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.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.