Take­da to pull key hy­poparathy­roidism drug from the mar­ket af­ter years of man­u­fac­tur­ing woes

Take­da on Tues­day morn­ing made an an­nounce­ment that al­most 3,000 peo­ple with the rare dis­ease known as hy­poparathy­roidism were fear­ing.

Due to un­re­solved sup­ply is­sues and man­u­fac­tur­ing woes, Take­da said it will cut its loss­es and dis­con­tin­ue its hy­poparathy­roidism drug, known as Nat­para (parathy­roid hor­mone), halt­ing all man­u­fac­tur­ing of the drug by the end of 2024, but the en­tire in­ven­to­ry will be avail­able un­til de­plet­ed or ex­pired, a com­pa­ny spokesper­son said via email.

The de­ci­sion to not re-com­mer­cial­ize Nat­para will be a blow to not on­ly the 2,400 peo­ple who were await­ing sup­plies of their re­li­able in­jec­tion since 2019, but al­so the ad­di­tion­al ap­prox­i­mate­ly 400 peo­ple who were ac­cess­ing the drugs via the com­pa­ny’s Spe­cial Use Pro­gram as Take­da sought to re­solve these man­u­fac­tur­ing is­sues over the past five years.

Heather No­vak, an au­thor and hy­poparathy­roid pa­tient who has ac­cessed Nat­para via the Spe­cial Use Pro­gram, told End­points News via email:

Take­da did not com­mu­ni­cate with Spe­cial Use pa­tients di­rect­ly—and I’m dev­as­tat­ed and ter­ri­fied. While Acendis Phar­ma’s new PTH re­place­ment in­jec­tion, Transcon PTH, was sub­mit­ted to the FDA for ap­proval last month, un­less it’s rushed, the time­line goes per­fect­ly, and in­sur­ance ap­proves with­out hes­i­ta­tion, I do not be­lieve it will be a vi­able al­ter­na­tive for Spe­cial Use pa­tients to trans­fer to by 2024 [No­vak lat­er clar­i­fied that she heard an ap­proval may come next spring]. This leaves over 400 peo­ple who can’t stop tak­ing a syn­thet­ic PTH hor­mone with­out life-threat­en­ing re­sults with­out op­tions.

Megan Os­trow­er, head of com­mu­ni­ca­tions at Take­da, told End­points via email that the com­pa­ny “first con­sult­ed and aligned with reg­u­la­to­ry au­thor­i­ties, and have com­mu­ni­cat­ed this up­date to pa­tients’ pre­scribers. We are reach­ing out di­rect­ly to all pa­tients en­rolled in the U.S. Spe­cial Use Pro­gram to al­low for these pa­tients to con­sult with their health­care teams to de­vel­op longer-term treat­ment plans, which is why we have com­mu­ni­cat­ed this in­for­ma­tion now.”

The dif­fi­cul­ties with hy­poparathy­roidism when un­treat­ed oc­cur due to low lev­els of cal­ci­um in the blood. Symp­toms can range from the more mild tin­gling or numb­ness in the fin­gers and toes, to se­vere mus­cle cramps and spasms, as well as breath­ing is­sues that can lead to hos­pi­tal­iza­tion, ac­cord­ing to the Na­tion­al Or­ga­ni­za­tion for Rare Dis­or­ders.

And pa­tients have ex­plained to End­points many of the com­pli­ca­tions that can arise when ac­cess is lost to Nat­para.

Ri­ta Mc­Cul­lough of Le Cen­ter, MN, told End­points via email in 2021 that with­in a week of the 2019 re­call, she end­ed up in the emer­gency de­part­ment twice. She said the con­di­tion af­fects her breath­ing and she has se­vere mus­cle spasms with­out Nat­para.

“I was crushed that I had to go back to all the pills and the alarm every two hours just so I can breathe,” she said.

Mc­Cul­lough, who al­so ac­cessed the drug via the spe­cial use pro­gram, told End­points on Tues­day that she hadn’t been in­formed of the dis­con­tin­u­a­tion pri­or to to­day’s PR.

“To say it’s con­cern­ing is an un­der­state­ment!” she said.

Chris­sy Bon­atch of In­di­ana, PA, who had pre­vi­ous­ly tak­en Nat­para but was not on the spe­cial use pro­gram told End­points, “We feel be­trayed and an­gry that af­ter all this time they just dropped the ball and quit fight­ing for us.”

The is­sues and re­call in 2019 for Nat­para cen­tered on rub­ber par­tic­u­lates orig­i­nat­ing from the rub­ber sep­tum of the car­tridge. And based on com­ments from Take­da on Tues­day, those par­tic­u­late prob­lems were nev­er solved.

Take­da has con­tin­ued to com­mu­ni­cate up­dates about per­sis­tent sup­ply chal­lenges sur­round­ing pro­tein par­ti­cle for­ma­tion that are unique and spe­cif­ic to NAT­PAR/NAT­PARA. Over the past sev­er­al years, Take­da has ex­plored nu­mer­ous ways to ad­dress the NAT­PAR/NAT­PARA pro­tein par­ti­cle is­sue to im­prove sus­tain­able sup­ply. Some of the spe­cif­ic steps have in­clud­ed fo­cused root cause analy­sis, com­pu­ta­tion­al mod­el­ing, eval­u­a­tion and im­ple­men­ta­tion of man­u­fac­tur­ing process changes and re­for­mu­la­tion re­search and de­vel­op­ment. Sep­a­rate­ly, af­ter eval­u­a­tion of the U.S. Com­plete Re­sponse Let­ter re­ceived ear­li­er this year, Take­da de­ter­mined it can­not im­ple­ment a so­lu­tion to the rub­ber par­ti­cle for­ma­tion is­sue

Bob Sanders, chair­man of the board of the non­prof­it Hy­poparathy­roidism As­so­ci­a­tion, pre­vi­ous­ly told End­points that he isn’t aware of any­one who has died from a lack of ac­cess to Nat­para. He said some who have lost ac­cess to Nat­para are tak­ing Eli Lil­ly’s For­teo off-la­bel, while oth­ers are on cal­ci­um sup­ple­ments and Cal­citri­ol, which is not al­ways ap­proved by in­sur­ance com­pa­nies and can cre­ate fi­nan­cial bur­dens for some pa­tients.

“We all hope that there is no cal­ci­um crash as most hos­pi­tals lack the un­der­stand­ing to treat a hy­popara pa­tient be­cause it is so rare,” Sanders said.

Take­da ac­quired Nat­para when it bought out Shire for $62 bil­lion in ear­ly 2019. In 2018, the last full year of Nat­para sales, the treat­ment brought in about $230 mil­lion. Since the Nat­para re­call in Sep­tem­ber 2019, no US rev­enue has been record­ed.

FDA tells Catal­ent to fix is­sues at two man­u­fac­tur­ing sites on its own

The CDMO Catalent will have to fix issues at two manufacturing plants in the US and Europe that were subject to inspections by the FDA this summer, giving the company room to correct the issues without facing further regulatory action.

The FDA gave Catalent a “voluntary action indicated” response to two inspections at the contract manufacturer’s site in Bloomington, IN, and Brussels, Belgium. Fixing the issues on its own is a preferable outcome to facing an “official action indicated” response, meaning that an official warning would be sent out or a sit-down with the FDA would be required.

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Sta­da to place $50M+ in­vest­ment in a new fa­cil­i­ty in Ro­ma­nia

While Romania may conjure up images of vast mountain ranges and tales of medieval kings, one generic manufacturer has broken ground on a new facility there.

German pharma company Stada said Monday that it has placed a €50 million ($51.9 million) investment into a 100,000 square-meter (1.08 million square-foot) site in Turda, Romania, a city in the Southeast of the country. According to a Stada spokesperson in an email to Endpoints News, the company has developed only 281,500 square feet of the site so far.

Vac­cine doc­u­ments, young lead­ers and mar­ket tur­moil: End­points' 10 biggest sto­ries of 2022

It’s been a volatile year in the world of biopharma. Market declines reset M&A valuations, and may be beginning to tempt bigger buyers back into dealmaking. Russia’s war in Ukraine disrupted drug sales and clinical trials. A new generation of young biotech leaders emerged in the Endpoints 20(+1) Under 40. And as capital runs dry in a tough environment for raising new funds, companies big and small are taking a look at their headcounts and operations for ways to make it through lean times.

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Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Albert Bourla, Pfizer CEO (John Thys/POOL/AFP via Getty Images)

Pfiz­er CEO un­der fire from UK watch­dog over vac­cine com­ments — re­port

Pfizer CEO Albert Bourla told the BBC last December that he had “no doubt in my mind that the benefits, completely, are in favor” of vaccinating 5- to 11-year-olds for Covid-19. Almost a year later, those comments have reportedly landed him in trouble with a UK pharma watchdog.

Children’s advocacy group UsForThem filed a complaint with the UK’s Prescription Medicines Code of Practice Authority (PMCPA) last year accusing Bourla of making “disgracefully misleading” statements during the BBC interview, including one that “Covid in schools is thriving.” At the time, UK regulators had not yet cleared the vaccine for the 5 to 11 age group, though the vaccine did have a positive opinion from the EMA’s human medicines committee.

Post-Brex­it UK trade re­port shows 'wor­ry­ing' signs for life sci­ences

The UK life sciences industry has always been a a bright spot for its American trade partners, but a new report from the UK government’s Board of Trade raises some fresh concerns about the UK life sciences in the post-Brexit environment.

The report, published Monday, showed that life science-related trade between the UK and its US and European partners declined, sometimes substantially, over the last five years. For instance, UK life sciences exports from 2016-2021 declined by 17% to Spain, -14% to Italy, -13% to Poland, and -11% to Germany.

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Rachael Rollins (Charles Krupa/AP Images)

US seeks jail time for co-CEO of New Eng­land com­pound­ing cen­ter af­ter dead­ly 2012 fun­gal out­break

The US attorney for the district of Massachusetts late last week called on the state’s district court to sentence the former co-owner of the now-defunct New England Compounding Center to 18 months of jail time for his role in the center’s quality deviations that led to more than 100 people dead from a fungal meningitis outbreak.

Gregory Conigliaro was convicted of conspiring with more than a dozen others at NECC to deceive the FDA and misrepresent the fact that the center was only dispensing drugs pursuant to patient-specific prescriptions.

Merck targets vaccine-hesitant parents in its latest 'Why Vaccines' campaign. (Image: Shutterstock)

Mer­ck­'s lat­est 'Why Vac­ci­nes' cam­paign seeks to bet­ter in­form vac­cine-hes­i­tant moms

From Hollywood couple endorsements to targeted equity efforts, Merck has been pushing the value of vaccinations, especially since the Covid-19 pandemic disruption. Now the pharma is turning to a new target — vaccine-hesitant parents, and moms in particular.

Merck’s “Why Vaccines” latest social media and digital campaign spotlights real-life new moms who have questions about vaccinating their children.

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