AZ considers filings for long-acting Strensiq follow-up AstraZeneca's long-acting Strensiq follow-up efzimfotase alfa has hit the mark in children with rare disease HPP, but the data in adults is unclear.

#AstraZeneca's #Alexion unit is planning to file a new, long-acting follow-up to its #Strensiq therapy for ultra-rare disease #hypophosphatasia (HPP), although its phase 3 readouts have shown mixed results.

pharmaphorum.com/news/az-cons...

As a patient who's living with a rare genetic condition (hypophosphatasia) and is a beneficiary of such innovations & support #Alexion Pharmaceuticals #PANTHERx Rare Pharmacy, these shared experiences and goals are a constant reminder that life is precious and each new day brings hope for progress.

More good news! My case manager at my specialty pharmacy confirmed that my commercial insurance carrier will cover my treatment with #Strensiq. Initially, my co-pay was $1,220 per month; but with the #Alexion OneSource co-pay program, my co-pay is now $0 per month. I'm sooooo fortunate. 🙏🏼

Good news, my friends. Today, I learned from #Alexion Pharmaceuticals ( #AstraZeneca Rare Disease) that I've been approved to start treatment with #Strensiq for #hypophosphatasia #osteoporosis. Soon, I'll learn from its distributing pharmacy the details on insurance coverage (hopefully, 100%). 🙏🏼

Koselugo（セルトメチニブ）、神経線維腫症1型成人患者に対する初のFDA承認薬に — 第3相KOMET試験結果に基づく | STELLANEWS.LIFE アストラゼネカ傘下のアレクシオンは、神経線維腫症1型（NF1）に伴う症候性・手術不能な神経叢神経線維腫を有する成人に対し、MEK阻害薬Koselugo（セルトメチニブ）がFDAの承認を取得したと発表。KOMET試験で20％の奏効率を示し、治療選択肢が拡大。

FDA approves AstraZeneca’s Koselugo (selumetinib) for adults with neurofibromatosis type 1 (NF1).

🔗 Full article: stellanews.life/technology_c...

#Koselugo #Selumetinib #NF1 #AstraZeneca #Alexion #RareDisease #FDAApproval #MEKinhibitor #ClinicalTrials

【Koselugo（セルメチニブ）、成人NF1患者に対する初のFDA承認薬】
アストラゼネカ（アレクシオン部門）のMEK阻害薬セルメチニブ（Koselugo）が、
神経線維腫症1型（NF1）に伴う症候性・手術不能の叢状神経線維腫（PN）を有する成人患者に対して米国初の承認薬に。
🧬 第3相KOMET試験で
　・奏効率：20％（プラセボ5％）
　・奏効の86％が6か月以上持続
🔹 安全性は小児適応時のデータと整合

#Koselugo #セルメチニブ #NF1 #神経線維腫症 #アストラゼネカ #Alexion #希少疾患 #MEK阻害薬 #KOMET試験

Seltene Erkrankung: Medizinische Versorgung per Zufallsprinzip? Die Diagnose und Therapie einer seltenen Erkrankung ist zu oft vom Zufall abhängig. Auf einer Veranstaltung von Alexion und AstraZeneca Computational Pathology sprachen Fachleute darüber.

Die #Diagnose und #Therapie einer #seltenenErkrankung ist zu oft vom Zufall abhängig. Auf einer Veranstaltung von #Alexion und #AstraZeneca Computational Pathology sprachen #Fachleute darüber.

pharma-fakten.de/news/seltene...

This drug is manufactured by #Alexion ( #AstraZeneca pharmaceuticals) and dispensed by a specialty pharmacy for the entire US. It’s an exorbitantly expensive drug. Depending on body weight, a year’s supply of STRENSIQ can cost up to $2.5 million.

(7/10)

AZ boasts phase 3 win for self-administered gMG drug

#AstraZeneca’s #Alexion is touting positive phase 3 results for a new once-weekly drug for the debilitating neuromuscular condition gMG — but will it be enough to overcome increasingly stiff competition in the space?

pharmaphorum.com/news/az-boas...

Alexion, DHL Express join forces in Ireland’s first 100% switch to SAF 100% sustainable aviation fuel will be used across all Alexion European routes, covering shipments to 19 countries.

Alexion, AstraZeneca Rare Disease, and DHL Express announced a partnership to reduce greenhouse gas emissions (GHG) from the air freight of highly specialised medicines manufactured in Ireland.

#Alexion #DHL #DHLExpress #Ireland #DHLGoGreenPlus #AstraZenecaRareDisease #greenhousegasemissions