UP­DAT­ED: J&J nabs la­bel ex­pan­sion for Ste­lara in pe­di­atric pso­ri­at­ic arthri­tis

Near­ly a decade af­ter J&J’s Ste­lara was ap­proved to treat adults with pso­ri­at­ic arthri­tis, the phar­ma gi­ant is ex­pand­ing the la­bel to in­clude the small per­cent­age of chil­dren who suf­fer from the au­toim­mune dis­ease.

Ter­ence Rooney

Reg­u­la­tors gave Ste­lara the green light on Mon­day for chil­dren ages six and old­er with ac­tive pso­ri­at­ic arthri­tis (PsA). The in­jectable will be ad­min­is­tered in two starter dos­es, fol­lowed by four shots per year.

“We know ac­tive pe­di­atric pso­ri­at­ic arthri­tis is a chal­leng­ing in­flam­ma­to­ry dis­ease giv­en its rar­i­ty and that symp­toms, such as swollen joints and skin le­sions, can vary sig­nif­i­cant­ly in pre­sen­ta­tion and sever­i­ty,” Ter­ence Rooney, VP of Janssen R&D’s rheuma­tol­ogy and ma­ter­nal fe­tal dis­ease area, said in a state­ment.

Be­cause of a lim­it­ed avail­abil­i­ty to pe­di­atric PsA pa­tients, reg­u­la­tors based their ap­proval on safe­ty and ef­fi­ca­cy da­ta ex­trap­o­lat­ed from mul­ti­ple Phase III stud­ies in adult and pe­di­atric pa­tients with plaque pso­ri­a­sis and PsA, ac­cord­ing to Janssen. The com­pa­ny said in a news re­lease:

An analy­sis of the da­ta demon­strat­ed that PK ex­po­sure of STE­LARA in these pe­di­atric PsO pa­tients with ac­tive PsA was con­sis­tent with that of Phase 3 clin­i­cal tri­als of STE­LARA in pe­di­atric PsO pa­tients with­out ac­tive PsA, as well as with adult pa­tients with mod­er­ate to se­vere plaque PsO or adult pa­tients with ac­tive PsA, while da­ta on com­mon ef­fi­ca­cy end­points were sim­i­lar in these pe­di­atric PsO pa­tients with ac­tive PsA.

Janssen ini­tial­ly ap­plied to ex­pand the la­bel to chil­dren five years and old­er back in Oc­to­ber. The com­pa­ny had sub­mit­ted ex­trap­o­lat­ed da­ta from near­ly 4,000 pa­tients, it said at the time.

“These stud­ies in­clud­ed pa­tients ages 6 and up. Ini­tial­ly, we were hope­ful to ex­tend this la­bel to pa­tients one year younger than the study pop­u­la­tion, how­ev­er to­geth­er with the FDA, we ul­ti­mate­ly de­cid­ed to in­clude ages 6 and up, in keep­ing with the low­er age lim­it of the pe­di­atric pop­u­la­tion in CAD­MUS Jr. study,” Jen­nifer David­son, VP of im­munol­o­gy med­ical af­fairs in Janssen’s sci­en­tif­ic af­fairs unit, told End­points News in an email.

Ste­lara tar­gets in­ter­leukin (IL)-12 and IL-23 cy­tokines, which are known to play a key role in in­flam­ma­to­ry and im­mune re­spons­es. The drug was first ap­proved for adults with pso­ri­a­sis back in 2009, and has picked up sev­er­al in­di­ca­tions since in­clud­ing ul­cer­a­tive col­i­tis and Crohn’s dis­ease. It was first ap­proved to treat pe­di­atric plaque pso­ri­a­sis pa­tients aged six years and old­er back in 2020.

Pe­di­atric PsA oc­curs in on­ly 5% to 8% of chil­dren and ado­les­cents with chron­ic in­flam­ma­to­ry arthri­tis, ac­cord­ing to Janssen. Symp­toms re­sem­ble adult PsA, in­clud­ing joint in­flam­ma­tion and skin le­sions.

A re­cent analy­sis from Spher­ix Glob­al In­sights re­vealed that der­ma­tol­o­gists pre­fer IL-23s to treat pa­tients with pso­ri­a­sis and pso­ri­at­ic arthri­tis, as op­posed to IL-17s or oth­er cat­e­gories like JAK in­hibitors that have faced in­creased scruti­ny from the FDA. Mean­while, rheuma­tol­o­gists said they “heav­i­ly” pre­fer IL-17s.

When asked which IL-23 drug they fa­vor, der­ma­tol­o­gists over­whelm­ing­ly chose Ab­b­Vie’s Skyrizi, which is cur­rent­ly ap­proved to treat on­ly adults.

“As a glob­al leader in im­munol­o­gy, Janssen is ded­i­cat­ed to re­duc­ing the bur­den of chron­ic au­toim­mune dis­eases, and this ad­di­tion­al ap­proval for STE­LARA builds on our lega­cy of bring­ing im­por­tant treat­ment op­tions to younger pa­tients,” David­son said in a news re­lease.

Ste­lara raked in $2.6 bil­lion last quar­ter, ac­cord­ing to J&J’s half-year re­port. The drug made Eval­u­ate Van­tage’s list of top 10 ex­pect­ed block­busters this year, rank­ing sev­enth place at an an­tic­i­pat­ed $10.1 bil­lion.

This sto­ry has been up­dat­ed with a com­ment from Janssen. 

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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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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:

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.

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.

FDA's vac­cine ad­comm to re­view first fe­cal trans­plant to treat C. dif­fi­cile in­fec­tions

Back in 2018, Swiss drugmaker Ferring Pharmaceuticals made a big bet on Minnesota-based Rebiotix, buying up the company for its experimental poop-based drug implant to treat an infection caused by C. difficile, a potentially dangerous bacteria, in a new way.

Four years later, Ferring’s fecal microbiota transplant, dubbed RBX2660 or Rebyota, will face the FDA’s adcomm of outside vaccine experts on Sept. 22, debating whether the agency should license the transplant as a treatment for adults following antibiotic treatment for recurrent C. difficile infection.