EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU mem­ber states on alert over the short­age of two drugs that counter heart at­tacks due to an uptick in de­mand.

On Fri­day, the EMA sent out a warn­ing that two Boehringer In­gel­heim drugs are ex­pe­ri­enc­ing a short­age: Ac­til­yse and Metal­yse. The drugs are used as emer­gency treat­ments for adults ex­pe­ri­enc­ing acute my­ocar­dial in­farc­tion, or a heart at­tack, by dis­solv­ing blood clots that have formed in the blood ves­sels.

The EMA warn­ings said that a rise in de­mand for the drugs is lead­ing to an over­all short­age that is ex­pect­ed to last un­til 2024. Both drugs are man­u­fac­tured at Boehringer’s fa­cil­i­ty in Bib­er­ach, Ger­many.

A Boehringer In­gel­heim spokesper­son told End­points News in an email that the spe­cif­ic caus­es of the short­age in­clude an ag­ing pop­u­la­tion be­ing more sus­cep­ti­ble to heart at­tacks and strokes, as well as rec­om­men­da­tions for the drugs be­ing ex­pand­ed.

In 2022, health­care pro­fes­sion­als are treat­ing ever-in­creas­ing num­bers of pa­tients re­quir­ing throm­bolyt­ic treat­ments such as Ac­til­yse and Metal­yse. In an­tic­i­pa­tion of ris­ing de­mand, Boehringer In­gel­heim has more than dou­bled its pro­duc­tion ca­pac­i­ty at its fa­cil­i­ty in Bib­er­ach, Ger­many, over the last 10 years.

“With ris­ing glob­al de­mand for throm­bolyt­ic treat­ments de­spite all ef­forts we have reached a point where the pro­duc­tion of these med­i­cines is no longer keep­ing up with de­mand,” the spokesper­son said.

With Boehringer In­gel­heim at max­i­mum pro­duc­tion ca­pac­i­ty in Bib­er­ach, the EMA said in its warn­ing that, with­in the next three years, a new man­u­fac­tur­ing site will be es­tab­lished to in­crease ca­pac­i­ty. The com­pa­ny spokesper­son told End­points that it is plan­ning to trans­fer all Ac­til­yse drug man­u­fac­tur­ing to a new site in Vi­en­na, al­low­ing space to free up in Bib­er­ach for Metal­yse pro­duc­tion.

In the mean­time, Boehringer In­gel­heim is tak­ing sev­er­al mea­sures to ex­tend the sup­ply. It is plan­ning to sub­mit a vari­a­tion ap­pli­ca­tion glob­al­ly to ex­tend the shelf life of Metal­yse from 24 to 36 months to avoid the re­turn of un­used vials af­ter the ex­pi­ra­tion date and in­crease the vol­ume of Ac­til­yse’s 20mg dos­es to dis­cour­age the use of the 50mg ver­sion and re­duce prod­uct wastage.

Boehringer In­gel­heim is al­so de­vel­op­ing a man­u­fac­tur­ing process for Ac­til­yse that will give it a high­er yield, which was ap­proved by EU health au­thor­i­ties last month.

For now, the EMA is ad­vis­ing health providers to ex­tend sup­plies for as long as pos­si­ble, and any in­for­ma­tion on al­ter­na­tives should be re­ferred to na­tion­al agen­cies. Ad­di­tion­al­ly, pa­tients should reach out to health providers for more in­for­ma­tion or to re­ceive any al­ter­na­tives.

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.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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

Up­dat­ed: 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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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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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.

#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: In bid for first FDA nod in ge­o­graph­ic at­ro­phy, Apel­lis claims an­oth­er first in eye dis­ease field

CHICAGO — Eight weeks before patients and industry find out if the FDA approves the first treatment for geographic atrophy, an advanced form of age-related macular degeneration, the biotech behind the drug is out with some new data on a secondary endpoint.

In what study investigator Charles Wykoff called the “first direct evidence of function preservation by slowing GA growth” in an investigational treatment, Apellis Pharmaceuticals’ drug pegcetacoplan led to less loss of retinal sensitivity versus sham  within 250 microns on either side of the GA lesion over 24 months.

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BioCryst's new website for its HAE unbranded campaign encourages patients to take charge of treatment decisions.

BioCryst launch­es aware­ness cam­paign around man­age­ment of rare vas­cu­lar dis­ease

While hereditary angioedema (HAE) is rare, treatment options for the condition have become much more common. So BioCryst Pharmaceuticals is taking a new angle in its recently launched HAE awareness campaign encouraging patients to take control of their disease management.

“Hereditary angioedema (HAE) isn’t the author of your story — you are … #cHAEngetheplan. Not the goal,” the US campaign website advises.

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EMA makes new rec­om­men­da­tions for Im­bru­vi­ca, med­i­cines with ter­li­pressin over res­pi­ra­to­ry and car­dio­vas­cu­lar risks

The EMA is handing out a new recommendation surrounding J&J and AbbVie’s cancer drug Imbruvica and some medicines containing terlipressin.

On Friday, the EMA’s safety committee (PRAC) stated that it has taken new measures to reduce the risk of respiratory failure and sepsis when medicines containing terlipressin that are used to treat people with hepatorenal syndrome (HRS-1), particularly for patients with advanced acute-on-chronic liver disease or advanced kidney failure.