In a come­back, Por­to­la scores FDA ac­cel­er­at­ed ap­proval for once-re­ject­ed An­dexxa

Some­how stay­ing on the good side of the FDA, Por­to­la $PT­LA has scored an elu­sive reg­u­la­to­ry win for their once-re­ject­ed drug an­dex­anet al­fa.

The an­ti-an­ti­co­ag­u­lant — to be mar­ket­ed as An­dexxa — won the OK via the ac­cel­er­at­ed ap­proval path­way, mean­ing that Por­to­la will have to run a post-mar­ket­ing tri­al to demon­strate an abil­i­ty to con­trol bleed­ing in pa­tients.  The ap­proval was based on da­ta from two Phase III stud­ies, con­duct­ed among healthy vol­un­teers, that showed An­dexxa could re­verse an­ti­co­ag­u­lant ac­tiv­i­ty of the Fac­tor Xa in­hibitors Xarel­to (ri­varox­a­ban) and Eliquis (apix­a­ban). The drug al­so comes with a black box warn­ing and is in­di­cat­ed for use when re­ver­sal of an­ti­co­ag­u­la­tion is need­ed due to life-threat­en­ing or un­con­trolled bleed­ing.

Bill Lis

Por­to­la has fought long and hard for this win. It first sub­mit­ted an NDA for An­dexxa late 2015, on­ly to be caught off guard with an FDA re­jec­tion. At the time, the South San Fran­cis­co biotech said the agency was look­ing for more in­for­ma­tion on man­u­fac­tur­ing and how the an­ti­dote works in oth­er blood thin­ning drugs, such as edox­a­ban (Savaysa) and enoxa­parin (Lovenox).

The team filed again in Au­gust 2017, ex­act­ly a year af­ter it got the com­plete re­sponse let­ter. Reg­u­la­tors have since pushed back the ac­tion date from Feb­ru­ary to May 4, and Por­to­la hint­ed in a re­cent earn­ings call that there could be fur­ther de­lays as the FDA re­quest­ed fur­ther da­ta, send­ing stock down. But noth­ing came out of that warn­ing, and Por­to­la spread word about the OK late Thurs­day — right be­fore the orig­i­nal ac­tion date.

A spokesper­son told me that the CRL Por­to­la re­ceived in 2016 “fo­cused al­most sole­ly on man­u­fac­tur­ing con­cerns, which Por­to­la ad­dressed.”

“An­dexxa is a break­through prod­uct, and as such, clin­i­cal was much faster than man­u­fac­tur­ing,” they wrote. “In oth­er words, at that time, man­u­fac­tur­ing had to catch up with clin­i­cal.”

In a lengthy press re­lease, Por­to­la did not elab­o­rate on how it re­solved the FDA’s pre­vi­ous con­cerns, choos­ing in­stead to high­light the mar­ket po­ten­tial of its re­ver­sal agent in an era when Fac­tor Xa in­hibitors have be­come in­creas­ing pop­u­lar. One of them is Por­to­la’s own Bevyxxa, whose ap­proval raised some eye­brows since it bare­ly failed a piv­otal tri­al. Eu­ro­pean reg­u­la­tors gave it a thumbs down.

“We are proud that An­dexxa is a first-in-class med­i­cine dis­cov­ered in our labs,” said CEO Bill Lis. “We re­main com­mit­ted to our sci­en­tif­ic lead­er­ship in the fields of throm­bo­sis and hema­to­log­ic can­cers.”

The com­pa­ny now plans to launch an ear­ly sup­ply pro­gram of “Gen­er­a­tion 1” An­dexxa in June, with broad­er com­mer­cial launch an­tic­i­pat­ed in ear­ly 2019, as­sum­ing the FDA green­lights the man­u­fac­tur­ing process of its Gen­er­a­tion 2 prod­uct.

Mean­while, the post-mar­ket­ing tri­al is slat­ed to be­gin in 2019 and be re­port­ed in 2023.

Por­to­la shares edged up 3% in pre-mar­ket trad­ing. Ac­cord­ing to Eval­u­atePhar­maAn­dexxa car­ries a 2022 sell­side rev­enue fore­cast of $749m.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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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.

Bris­tol My­ers Squibb sues No­var­tis for roy­al­ties sur­round­ing the use of trans­genic mice

Two Big Pharma companies are going to the mat over genetically modified mice in a licensing dispute.

Bristol Myers Squibb is suing Novartis in New York over a dispute concerning an evaluation, research and commercialization agreement stretching back to the late ’90s initially inked between Novartis and BMS’ predecessor Medarex. The deal in question allowed Novartis to use Medarex’s patented transgenic mice to develop therapeutic drugs. Novartis agreed to pay Medarex – and subsequently BMS – a royalty on sales of drugs it developed using the mice.