Glass half emp­ty: Achao­gen re­ceives split vote from FDA ex­perts on lead an­tibi­ot­ic, shares plunge

An FDA ad­vi­so­ry com­mit­tee has hand­ed down a split vote — yes for one in­di­ca­tion and no for an­oth­er — on Achao­gen’s lead an­tibi­ot­ic, ruf­fling the high ex­pec­ta­tions it set up go­ing in.

In­vestors weren’t hap­py, send­ing Achao­gen stocks down close to 29% in af­ter-hours trad­ing.

The split de­ci­sion car­ries in­ter­est­ing im­pli­ca­tions for oth­er an­tibi­ot­ic mak­ers. In a first, Achao­gen $AKAO sub­mit­ted an NDA with a re­quest to use the Lim­it­ed Pop­u­la­tion An­tibac­te­r­i­al Drug (LPAD) path­way in re­view­ing the drug, pla­zomicin, for the treat­ment of blood­stream in­fec­tions (BSI) due to Car­bapen­em-re­sis­tant En­ter­obac­te­ri­aceae. That hap­pens to be the in­di­ca­tion that the ad­comm vot­ed down; all 15 of them vot­ed yes when it came to treat­ing pa­tients with com­pli­cat­ed uri­nary tract in­fec­tions (cU­TI).

Pack­aged in­to the 2016 21th Cen­tu­ry Cures Act, the LPAD reg­u­la­to­ry path­way al­lows for ac­cep­tance of greater un­cer­tain­ty or high­er risk in pa­tients with se­ri­ous dis­eases (and an un­met need) pro­vid­ed that sub­stance ev­i­dence of ef­fec­tive­ness is pre­sent­ed.

A large part of what seems to have doomed Achao­gen’s plans for the BSI in­di­ca­tion was a small­er-than-ex­pect­ed sam­ple size that com­pli­cat­ed sta­tis­ti­cal analy­sis. Ear­ly in the meet­ing, an FDA clin­i­cal re­view­er point­ed out that the biotech had to amend pro­to­cols for a Phase III study (ACHN-490-007) be­cause they could on­ly en­roll 37 pa­tients out of the 286 planned. That re­sult­ed in sta­tis­ti­cal lim­i­ta­tions in con­clud­ing that pla­zomicin had su­pe­ri­or ef­fi­ca­cy com­pared to col­istin in the com­para­tor arm, even though nu­mer­i­cal trends point­ed to that con­clu­sion.

The re­view­er went on to sug­gest look­ing at the da­ta through the lens of non­in­fe­ri­or­i­ty, which the da­ta would al­low for, but cau­tioned that such as­sess­ments are com­pli­cat­ed by de­sign fea­tures the FDA agreed to back when Achao­gen was plan­ning a su­pe­ri­or­i­ty tri­al. Among those fea­tures: pri­or or con­comi­tant an­tibac­te­r­i­al ther­a­py, as well as lax­er cri­te­ria for pa­tients re­gard­ing bac­te­ria cul­ture in their bod­ies. That ad­di­tion­al un­cer­tain­ty re­gard­ing base­line sta­tus for pa­tients didn’t help, ei­ther.

Af­ter dis­cussing the sta­tis­ti­cal plan and clar­i­fy­ing how best to bal­ance the LPAD con­sid­er­a­tion with ben­e­fit-risk as­sess­ments, 11 com­mit­tee mem­bers vot­ed against pla­zomicin in BSI, most­ly con­cerned that the study was un­der­pow­ered to sup­port con­clu­sions of ef­fi­ca­cy. The oth­er four dis­agreed, cit­ing the lim­it­ed use and un­met need.

For cU­TI, on the oth­er hand, com­mit­tee mem­bers had no prob­lem unan­i­mous­ly en­dors­ing pla­zomicin based on a study that showed its was non­in­fe­ri­or to meropen­em, even con­sid­er­ing a larg­er mar­gin of 15% (ver­sus a con­ven­tion­al 10%) the FDA sanc­tioned.

Achao­gen CEO Blake Wise had this to say in a short state­ment:

We are en­cour­aged by the Com­mit­tee’s unan­i­mous vote in fa­vor of pla­zomicin for com­pli­cat­ed uri­nary tract in­fec­tions (cU­TI). The dis­cus­sion un­der­scored the re­al-world chal­lenges that health­care providers face every day giv­en lim­it­ed or in­ad­e­quate treat­ment op­tions for cer­tain pathogens. Re­gard­ing blood­stream in­fec­tions, the Lim­it­ed-Pop­u­la­tion An­tibac­te­r­i­al Drug path­way, or LPAD, is a nov­el ap­proach that en­ables the FDA to con­sid­er the ben­e­fits and risks for the sick­est pa­tients who have few or no avail­able treat­ment op­tions, and to ap­prove an­tibi­otics like pla­zomicin that we be­lieve, have the po­ten­tial to ad­dress these lim­it­ed pa­tient pop­u­la­tions.

South San Fran­cis­co-based Achao­gen is one of a hand­ful of biotechs in late-stage de­vel­op­ment with new an­tibi­otics in an era of ris­ing drug re­sis­tance to the main­stays in the field. Whether it will be­come a trail­blaz­er or a cau­tion­ary tale will be­come clear by its PDU­FA date of June 25.

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.

Phillip Gomez, SIGA CEO

UP­DAT­ED: On the back of SIGA Tech­nolo­gies' win with the FDA, the mon­key­pox virus sees the com­pa­ny spring­ing to fur­ther ac­tion

As the cases of monkeypox now sit at well over 100 worldwide and have spread to multiple continents, the orders for any type of vaccine against monkeypox are seeing nations and medical bodies looking to get their hands on anything and everything. And now SIGA Technologies seems to be getting in on the action.

According to Euronews, SIGA Technologies, a pharmaceutical company that is focused on providing medical countermeasures to biological and chemical attacks, is now in talks with several European authorities looking to stockpile its antiviral that can counter monkeypox. The drug known as tecovirimat or Tpoxx was approved by the FDA in 2018 as a vaccine for smallpox but was approved by the European Medicines Agency to also act against monkeypox, cowpox and complications from immunization with vaccinia.

Lutz Hegemann, Novartis president of global health

No­var­tis li­cens­es out leukemia drug as part of new glob­al coali­tion to in­crease ac­cess to can­cer treat­ments

The Union for International Cancer Control (UICC) has gathered a slate of Big Pharmas for its new collaboration in hopes of increasing access to cancer medicines in lower income countries, UICC announced yesterday.

Dubbed ATOM, or Access to Oncology Medicines, the coalition includes AstraZeneca, BeiGene, Novartis, Bristol Myers Squibb, Roche, Gilead, and Sanofi, among other organizations. The goal of the partnership is to increase generic and biosimilar development of cancer drugs as well as license out essential medicines to these countries. The third part of the partnership includes building up the infrastructure to diagnose cancers and properly handle cancer medicines.

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Bris­tol My­ers dusts off an old Cel­gene chemother­a­py for use in chil­dren with a rare blood can­cer

Bristol Myers Squibb is bringing new life to a chemotherapy drug from the old Celgene pipeline as it touts another approval in a rare form of blood cancer affecting young children.

The FDA on Friday approved Vidaza (chemically known as azacitidine) for pediatric patients 1 month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML). The new indication marks the first approval for Vidaza in more than a decade, though it’s commonly used to treat acute myeloid leukemia and myelodysplastic syndromes (MDS).