FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednes­day, in part due to pan­dem­ic-re­lat­ed trav­el re­stric­tions.

North Car­oli­na-based Liq­uidia Tech­nolo­gies was hand­ed a CRL for its lead pul­monary ar­te­r­i­al hy­per­ten­sion drug, cit­ing the need for more CMC da­ta and on-site pre-ap­proval in­spec­tions, which the FDA hasn’t been able to con­duct due to trav­el re­stric­tions. The agency al­so de­ferred its de­ci­sion on Re­vance Ther­a­peu­tics’ BLA for its frown line treat­ment, be­cause it needs to in­spect the com­pa­ny’s north­ern Cal­i­for­nia man­u­fac­tur­ing fa­cil­i­ty. The ac­tion, Re­vance em­pha­sized, was not a CRL.

The FDA said it’s “ac­tive­ly work­ing to de­fine an ap­proach for out­stand­ing in­spec­tions,” ac­cord­ing to Re­vance.

Neal Fowler

“With the pan­dem­ic, as you know, they’ve been very chal­lenged to do these,” Liq­uidia CMO Tushar Shah said dur­ing a call with in­vestors on Wednes­day morn­ing. “We an­tic­i­pate a lot of com­pa­nies are strug­gling, and in the best case sce­nario, we would have an­tic­i­pat­ed some­time mid-next year would have been the ear­li­est they would prob­a­bly get to in­spec­tion,” he con­tin­ued.

De­spite the set­back, Liq­uidia is still eye­ing a 2022 launch for LIQ861 — if it gets ap­proval. The tre­pros­tinil in­hala­tion pow­der was the fo­cal point of Liq­uidia’s 2018 IPO fil­ing, which priced at $11 per share, the mid­point of a $10 to $12 range. The biotech raked in $50 mil­lion, $30 to $32 mil­lion of which was tagged for LIQ861.

On Wednes­day, Liq­uidia’s $LQ­DA stock was down 6.13%,with shares pric­ing at $2.91 apiece. The biotech says the ad­di­tion­al CMC da­ta sought by the FDA per­tain to the “drug prod­uct and de­vice bio­com­pat­i­bil­i­ty,” and that the agency didn’t ask for fur­ther clin­i­cal stud­ies, or stud­ies re­lat­ed to tox­i­col­o­gy or clin­i­cal phar­ma­col­o­gy.

Liq­uidia has a tech­nol­o­gy col­lab­o­ra­tion go­ing with Glax­o­SmithK­line, which it ex­pand­ed to in­clude three ad­di­tion­al pro­grams last year. The biotech is on a mis­sion to un­seat Unit­ed Ther­a­peu­tics’ Ty­va­so and its neb­u­liz­er, which net­ted $415.6 mil­lion last year. John­son & John­son al­so en­tered the PAH space when it bought Acte­lion for $30 bil­lion back in 2017.

If Re­vance’s in­jectable Dax­i­bot­u­linum­tox­i­nA gets ap­proved, it’ll go head-to-head with Al­ler­gan’s Botox. The drug was test­ed against mod­er­ate to se­vere glabel­lar lines, more com­mon­ly known as frown lines. It met its pri­ma­ry and sec­ondary end­points in two Phase III tri­als and was shown to last for six months, ac­cord­ing to Al­ler­gan, while Botox gen­er­al­ly lasts for three to four.

Mark Fo­ley

Al­ler­gan com­ment­ed back in 2017, though, that it doesn’t be­lieve Re­vance’s da­ta “will sup­port a longer du­ra­tion claim as the on­ly com­pos­ite da­ta (2-point im­prove­ment, none or mild, and both in­ves­ti­ga­tor and pa­tient) is at 30 days not at 6 months.”

The FDA ac­cept­ed Re­vance’s BLA back in Q1. Its stock $RVNC was up 4.02% on Wednes­day, sell­ing at $24.35 a share. News of the FDA’s de­fer­ral comes just over a year af­ter co-founder Dan Browne stepped down as CEO “due to mis­judg­ment in han­dling an em­ploy­ee mat­ter,” leav­ing board mem­ber Mark Fo­ley, for­mer chief of Zel­tiq Aes­thet­ics, to fill his shoes.

“We look for­ward to con­tin­ued in­ter­ac­tion with the Agency and re­main ready to sup­port FDA’s pre-ap­proval in­spec­tion as soon as pos­si­ble. We are for­tu­nate that we man­u­fac­ture our prod­uct at a sin­gle lo­ca­tion in the U.S., which should put us at an ad­van­tage com­pared to in­ter­na­tion­al man­u­fac­tur­ing lo­ca­tions once trav­el re­sumes,” Fo­ley said in a state­ment.

He lat­er added that he be­lieves Re­vance “is in an ex­cel­lent po­si­tion, both com­mer­cial­ly and fi­nan­cial­ly, to weath­er a change to the tim­ing of this po­ten­tial ap­proval.”

A pre­vi­ous ver­sion of this ar­ti­cle in­cor­rect­ly at­trib­uted a quote by Liq­uidia CMO Tushar Shah to CEO Neal Fowler. 

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,900+ biopharma pros reading Endpoints daily — and it's free.

FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.