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.”

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

BREAK­ING: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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FDA's ad­vanced ther­a­pies of­fice pro­vides more clar­i­ty on gene ther­a­py CMC con­sid­er­a­tions

As the Office of Tissue and Advanced Therapies (OTAT) transforms into the Office of Therapeutic Products (OTP), with new user fee funds and “super office” status, the department focused on cell and gene therapies also opened its doors to a town hall Thursday offering clarification on guidance and regulations for manufacturers.

Some of the major concerns from manufacturers were the CMC considerations between first-in-human studies and late-phase studies supporting a marketing approval.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Pa­tient re­port finds con­sti­pa­tion con­di­tion not well-man­aged, open­ing door for bet­ter ed­u­ca­tion from phar­ma

Advertising for constipation treatments often uses light-hearted humor in an effort to spur open discussions about the sometimes stigmatized topic. However, that may not be enough to get people to take the condition seriously, a new patient report from Phreesia finds.

Fewer than one-fifth (17%) of patients with constipation surveyed understand the longer-term health risks of constipation such as hemorrhoids and bowel incontinence. Many are trying to manage their condition with over-the-counter medicines, but often for much longer than recommended. An equal 68% say they use home remedies or OTC meds to manage constipation. But while 90% understand that OTCs are not intended for long-term use, 50% have used an OTC constipation medicine for more than a year.

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