FDA re­jects Nabri­va a sec­ond time; Nabri­va pins it on trav­el re­stric­tions

A lit­tle over a year af­ter the FDA is­sued Nabri­va its first CRL for the an­tibi­ot­ic Con­tepo, the agency has is­sued an­oth­er.

By Nabri­va’s ac­count, the agency’s rea­son­ing hasn’t changed. They again cit­ed un­re­solved man­u­fac­tur­ing is­sues at the com­pa­ny’s CMO fa­cil­i­ties in Eu­rope and asked for no new clin­i­cal da­ta. The prob­lem, Nabri­va said, was that be­cause of trav­el re­stric­tions, the agency wasn’t able to do an on­site in­spec­tion of the fa­cil­i­ties to con­firm that the is­sues raised last year had been fixed.

Be­cause nei­ther com­pa­nies nor the FDA re­lease CRLs, it’s im­pos­si­ble to con­firm the claim, but it wouldn’t be the first time trav­el re­stric­tions have ap­peared to in­ter­fere with the re­view process. The agency warned in May that re­view times could be de­layed. And ear­li­er this month, No­var­tis an­nounced that the de­ci­sion date on its MS drug Arz­er­ra had been pushed from June to Sep­tem­ber. Though the com­pa­ny didn’t spec­i­fy rea­sons for the de­lay, one in­vestor at their Q1 call not­ed pos­si­ble out­stand­ing man­u­fac­tur­ing is­sues in Eu­rope that Covid-19 could have made dif­fi­cult to re­solve.

The re­jec­tion is a blow to a com­pa­ny that, like oth­er an­tibi­ot­ic mak­ers, has al­ready been ail­ing for some time. Al­though the Irish biotech reached $12 a share in 2017 off of pos­i­tive Phase III re­sults for the lead an­tibi­ot­ic and al­though that drug would soon be ap­proved, their shares tum­bled as oth­er suc­cess­ful an­tibi­ot­ic mak­ers filed for bank­rupt­cy and in­vestors be­gan to re­al­ize the grim fi­nan­cial log­ic of an­tibi­otics: To pre­vent re­sis­tance, you give new drugs on­ly as a last re­sort; a new drug, how­ev­er promis­ing, can be­come fa­tal­ly low-vol­ume.

The lat­est re­jec­tion cut shares by 15% pre-mar­ket, but it had al­ready en­tered pen­ny-stock ter­ri­to­ry.

The drug, known chem­i­cal­ly as fos­fomycin, has been avail­able in many coun­tries out­side the US since the ear­ly 1970s. Though of­ten giv­en oral­ly, Nabri­va had sub­mit­ted an in­tra­venous for­mu­la­tion for ap­proval in com­pli­cat­ed uri­nary tract in­fec­tions. The com­pa­ny ac­quired it from Za­vante in 2018 for 8.2 mil­lion shares — at the time worth around $25 mil­lion — and $100 mil­lion in mile­stones.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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Richard Pazdur (via AACR)

Ac­cel­er­at­ed ap­proval re­forms need mean­ing­ful con­fir­ma­to­ry tri­al im­prove­ments, pro­fes­sors write in Sci­ence

Outside of Covid-19, 2021 has been the year of the accelerated approval.

Beginning last spring, FDA openly challenged six “dangling” accelerated approvals (hadn’t confirmed their clinical benefit yet), three of which were later pulled by the companies.

Then in June, FDA pulled out the accelerated approval pathway, seemingly out of nowhere, to sign off on Biogen’s controversial Alzheimer’s drug Aduhelm. It hadn’t even been mentioned at the drug’s adcomm.

Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Lan Huang, BeyondSpring CEO

Months af­ter shock­ing in­vestors with lung can­cer win, Be­yond­Spring's lead drug hits road­block at the FDA

BeyondSpring shocked investors in early August after its once-marginal lead drug suddenly showed a lot of promise in a common form of lung cancer. With hopes high, the FDA has now slammed the door on that drug in another indication — does that spell bad news for BeyondSpring’s Cinderella story?

The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

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