Regulatory channel feed

Bio­phar­ma ex­ecs rou­tine­ly shun re­veal­ing the hard truth when it comes to RTFs. And some peo­ple at the FDA think that's a big prob­lem

Within drug development circles, there’s nothing quite as embarrassing as a refuse-to-file letter from the FDA. So it’s probably no big surprise to hear that the average biopharma outfit prefers to hide under any available rock before frankly explaining what derailed their applications right from the start.

FDA staffers took a close look at 103 RTF letters — out of 2,475 applications, or 4% of the total — that were sent privately to drug developers, counting 644 reasons why the agency barred the door to a new drug application. The agency’s hands are tied on public disclosures, though, leaving it to the companies to offer an explanation. And that has left companies free to either publicly ignore what happened, or simply mislead people about the setback.

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Mallinck­rodt be­comes the lat­est vic­tim of the FDA's pan­dem­ic-re­lat­ed de­lays as it push­es off ap­pli­ca­tion for skin graft

With Covid-19 locking down travel across the world, not even drug safety regulators have been spared from costly delays. For the FDA, whose inspection schedule keeps the trains on time for new marketing approvals, those delays are continuing to prove costly.

The FDA has pushed review of Mallinckrodt’s allogeneic skin graft for burns, dubbed StrataGraft, after the agency admitted it wasn’t able to conduct a plant inspection in the needed timeframe for a decision, the drugmaker said last week.

Janet Woodcock, acting FDA commissioner (AP Images)

Janet Wood­cock slams calls for FDA 'fire­wal­l' amid ad­u­canum­ab de­ba­cle, cit­ing need for col­lab­o­ra­tive drug de­vel­op­ment

The FDA has long taken a collaborative approach to working with drugmakers on approval applications, all in the interest, it says, of bringing the best molecules to market. But when the drug is a lemon, the cozy relationship between regulators and industry courts easy blame.

Could a “firewall” between regulators and drugmakers prevent lousy drugs from getting the agency’s backing? Acting commissioner Janet Woodcock says no way.

Weeks af­ter drop­ping big re­sponse da­ta, Am­gen lines up pri­or­i­ty re­view for KRAS hope­ful so­tora­sib in NSCLC

Amgen is looking to go where no other drugmaker has gone before with the KRAS inhibitor sotorasib, which posted big Phase II lung cancer response data in late January. The FDA has been listening, and now it’s ready to give Amgen its ear.

The FDA will speedily review sotorasib’s application to treat second-line patients with locally advanced or metastatic non-small cell lung cancer in what would be the first successful bid to inhibit KRAS, Amgen said in a release.

No­var­tis' En­tresto, fac­ing a big come­back in hard-to-treat heart fail­ure pa­tients, scores FDA nod af­ter ad­comm cake­walk

Hard-and-fast rules in drug development are hard to come by, but one has long been the standard for market approvals — you have to hit your primary endpoints in pivotal studies. But for Novartis’ heart failure med Entresto, what’s a Phase III miss between friends?

The FDA on Tuesday approved Entresto, a combination of neprilysin inhibitor sacubitril and angiotensin receptor II blocker valsartan, to treat heart failure patients with a preserved ejection fraction (HFpEF), making it the first drug on the market for that population.

Sesen Bio, af­ter years on the edge of ir­rel­e­van­cy, scores speedy re­view for long ges­tat­ing blad­der can­cer pro­gram

After years spent in the penny stock range, Sesen Bio has nabbed a long-awaited FDA review for its antibody-drug conjugate bladder cancer program.

The FDA will give an accelerated audit to Sesen and its bladder cancer candidate Vicineum, an antibody-drug conjugate acquired in the company’s buyout of Toronto-based Viventia back in 2016. Sesen is penciled in for an Aug 18 PDUFA, and there is no adcomm on the docket right now, the company said.

Casey Lynch, Cortexyme CEO

Up­dat­ed: Cor­texyme's un­ortho­dox Alzheimer's ap­proach tripped up by FDA hold

The FDA has thrown a minor wrench in Cortexyme’s unorthodox plan to treat Alzheimer’s.

On Monday, the Bay Area biotech announced that the agency had put one of the studies in their pivotal program on hold after the company reported that patients experienced liver problems while taking their lead drug. The issues were reversible and had no known long-term effects, the company said.

The hold will not affect the Phase II/III, blinded, randomized controlled study they hope to read out towards the end of this year. Instead, the agency has decided to end, for the time being, an open-label extension study that had been measuring their molecule’s long-term effects on patients who already completed the initial 48-week study. No new patients will be enrolled and patients currently in the trial will be discontinued.

Astel­las, Seagen pave the way for Pad­cev's glob­al roll­out with full con­fir­ma­to­ry da­ta for urothe­lial can­cer in hand

Astellas and Seagen made a big splash with their Nectin-4 targeting therapy Padcev in late 2019, scoring a conditional FDA approval to target urothelial cancer. Now, the results are in for the confirmatory study for that indication, and it looks like a resounding win for Padcev.

Astellas and Seagen’s Padcev extended patients’ lives to an average 12.9 months compared with nine months for a chemo control, according to full data from the Phase III EV-301 study presented Friday at the virtual ASCO GU meeting.

FDA slaps Flori­da's Al­lay Phar­ma­ceu­ti­cals with warn­ing let­ter over vague re­spons­es to drug po­ten­cy is­sues

Inadequate responses to findings of potency discrepancies, blending issues and out-of-specification samples during a May 2020 inspection recently landed tablet manufacturer Allay Pharmaceuticals with a scorching FDA warning letter.

In a heavily-redacted version of the Jan 27 letter that was made available this week, the FDA chided the Hialeah, Florida manufacturer for vague and insufficient responses across the board to three major observations discovered during the May 5 to 15, 2020 site inspection. Those observations were communicated through a Form 483 letter, and Allay’s responses to the FDA on June 6 clearly irked the agency.

John Crowley (File photo)

UP­DAT­ED: Am­i­cus shares slammed as lead PhI­II drug fails a key head-to-head test for Pompe dis­ease — but the CEO still projects an FDA OK and bright fu­ture

Amicus stock got hammered Thursday afternoon after the biotech put out word that the crown jewel in the pipeline just failed the primary endpoint in a head-to-head study with the standard of care for Pompe disease.

CEO John Crowley, though, has a long track record at spotlighting the positive, which was on full display in its statement on the failed study. Researchers for the company highlighted some marginally positive secondary data with an improvement in respiratory function from a post hoc analysis that they plan to roll ahead to the FDA.

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