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. 

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

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