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.”

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Aduhelm OK 'bit­ter­sweet' for ALS ad­vo­cates; Con­trast­ing Covid-19 vac­cine read­outs; GSK joins TIG­IT bat­tle; 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.

With the busiest days of June now behind us, we’re starting to think seriously about the second half of the year. In August, we have scheduled a special report where Endpoints will compile a list of the 20 most influential R&D executives in biopharma. Know a luminary who should definitely be included? Nominate them now.

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Michael Chambers (L) and John Ballantyne

Dana­her strikes deal to buy boom­ing next-gen man­u­fac­tur­er Alde­vron for $9.6B

Life sciences conglomerate Danaher Corp. $DHR has struck a deal to buy the fast-growing Aldevron, one of the world’s top manufacturers of hotly sought-after plasmid DNA, mRNA and recombinant proteins for the burgeoning world of vaccine and drugmakers pushing some game-changing technologies.

Buyout talks set the stage for Danaher to settle on a $9.6 billion cash pact to acquire the private Fargo, ND-based company — a key supplier for a disruptive new Covid vaccine as well as a host of gene and cell therapy and CRISPR gene editing players — founded by Michael Chambers and CSO John Ballantyne as a crew of 2 back in 1998.

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Who are the lu­mi­nar­ies dri­ving the biggest ad­vances in bio­phar­ma R&D? End­points News is ask­ing for your nom­i­na­tions for a spe­cial re­port

In biopharma, driving a drug to market is the ultimate goal — but none of that happens without a strong research and development program. At the most successful companies, those R&D efforts are spearheaded by true innovators in the field who are always looking for that next novel mechanism of action or breakthrough safety profile.

Now, Endpoints News is asking you to tell us who those guiding lights are.

Leen Kawas, Athira CEO

Biotech founder placed on leave as $400M Alzheimer's start­up idea comes un­der scruti­ny

Athira Pharma, the Alzheimer’s biotech that emerged out of obscurity last year and raised nearly $400 million for a dark-horse approach to treating neurodegeneration, has found itself in sudden turmoil.

On Tuesday evening, the company released a terse statement announcing that CEO and founder Leen Kawas had been placed on administrative leave while an independent review board investigated “actions stemming” from her doctoral research at Washington State University. Mark Litton, who joined the company as COO two years ago, will take over day-to-day operations, they said.

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Bris­tol My­ers breaks the bank on Ei­sai's fo­late re­cep­tor ADC drug, lay­ing out more than $3B+ for rights

For years, innovation in oncology has been a crapshoot with Big Pharma — the whales at the table — dropping the big bucks for the key to the next generation of tumor fighters. Bristol Myers Squibb hasn’t exactly made a name for being an innovator in the space, but that doesn’t mean it won’t splash in when it sees a potential winner.

Now, with a massive check in hand, the drugmaker is willing to put its intuition to the test.

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FDA's con­tro­ver­sial Aduhelm de­ci­sion leaves ALS pa­tients feel­ing spurned

The FDA’s controversial approval of Biogen’s Aduhelm drug for Alzheimer’s disease has been met with fierce resistance from all corners of the biopharma industry, but few seem to be as upset with the decision as ALS patients and advocacy groups.

For all that’s already been written and discussed about the agency’s announcement, from the drug’s exorbitantly high price of $56,000 per year to criticism over lowered standards, ALS patients see something more. ALS patients and associations say they largely regarded Aduhelm’s approval as a bittersweet double standard: happy that those with Alzheimer’s have a new drug available, but questioning how the FDA evaluated Biogen’s drug compared to the experimental programs being studied for their own disease.

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As drug­mak­ers spend $6B an­nu­al­ly on DTC ads, sen­a­tors re­vive bill to in­clude list prices in ads

A new GAO report on biopharma companies’ $6 billion annual spending on direct-to-consumer advertising is pushing US Senate Majority Whip Dick Durbin (D-IL) and Sen. Chuck Grassley (R-IA) to reintroduce legislation that would require price disclosures in the ads.

The GAO found that drugmakers spent almost half—$8.2 billion of the $17.8 billion from 2016 to 2018—on DTC ads for drugs in three therapeutic categories, including inflammatory conditions (e.g., arthritis, gout), endocrine and metabolic disorders (e.g., type 2 diabetes, hypothyroidism), and conditions affecting the central nervous system (e.g., depression, multiple sclerosis), according to the new report.

Med­ic­aid com­mis­sion to Con­gress: In­crease re­bates for ac­cel­er­at­ed ap­proval drugs

As the FDA continues to approve more new drugs under its accelerated approval pathway, the non-partisan Medicaid and CHIP Payment and Access Commission (MACPAC) is telling Congress to increase the statutory Medicaid rebates for such drugs until their clinical benefits have been verified.

Higher rebates for drugs with accelerated approvals, a move opposed by the biopharma industry, would mean lower net prices, lessening their financial burden on the health care system while incentivizing the companies to speed the verification of the drugs’ clinical benefits in confirmatory trials. Once those benefits are confirmed, the companies would return to the lower rebates when the accelerated approval is converted into a full approval, MACPAC suggests.