Takeda's rare dis­ease drug's man­u­fac­tur­ing and sup­ply woes con­tin­ue as FDA re­jects sup­ple­ment

Al­most 3 years af­ter Take­da had to re­call a drug that can help peo­ple with the rare con­di­tion known as hy­poparathy­roidism, those wait­ing for any sign that sup­plies of their re­li­able in­jec­tion from Take­da might reemerge will have to wait even longer.

Take­da an­nounced on Tues­day that the FDA re­ject­ed its Pri­or Ap­proval Sup­ple­ment (PAS) sub­mit­ted last Au­gust to ad­dress the for­ma­tion of rub­ber par­tic­u­lates that led to the ini­tial re­call in Sep­tem­ber 2019.

More than 2,000 peo­ple have been strand­ed with­out Nat­para (parathy­roid hor­mone) as they do not have ac­cess to the com­pa­ny’s Spe­cial Use Pro­gram (SUP), which is on­ly for about 400 of those who are at ex­treme risk of life-threat­en­ing com­pli­ca­tions. But when those man­u­fac­tur­ing is­sues will be re­solved re­mains an open ques­tion. The com­pa­ny pre­vi­ous­ly said that it might re­sume sup­ply by next week, but now that fore­cast has been thrown out.

Light on de­tails in its an­nounce­ment on the CRL, Take­da did make clear that it will con­tin­ue to pro­vide ac­cess to Nat­para free of charge via the SUP, and that prod­uct will be sup­plied un­der the dis­cre­tion of the FDA and un­til com­mer­cial prod­uct is avail­able. The com­pa­ny al­so ex­plained sep­a­rate man­u­fac­tur­ing is­sues it’s ad­dress­ing re­lat­ed to Nat­para and said it’s tak­ing a deep­er look at the CRL:

With the goal of lim­it­ing sup­ply in­ter­rup­tion for SUP pa­tients, we con­tin­ue to work on the sep­a­rate sup­ply chal­lenges sur­round­ing pro­tein par­ti­cle for­ma­tion that we have de­scribed over the past year. Those chal­lenges are un­re­lat­ed to the PAS and the re­call. It is im­por­tant to un­der­score that all prod­uct re­leased for pa­tient use con­tin­ues to meet Take­da’s qual­i­ty stan­dards and the safe­ty pro­file of NAT­PARA has not changed.

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 con­cerns are grow­ing among pa­tients that the drug may nev­er re­turn to mar­ket.

Heather No­vak, a Michi­gan-based au­thor who takes Nat­para to treat her hy­poparathy­roidism, told End­points News via email that she cur­rent­ly does still have ac­cess to her dose and sup­plies thanks to the SUP. “I have friends on high­er dos­es who have shift­ed strengths be­cause of the short­ages, but I am thank­ful­ly not one of them,” she said.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Am­gen takes next step with its Chi­na am­bi­tions, out-li­cens­ing drugs to Fo­s­un Phar­ma

In a bid to increase its market share in China, Amgen has agreed to a partnership with a Shanghai biotech — a collaboration and out-licensing agreement for two of its drugs.

Amgen and Fosun Pharma announced a deal Monday in a bid to increase Amgen’s presence in the country. The stated goal so far is to commercialize Amgen’s blockbuster psoriasis drug Otezla alongside Parsabiv, a drug for secondary hyperparathyroidism in adults with chronic kidney disease and on a specific type of dialysis.

As court case looms, Bris­tol My­ers touts la­bel ex­pan­sion for Breyanzi

As Bristol Myers Squibb braces for a court battle over a costly delay — at least for Celgene shareholders — for its CAR-T lymphoma treatment Breyanzi, the pharma giant is touting a label expansion in the second-line setting.

Breyanzi, also known as liso-cel, snagged a win on Friday in adults with large B-cell lymphoma (LBCL) who: don’t respond to chemotherapy, or relapse within 12 months; don’t respond or relapse after 12 months; or are not eligible for hematopoietic stem cell transplant after chemo due to their age or comorbidities.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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