Since Sep­tem­ber 2019, 2,400 peo­ple with the rare con­di­tion known as hy­poparathy­roidism have been wait­ing for any sign that sup­plies of their re­li­able in­jec­tion from Take­da might reemerge.

Last Wednes­day, how­ev­er, Take­da an­nounced that sup­plies won’t be ready for at least an­oth­er year, strand­ing those who still can’t ac­cess the drug, known as Nat­para (parathy­roid hor­mone), via the com­pa­ny’s Spe­cial Use Pro­gram (SUP), which is on­ly for about 400 of those who are at ex­treme risk of life-threat­en­ing com­pli­ca­tions.

“At this time we do not ex­pect a re­turn to mar­ket be­fore March 31, 2022,” Take­da said in a let­ter.

The dif­fi­cul­ties with hy­poparathy­roidism 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.

Even for those who still have ac­cess to Nat­para via Take­da’s spe­cial pro­gram, last week’s an­nounce­ment was a dif­fi­cult one.

“The an­nounce­ment was def­i­nite­ly a dev­as­tat­ing one for the en­tire hy­popara com­mu­ni­ty, es­pe­cial­ly af­ter the vir­tu­al hy­popara con­fer­ence in Oc­to­ber, where Take­da had re­marked that they hoped to an­nounce their plans to re­turn to mar­ket in March,” Heather No­vak, a De­troit-based ro­mance nov­el­ist who is on the SUP but told End­points News via email that she al­so deals with in­ter­mit­tent sup­ply is­sues.

In its up­date, Take­da of­fered few specifics on what’s caus­ing the man­u­fac­tur­ing is­sues at its con­trac­tor plants that are over­seen by Take­da. Back in 2019, FDA an­nounced the Class I (the high­est de­gree of po­ten­tial health haz­ard) re­call for Nat­para, and said it re­lates to rub­ber stop­per par­ti­cles clog­ging the nee­dle and lead­ing to un­der­dos­ing, ac­cord­ing to let­ters sent to pa­tients.

In its up­date, Take­da told pa­tients last week, “While we have made progress on the orig­i­nal is­sue that led to the U.S. re­call, which was the is­sue of rub­ber par­tic­u­lates orig­i­nat­ing from the rub­ber sep­tum of the NAT­PARA car­tridge, we have not yet reached a res­o­lu­tion.”

An­oth­er man­u­fac­tur­ing de­lay, which Take­da con­firmed is sep­a­rate from the orig­i­nal is­sue in 2019, is al­so af­fect­ing sup­plies the 100-mcg ver­sion of Nat­para un­der the SUP. No­vak ex­plained that when there’s an ex­pect­ed short­age of one one or more strengths of Nat­para, like the 100-mcg ver­sion, Take­da works with doc­tors to switch pre­scrip­tions or dos­ing for pa­tients to pre­vent any­one on the SUP from go­ing with­out it.

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 and the con­cerns are grow­ing.

“We’ve been told that they plan to keep the SUP go­ing un­til the drug re­turns to mar­ket. Many of us, of course, are won­der­ing IF it will re­turn to the US mar­ket,” No­vak said.

Those who haven’t gained ac­cess to the SUP are strug­gling even more with the Take­da de­lays.

Ri­ta Mc­Cul­lough of Le Cen­ter, MN, told End­points via email 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, not­ing that Nat­para did work for her. “The form let­ters Take­da re­leas­es are point­less. I want to know what’s go­ing on with the rub­ber par­tic­u­lates.”

Bob Sanders, chair­man of the board of the non­prof­it Hy­poparathy­roidism As­so­ci­a­tion, said 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 for pur­chase 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 told End­points News via email. “An­oth­er year with­out Nat­para rep­re­sents an­oth­er year of symp­toms and strug­gles.”

He added that his as­so­ci­a­tion has a lis­ten­ing ses­sion sched­uled with the FDA and he hopes to get a pa­tient-fo­cused drug de­vel­op­ment meet­ing sched­uled for next year “to talk about our con­cerns and how the agency can best help the pa­tient com­mu­ni­ty.”

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As co-director of an experimental cellular therapy process development and manufacturing group at UCSF specializing in T cell therapies for autoimmune conditions, Jonathan Esensten has learned a lot about the challenges involved when his group hand-fashions a cell therapy. Esensten — who was a postdoc in Wendell Lim’s lab and counts the legendary Jeffrey Bluestone as a mentor — gives them all high marks at being great at what they do, but time and again there are variations in the treatments they construct.

Anand Shah has served the American public in a unique way, crisscrossing over the last two administrations between serving as an attending radiation oncologist focused on prostate cancer at NIH, serving as CMO at the Center for Medicare and Medicaid Innovation, and most recently, leading the FDA’s operations on medical and scientific affairs from within the commissioner’s office.

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