Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The sto­ry of Am­ryt Phar­ma’s can­di­date for the ge­net­ic skin con­di­tion epi­der­mol­y­sis bul­losa, or EB, will soon en­ter an­oth­er chap­ter.

Af­ter the Irish drug­mak­er’s can­di­date, dubbed Oleogel-S10 and mar­ket­ed as Fil­su­vez, was hand­ed a CRL ear­li­er this year, the com­pa­ny an­nounced in a press re­lease that it plans to sub­mit a for­mal dis­pute res­o­lu­tion re­quest for the com­pa­ny’s NDA for Oleogel-S10.

Ac­cord­ing to the com­pa­ny, it re­cent­ly had a meet­ing with the FDA to dis­cuss the is­sues raised in the Feb­ru­ary CRL. The biotech ini­tial­ly re­port­ed that the FDA de­mand­ed “ad­di­tion­al con­fir­ma­to­ry ev­i­dence of ef­fec­tive­ness for Oleogel-S10 in EB.”

Fol­low­ing this meet­ing, Am­ryt said it plans to con­tin­ue down the for­mal dis­pute res­o­lu­tion path through CDER by which NDA ap­pli­cants can seek to re­solve sci­en­tif­ic or med­ical dis­putes “that can­not be re­solved at the di­vi­sion lev­el.”

“The For­mal Dis­pute Res­o­lu­tion process pro­vides an op­por­tu­ni­ty to raise our sci­en­tif­ic dis­agree­ment with­in CDER. We be­lieve this rep­re­sents the right ap­proach to­ward ap­proval in the US for Oleogel-S10, as we work to­ward bring­ing this ther­a­py to EB suf­fer­ers in des­per­ate need,” said Am­ryt CEO Joe Wi­ley in a state­ment.

The news that Am­ryt is push­ing back on the FDA comes on the heels of the Eu­ro­pean Com­mis­sion ap­prov­ing the drug. The EMA said the ben­e­fit of Fil­su­vez is its abil­i­ty to pro­mote the heal­ing of EB par­tial thick­ness wounds.

The news from the EMA and the con­tin­ued ac­tions against the FDA has been mu­sic to in­vestors’ ears. The com­pa­ny’s stock price $AMYT has risen 7% in the past five days but is still down 37% since the be­gin­ning of 2022.

The drug it­self has gone through an event­ful ride. Orig­i­nal­ly pick­ing up the drug from the small Ger­man drug­mak­er Birken, the can­di­date be­gan show­ing progress. In 2020, Phase III da­ta showed that across 223 pa­tients, those who re­ceived the drug were more like­ly to see their wounds close with­in 45 days than those on place­bo.

In 2021, the FDA grant­ed pri­or­i­ty re­view to the drug, ac­cel­er­at­ing the drug’s as­sess­ment time from 10 months to six months un­til run­ning in­to the CRL this year.

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.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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BREAK­ING: Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck 5 years ago, where the soft spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, less than two weeks after its supplemental BLA was accepted. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

No­vavax shares shred­ded as Covid vac­cine sales fall more than 90% in Q2

Months after Novavax celebrated its first profitable quarter as a commercial company, the Gaithersburg, MD-based company is back in the red.

Sales for Novavax’s Covid-19 vaccine slipped to $55 million last quarter, down from $586 million in Q1, CEO Stanley Erck revealed on Monday after market close. The company’s stock $NVAX plummeted more than 32% in after-hours trading.

Upon kicking off the call with analysts and investors, Erck addressed the elephant in the room:

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Sen­ate Dems cling to a sim­ple ma­jor­i­ty to pass some of the biggest drug pric­ing re­forms ever

The Pharmaceutical Research and Manufacturers of America — and their fleet of drug industry lobbyists on Capitol Hill — are known for never losing.

Whenever a big drug pricing bill comes up, an army of the industry group’s lobbyists descend onto the Hill and either smash it outright or dismantle it piece by piece.

But for perhaps the largest drug pricing reforms ever enacted, after more than a decade of Congress trying and failing to allow Medicare to negotiate prescription drug prices, those same lobbyists and their biopharma clients were dealt a stunning blow on Sunday afternoon.

Anna Protopapas, Mersana CEO

In $1.36B biobuck deal with GSK, Mer­sana touts 'biggest pre­clin­i­cal ADC deal ever'

Days after Enhertu reeled in another FDA nod, with the first-ever green light for HER2-low breast cancer, another antibody drug conjugate biotech claims it has secured the largest preclinical ADC pact to date for a single asset.

AstraZeneca and Daiichi Sankyo made waves with their nearly $7 billion collaboration back in spring 2019, but at that point, Enhertu was already nearing the FDA’s doors with clinical data. The latest ADC tie-up to enter the biopharma fray centers around a preclinical asset, Mersana Therapeutics’ XMT-2056.