UP­DAT­ED: 2,400 peo­ple re­main with­out rare dis­ease drug as Take­da man­u­fac­tur­ing woes con­tin­ue

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.”

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

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