FDA Commissioner Robert Califf on Capitol Hill, Feb. 8, 2023 (Drew Angerer/Getty Images)

FDA com­mis­sion­er floats ideas on how to bet­ter han­dle the pan­dem­ic

FDA Com­mis­sion­er Rob Califf joined the heads of the CDC and NIH in the hot seat to­day be­fore a key House sub­com­mit­tee, ex­plain­ing that there needs to be a much faster, more co­or­di­nat­ed way to over­see vac­cine safe­ty, and that for­eign bio­phar­ma in­spec­tions, halt­ed for years due to the pan­dem­ic, are slow­ly ramp­ing up again.

Califf, who stressed to the House En­er­gy and Com­merce’s Sub­com­mit­tee on Health that the CDC al­so needs bet­ter da­ta, made clear that the FDA’s abil­i­ty to mon­i­tor the safe­ty of vac­cines “would al­so ben­e­fit great­ly by a co­or­di­nat­ed fed­er­al pub­lic health da­ta re­port­ing au­thor­i­ty.”

FDA cur­rent­ly us­es its Bi­o­log­ics Ef­fec­tive­ness and Safe­ty (BEST) Ini­tia­tive, to an­a­lyze health in­sur­ance claims or elec­tron­ic health records (EHRs) from large da­ta sys­tems. But Califf made clear there are still lim­its to these us­es.

“While the BEST Ini­tia­tive has been es­sen­tial for our work and pro­vid­ed us with a ro­bust pic­ture of safe­ty da­ta, our abil­i­ty to an­a­lyze claims in­for­ma­tion is lim­it­ed by the fact that some vac­ci­na­tions are not record­ed in health in­sur­ance claims da­ta,” Califf’s writ­ten tes­ti­mo­ny says, adding:

Fur­ther, when in­sur­ance claims data­bas­es or EHRs de­tect an ad­verse event, FDA of­ten needs to quick­ly ver­i­fy in­for­ma­tion or ac­cess ad­di­tion­al in­for­ma­tion to eval­u­ate the ad­verse events of in­ter­est. When we re­quest records to ver­i­fy ad­verse events de­tect­ed by the BEST Ini­tia­tive data­bas­es it has tak­en FDA around 8-12 weeks in some cas­es to re­ceive vol­un­tary ac­cess to these records. Ad­di­tion­al­ly, co­or­di­nat­ed fed­er­al pub­lic health da­ta re­port­ing au­thor­i­ty would help the Agency to more swift­ly iden­ti­fy ad­verse event pat­terns and trends as­so­ci­at­ed with the use of vac­cines or oth­er MCMs, and swift­ly be able to com­mu­ni­cate with health care providers and pa­tients about safe­ty sig­nals.

Com­mit­tee Chair Cathy Mc­Mor­ris Rodgers (R-WA) ques­tioned Califf on why there aren’t more FDA staffers com­ing in­to the agency’s cam­pus in White Oak, MD, and Califf de­fend­ed his team, and in his writ­ten tes­ti­mo­ny, adding:

Our staff have had to be pulled off oth­er work and have been work­ing re­lent­less­ly on pan­dem­ic is­sues for the past three years, lead­ing to a sig­nif­i­cant back­log and fa­tigue.

But he al­so called for the cre­ation of a spe­cial­ized pro­gram with­in CBER to de­fend against emerg­ing pathogens to bet­ter po­si­tion FDA to deal with these kinds of threats.

“In con­sul­ta­tion with HHS part­ners, the pro­gram could: pro­vide rec­om­men­da­tions and guid­ance to de­vel­op­ers of vac­cines and oth­er med­ical prod­ucts and rel­e­vant fed­er­al part­ners; use re­al-world da­ta or re­al-world ev­i­dence to study the safe­ty and ef­fec­tive­ness of prod­ucts for ad­dress­ing bi­o­log­i­cal in­ci­dents and iden­ti­fy which prod­ucts may be best suit­ed for spe­cif­ic pathogens or for use in dif­fer­ent pop­u­la­tions; and fa­cil­i­tate prod­uct de­vel­op­ment in­clud­ing ad­vances in man­u­fac­tur­ing,” Califf wrote.

Rep. Gus Bili­rakis (R-FL) ques­tioned Califf on FDA’s for­eign in­spec­tions, which have rapid­ly de­clined dur­ing the pan­dem­ic. Be­tween April and Sep­tem­ber 2021, the FDA said it com­plet­ed just 37 for­eign drug in­spec­tions, which com­pares with more than 1,200 in 2019.

Califf ac­knowl­edged there’s more work to do on sup­ply chains, but it should be an in­ter­ac­tion be­tween FDA and in­dus­try. He not­ed that FDA now has ad­di­tion­al hir­ing au­thor­i­ty to ramp up the num­ber of in­spec­tors,”and we’re catch­ing up, par­tic­u­lar­ly in Chi­na due to lack of ac­cess un­til very re­cent­ly. We’ll have a lot to dis­cuss on how to make this bet­ter. It’s frag­ile,” Califf said.

As far as thoughts on the man­u­fac­tur­ing boom in Chi­na and In­dia over the last decade or so, Califf said, “We’ve got to be there … and I hope we can fix that and bring it back to the US.”

In his writ­ten tes­ti­mo­ny, Califf called on Con­gress to do more to align fed­er­al and state of­fi­cials on the sup­ply chain too.

“Al­low­ing for dis­clo­sure of non-pub­lic in­for­ma­tion to these agen­cies with com­ple­men­tary func­tions re­lat­ed to FDA-reg­u­lat­ed prod­ucts could achieve faster and more ef­fec­tive ac­tion to pro­tect the pub­lic health dur­ing na­tion­al pub­lic health emer­gen­cies, oth­er state/lo­cal dis­as­ter de­c­la­ra­tions, out­breaks or oth­er pub­lic health events, and for rou­tine reg­u­la­to­ry over­sight,” he wrote.

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Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Ivana Magovčević-Liebisch, Vigil Neuroscience CEO

FDA lifts par­tial clin­i­cal hold on Vig­il Neu­ro­science's TREM2 an­ti­body, re­mov­ing dos­ing cap

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

Man­u­fac­tur­ing roundup: Catal­ent to pro­duce low-cost ver­sion of nalox­one; CSL opens R&D site

Catalent will be manufacturing a low-cost version of the opioid overdose treatment naloxone as part of a contract with Harm Reduction Therapeutics.

Catalent plans to manufacture the treatment at its facility in Morrisville, NC. No financial details on the deal were disclosed.

Harm Reduction was granted priority review status for the NDA on its spray last year. The company has been working on a naloxone product since 2017. It is anticipating approval in July of this year and a US launch in early 2024.

As­pen looks to re­bound in pro­duc­tion and rev­enue af­ter Covid-19

Last year, South African-based vaccine manufacturer Aspen Pharmacare was facing reports that it had not received a single order for its manufactured Covid-19 shots and that manufacturing lines were sitting idle. But now the vaccine producer is looking to turn things around.

Aspen’s disclosure of its financial results in March unveiled that manufacturing revenue had decreased by 12% to R 603 million ($33.8 million), which Lorraine Hill, Aspen Group’s COO, said is attributable to lower Covid vaccine sales.

Ribbon cutting ceremony for Thermo Fisher's new cell therapy manufacturing site in San Francisco

Ther­mo Fish­er moves on cam­pus with new cell man­u­fac­tur­ing site in San Fran­cis­co

Thermo Fisher Scientific is putting down more roots in the Bay Area.

The manufacturer opened the doors to a new cell therapy manufacturing facility next to the University of California-San Francisco Medical Center’s Mission Bay campus and on the university’s campus.

UCSF and Thermo Fisher have had a partnership since 2021, with the new site focusing on manufacturing cell therapeutics for certain cancers, including glioblastoma and multiple myeloma. The new site plans to use Thermo Fisher’s expertise in manufacturing services to help UCSF accelerate the development of cell therapies and eventually get them into the clinic, said Dan Herring, the general manager of cell therapy services at Thermo Fisher, in an interview with Endpoints News.

Sulagna Bhattacharya, Nanoscope Therapeutics CEO

Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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FDA grants full ap­proval to Keytru­da in tu­mor-ag­nos­tic set­ting; Can­del paus­es tri­al en­roll­ment

In a first, Merck has secured a full approval for Keytruda in a tumor agnostic setting — as a treatment for any unresectable or metastatic solid tumors that are classified as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR).

The FDA granted Keytruda accelerated approval in this indication in 2017, and GSK’s Jemperli followed suit in 2021. But now it’s converted to a full approval for Keytruda. Before prescribing, doctors would have to make sure patients carry this biomarker, using an FDA-approved test.