Akcea, Io­n­is win over most FDA ex­perts to their rare dis­ease drug volane­sors­en, vast­ly im­prov­ing odds of suc­cess

Akcea Ther­a­peu­tics $AK­CA faced a tough up­hill bat­tle in search of an ap­proval for their rare dis­ease drug volane­sors­en to­day — and they won.

The FDA ad­vi­so­ry com­mit­tee vote on the drug — spun out from Io­n­is $IONS — was 12 for, 8 against. The FDA, of course, doesn’t have to go along. But for a rare dis­ease, they usu­al­ly do — par­tic­u­lar­ly un­der this ad­min­is­tra­tion.

Akcea’s shares shot up 20% in pre-mar­ket trad­ing on Fri­day, start­ing the day with a $1.8 bil­lion mar­ket cap, while Io­n­is saw a spike of 5%.

FDA reg­u­la­tors didn’t make the task very easy, high­light­ing their deep con­cerns with the sud­den plunge in platelets that af­flict­ed a num­ber of pa­tients through the clin­i­cal tri­als for the drug. In­ves­ti­ga­tors nev­er found a re­li­able way to screen for throm­bo­cy­tope­nia in the tri­al work, and their in­ter­nal re­view plant­ed a red flag on the fact that doc­tors pre­scrib­ing this drug would al­so like­ly be blind­sided by un­ex­pect­ed cas­es.

Io­n­is re­port­ed a lit­tle more that a year ago that it had achieved its ef­fi­ca­cy end­point in Phase III. But five pa­tients were forced out of the tri­al due to a threat­en­ing de­cline in platelet counts. Grade 4 throm­bo­cy­tope­nia oc­curred in three pa­tients, which end­ed af­ter they stopped dos­ing. There were no with­drawals due to platelet counts af­ter the com­pa­ny be­gan mon­i­tor­ing the side ef­fect.

If it does get an OK, those facts on safe­ty are like­ly to haunt the com­pa­ny’s sales ef­forts.

In its fa­vor, though, was a sol­id set of da­ta demon­strat­ing the drug’s ef­fect in reg­u­lat­ing plas­ma triglyc­eride for pa­tients with rare cas­es of fa­mil­ial chy­lomi­crone­mia syn­drome — but with­out the clear clin­i­cal im­pact that would have helped push this drug over the top with big­ger num­bers in its fa­vor.

“We thank all the mem­bers of the Com­mit­tee for their time in con­duct­ing a thor­ough and thought­ful re­view of Waylivra, a new po­ten­tial first and on­ly ther­a­py for peo­ple with fa­mil­ial chy­lomi­crone­mia syn­drome, or FCS. FCS is ul­tra-rare, se­vere and po­ten­tial­ly fa­tal with no ther­a­peu­tic op­tions. The da­ta, in­clud­ing re­sults from two phase three clin­i­cal tri­als, demon­strate clear im­prove­ment on sev­er­al im­por­tant mea­sures of dis­ease in these pa­tients. We be­lieve that these re­sults pro­vide strong sup­port to make Waylivra avail­able to treat peo­ple with FCS,” said Paula Soteropou­los, chief ex­ec­u­tive of­fi­cer of Akcea Ther­a­peu­tics. “We look for­ward to work­ing with the FDA to com­plete the fi­nal stages of reg­u­la­to­ry re­view for Waylivra. We are com­mit­ted to the FCS com­mu­ni­ty and will con­tin­ue to fo­cus on bring­ing Waylivra to peo­ple suf­fer­ing with this dev­as­tat­ing dis­ease.”

 

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).