HHS reg­u­la­to­ry agen­da for 2021 is light on new pro­posed and fi­nal FDA rules

The im­pact of the coro­n­avirus on the FDA’s work plans is ev­i­dent in the lat­est se­mi-an­nu­al reg­u­la­to­ry agen­da for HHS, which in­cludes on­ly two new bio­phar­ma-re­lat­ed pro­posed rules and one fi­nal rule.

The two pro­posed rules cen­ter on one that seeks to change FDA med­ica­tion guide reg­u­la­tions and an­oth­er to clar­i­fy FDA’s study con­duct ex­pec­ta­tions for an­a­lyt­i­cal and clin­i­cal phar­ma­col­o­gy, bioavail­abil­i­ty (BA) and bioe­quiv­a­lence (BE) stud­ies.

In the case of up­dat­ing FDA med­ica­tion guide reg­u­la­tions, the agency is seek­ing to re­quire drug spon­sors of out­pa­tient drugs to sub­mit a new form of pa­tient la­bel­ing to be re­viewed by the FDA.

“The pro­posed rule would in­clude re­quire­ments for Pa­tient Med­ica­tion In­for­ma­tion de­vel­op­ment and dis­tri­b­u­tion,” HHS says. “The pro­posed rule would re­quire clear and con­cise­ly writ­ten pre­scrip­tion drug prod­uct in­for­ma­tion pre­sent­ed in a con­sis­tent and eas­i­ly un­der­stood for­mat to help pa­tients use their pre­scrip­tion drug prod­ucts safe­ly and ef­fec­tive­ly.”

For the oth­er pro­posed rule that HHS ex­pects to un­der­take in the fore­see­able fu­ture, FDA is propos­ing to amend 21 CFR 320, in cer­tain parts, and es­tab­lish a new 21 CFR 321 to clar­i­fy FDA’s study con­duct ex­pec­ta­tions for an­a­lyt­i­cal and clin­i­cal phar­ma­col­o­gy, BA and BE stud­ies that sup­port hu­man re­search and mar­ket­ing ap­pli­ca­tions.

“The pro­posed rule would spec­i­fy need­ed ba­sic study con­duct re­quire­ments to en­able FDA to en­sure those stud­ies are con­duct­ed ap­pro­pri­ate­ly and to ver­i­fy the re­li­a­bil­i­ty of study da­ta from those stud­ies,” HHS said. “This reg­u­la­tion would align with FDA’s oth­er good prac­tice reg­u­la­tions, would al­so be con­sis­tent with cur­rent in­dus­try best prac­tices, and would har­mo­nize the reg­u­la­tions more close­ly with re­lat­ed in­ter­na­tion­al reg­u­la­to­ry ex­pec­ta­tions.”

Oth­er pre­vi­ous­ly pro­posed FDA rule­mak­ings, such as re­vi­sions around when the FDA can refuse a gener­ic drug ap­pli­ca­tion or the mod­ern­iza­tion of FDA’s bi­o­log­ics reg­u­la­tions, were not in­clud­ed in the lat­est HHS agen­da.

The one fi­nal FDA rule on the HHS’ agen­da this year deals with a pro­posed rule from 2019 that will amend the agency’s 503A Bulks List, which is a list of bulk drug sub­stances that can be used in com­pound­ing.

The fi­nal rule will add five ad­di­tion­al bulk drug sub­stances to the list, in­clud­ing Glu­taralde­hyde, Gly­col­ic acid, L-cit­rulline, Pyru­vic acid and Trichloroacetic acid. The rule will al­so iden­ti­fy 26 bulk drug sub­stances that FDA has de­cid­ed not to in­clude on the 503A Bulks List. Ad­di­tion­al sub­stances nom­i­nat­ed by the pub­lic for in­clu­sion on this list are cur­rent­ly un­der con­sid­er­a­tion and will be the sub­ject of a fu­ture rule­mak­ing, HHS said.

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.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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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.

Like the flu vac­cine every year, the FDA could move quick­ly on a vari­ant-tar­get­ed Covid vac­cine

In the same way that the FDA signs off on flu vaccines every year without requiring large clinical trials to measure their efficacy, the FDA may employ a similar strategy in authorizing variant-focused versions of the mRNA vaccines.

As the world braces for more data on the latest variant Omicron, which may reduce vaccine efficacy, top vaccine developers like Moderna and Pfizer-BioNTech have promised they can pull together a new vaccine targeted against a specific Covid variant in about 100 days. Since Omicron emerged last week, Pfizer-BioNTech, Moderna and J&J have all said they’ve begun work on Omicron-specific vaccines, if needed.

More man­u­fac­tur­ing is­sues: Fen­nec preps for sec­ond CRL for po­ten­tial hear­ing loss drug

Shares of Fennec Pharmaceuticals stock were cut almost in half early Monday as the company said manufacturing issues caused another FDA rejection of its reformulated version of sodium thiosulfate, which is intended to help kids who lose hearing due to chemo treatment.

The biotech had resubmitted an NDA for the drug to treat platinum-based, chemo-related ototoxicity in young children earlier this year. The first NDA was denied by the FDA last year, with the agency citing manufacturing issues with the biotech’s supplier.

Giovanni Caforio, Bristol Myers Squibb CEO (Pablo Martinez Monsivais/AP Images)

Amid JAK de­ba­cle, Bris­tol My­ers set­tles in for FDA's long re­view of po­ten­tial first TYK2 drug

Bristol Myers Squibb has $4 billion hopes for its late-stage TYK2 inhibitor deucravacitinib, but the FDA’s recent negative review for the JAK inhibitor class has dampened hopes somewhat. Now, the agency will get its first good look at TYK2, and Bristol Myers will have to wait and hold its breath.

The FDA has set a target review date of Sept. 10, 2022, for deucravacitinib, a potential first-in-class oral inhibitor for the TYK2 signaling pathway in psoriasis that would be a challenger to the controversial JAK inhibitors, the drugmaker said Monday.

Shankar Musunuri, Ocugen CEO

Ocu­gen hits an­oth­er snag in bring­ing its Bharat Biotech-part­nered Covid-19 vac­cine to the US

Back in July, Ocugen and Bharat Biotech unveiled some Phase III data suggesting their Covid-19 vaccine, Covaxin, was 77.8% effective at preventing overall disease in India. They’re now looking to launch an immuno-bridging study to see if those numbers hold up in the US — but on Friday, the FDA said not so fast.

Regulators have placed a clinical hold on the partners’ IND, Ocugen shared on Friday. Ocugen’s stock $OCGN sank 9% on the news Friday, but was up almost 7% in pre-market trading on Monday.

Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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