ing non-in­fe­ri­or­i­ty and sim­i­lar safe­ty pro­file to Neu­las­ta.

Man­u­fac­tur­ing has been a sore point for the eflape­gras­tim pro­gram. Spec­trum chose to re­scind its mar­ket­ing ap­pli­ca­tion last March af­ter the FDA re­quest­ed more da­ta.

Joe Tur­geon

This time around, though, pres­i­dent and CEO Joe Tur­geon sees the ball in the oth­er court.

“The man­u­fac­tur­ing fa­cil­i­ty is ready for in­spec­tion and we are ea­ger to as­sist the FDA in com­plet­ing their as­sess­ment as soon as pos­si­ble,” he said.

What does this mean for oth­er NDAs or BLAs in the pipeline? We’re still wait­ing to hear back from the FDA, but Sal­im Syed — who’s been track­ing Bris­tol My­ers Squibb’s CVR like a hawk — doesn’t see a read-through to the all-im­por­tant ap­pli­ca­tion for Juno’s BC­MA-tar­get­ing CAR-T. His ra­tio­nale may of­fer some in­sight in­to who’s more or less like­ly to be af­fect­ed:

  1. JCAR017 has both break­through ther­a­py des­ig­na­tion and pri­or­i­ty re­view. Rolon­tis doesn’t. This im­plies that the FDA prob­a­bly has JCAR017 high­er up on the peck­ing or­der for re­views it needs to con­duct.
  2. Both of JCAR017’s man­u­fac­tur­ing fa­cil­i­ties (the JuMP fa­cil­i­ty in Both­well, WA and vec­tor CMO fa­cil­i­ty in TX) are in the US, so prob­a­bly eas­i­er to get to them than to the Rolon­tis South Ko­re­an fa­cil­i­ty.
  3. As far as we can tell, the JuMP fa­cil­i­ty has al­ready been in­spect­ed. We would sus­pect the TX fa­cil­i­ty may have al­ready been in­spect­ed as well or would be pri­or to the JCAR017 PDU­FA of No­vem­ber 16, 2020.

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.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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An­oth­er warn­ing let­ter for Lupin as FDA iden­ti­fies de­fi­cien­cies at In­dia-based site

With few new details of what needs fixing, Lupin disclosed last week that the FDA recently sent a warning letter to its Tarapur, India-based site.

After an inspection from March 22 to April 4, Lupin disclosed in an April stock filing that it received a Form 483 with four observations, but it didn’t offer any details on the observations.

Similar to comments made in April, the company said last week it does not believe the FDA slap will disrupt its drug supplies or the existing revenues from operations of this facility.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Silviu Itescu, Mesoblast CEO

Mesoblast sends in im­proved po­ten­cy as­say, look­ing to re­sub­mit to FDA on acute graft-ver­sus-host dis­ease drug

In 2020, the FDA rejected Mesoblast’s remestemcel-L, or Ryoncil, its lead candidate for pediatric acute graft-versus-host disease (aGVHD) that didn’t respond to steroids. The FDA raised a number of concerns, first objecting to Mesoblast’s single arm, open-label trial, though regulators struggled to describe how a randomized trial would work, since pediatricians and parents were reluctant to put children with aGVHD in a placebo arm.

Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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