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Arrowhead Pharmaceuticals Launches on Rare Disease Day New Patient-Centric Resources for Those Affected by Familial Chylomicronemia Syndrome (FCS)

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Arrowhead Pharmaceuticals (NASDAQ: ARWR) has expanded its 'We'll Get There Soon' campaign on Rare Disease Day with new patient-centric resources for Familial Chylomicronemia Syndrome (FCS). The initiative includes the launch of www.LowerMyTGs.com, a dedicated educational website, and the release of the 'Spotlight on FCS' white paper.

FCS is an ultra-rare genetic disorder affecting 1-13 people per million globally, impairing the body's ability to process triglycerides. Patients typically experience dangerously high triglyceride levels exceeding 880 mg/dL, increasing the risk of acute pancreatitis. Expert guidelines recommend maintaining levels below 500 mg/dL.

The new resources, developed in collaboration with the FCS Foundation, Action FCS, and FH Europe Foundation, provide educational content, patient testimonials, and community connection opportunities. The initiative aims to amplify patient voices and address the underdiagnosis of FCS while supporting those affected by extremely high triglycerides.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) ha ampliato la sua campagna 'Arriveremo Presto' in occasione della Giornata delle Malattie Rare con nuove risorse centrate sui pazienti per la Sindrome da Camilomicrocitemia Familiare (FCS). L'iniziativa include il lancio di www.LowerMyTGs.com, un sito web educativo dedicato, e la pubblicazione del white paper 'Focus sulla FCS'.

La FCS è un disturbo genetico ultra-raro che colpisce 1-13 persone ogni milione a livello globale, compromettendo la capacità del corpo di elaborare i trigliceridi. I pazienti sperimentano tipicamente livelli di trigliceridi pericolosamente elevati, superiori a 880 mg/dL, aumentando il rischio di pancreatite acuta. Le linee guida degli esperti raccomandano di mantenere i livelli al di sotto di 500 mg/dL.

Le nuove risorse, sviluppate in collaborazione con la FCS Foundation, Action FCS e la FH Europe Foundation, offrono contenuti educativi, testimonianze di pazienti e opportunità di connessione con la comunità. L'iniziativa mira ad amplificare le voci dei pazienti e affrontare la sotto-diagnosi della FCS, supportando coloro che sono colpiti da trigliceridi estremamente elevati.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) ha ampliado su campaña 'Llegaremos Pronto' en el Día de las Enfermedades Raras con nuevos recursos centrados en el paciente para el Síndrome de Quilomicronemia Familiar (FCS). La iniciativa incluye el lanzamiento de www.LowerMyTGs.com, un sitio web educativo dedicado, y la publicación del documento técnico 'Enfoque en la FCS'.

La FCS es un trastorno genético ultra-raro que afecta a 1-13 personas por millón a nivel mundial, perjudicando la capacidad del cuerpo para procesar triglicéridos. Los pacientes suelen experimentar niveles peligrosamente altos de triglicéridos que superan los 880 mg/dL, aumentando el riesgo de pancreatitis aguda. Las pautas de expertos recomiendan mantener los niveles por debajo de 500 mg/dL.

Los nuevos recursos, desarrollados en colaboración con la FCS Foundation, Action FCS y la FH Europe Foundation, proporcionan contenido educativo, testimonios de pacientes y oportunidades de conexión comunitaria. La iniciativa tiene como objetivo amplificar las voces de los pacientes y abordar el subdiagnóstico de la FCS, al tiempo que apoya a aquellos afectados por triglicéridos extremadamente altos.

Arrowhead Pharmaceuticals (NASDAQ: ARWR)는 희귀 질환의 날을 맞아 '곧 도착할 것입니다' 캠페인을 확대하여 가족성 카일로미크론혈증 증후군(FCS)을 위한 새로운 환자 중심 자원을 발표했습니다. 이 이니셔티브에는 www.LowerMyTGs.com이라는 전용 교육 웹사이트의 출시와 'FCS에 대한 조명' 백서의 발행이 포함됩니다.

FCS는 전 세계적으로 백만 명당 1-13명의 사람에게 영향을 미치는 초희귀 유전 질환으로, 신체의 트리글리세리드를 처리하는 능력을 저해합니다. 환자들은 일반적으로 880 mg/dL를 초과하는 위험한 수준의 트리글리세리드를 경험하며, 이는 급성 췌장염의 위험을 증가시킵니다. 전문가 가이드라인은 500 mg/dL 이하로 유지할 것을 권장합니다.

FCS 재단, Action FCS 및 FH 유럽 재단과 협력하여 개발된 새로운 자원은 교육 콘텐츠, 환자 증언 및 커뮤니티 연결 기회를 제공합니다. 이 이니셔티브는 환자의 목소리를 증대시키고 FCS의 저진단 문제를 해결하며, 극도로 높은 트리글리세리드로 영향을 받는 사람들을 지원하는 것을 목표로 합니다.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) a élargi sa campagne 'Nous y arriverons bientôt' à l'occasion de la Journée des Maladies Rares avec de nouvelles ressources centrées sur les patients pour le Syndrome de Chylomicronémie Familiale (FCS). L'initiative comprend le lancement de www.LowerMyTGs.com, un site web éducatif dédié, ainsi que la publication du document blanc 'À la une sur la FCS'.

La FCS est un trouble génétique ultra-rare qui touche 1 à 13 personnes par million dans le monde, affectant la capacité du corps à traiter les triglycérides. Les patients connaissent généralement des niveaux de triglycérides dangereusement élevés dépassant 880 mg/dL, augmentant le risque de pancréatite aiguë. Les directives des experts recommandent de maintenir les niveaux en dessous de 500 mg/dL.

Les nouvelles ressources, développées en collaboration avec la FCS Foundation, Action FCS et la FH Europe Foundation, fournissent un contenu éducatif, des témoignages de patients et des opportunités de connexion communautaire. L'initiative vise à amplifier les voix des patients et à aborder le sous-diagnostic de la FCS tout en soutenant ceux qui sont touchés par des triglycérides extrêmement élevés.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) hat seine Kampagne 'Wir werden bald da sein' zum Tag der seltenen Krankheiten mit neuen patientenorientierten Ressourcen für das familiäre Chylomikronämie-Syndrom (FCS) erweitert. Die Initiative umfasst den Start von www.LowerMyTGs.com, einer speziellen Bildungswebsite, sowie die Veröffentlichung des Whitepapers 'Fokus auf FCS'.

FCS ist eine ultrarare genetische Erkrankung, die weltweit 1-13 Personen pro Million betrifft und die Fähigkeit des Körpers beeinträchtigt, Triglyceride zu verarbeiten. Patienten erleben typischerweise gefährlich hohe Triglyceridwerte von über 880 mg/dL, was das Risiko einer akuten Pankreatitis erhöht. Expertenrichtlinien empfehlen, die Werte unter 500 mg/dL zu halten.

Die neuen Ressourcen, die in Zusammenarbeit mit der FCS Foundation, Action FCS und der FH Europe Foundation entwickelt wurden, bieten Bildungsinhalte, Patientenerfahrungen und Möglichkeiten zur Vernetzung der Gemeinschaft. Die Initiative zielt darauf ab, die Stimmen der Patienten zu verstärken und die Unterdiagnose von FCS anzugehen, während sie diejenigen unterstützt, die von extrem hohen Triglyceriden betroffen sind.

Positive
  • Launch of comprehensive patient education platform expanding market presence in FCS space
  • Strategic partnerships with multiple FCS foundations strengthening market position
  • Advancement in RNAi-based therapy development for rare diseases
Negative
  • None.
  • New Patient Resource Hub: Arrowhead expands its We’ll Get There Soon campaign with the launch of a dedicated patient website, providing education, tools, and support for those living with FCS and extremely high triglycerides
  • Amplifying Patient Voices: The Spotlight on FCS white paper shares real patient experiences, highlighting the challenges of managing this ultra-rare disease and the need for greater awareness
  • Commitment to the FCS Community: On Rare Disease Day and beyond, Arrowhead remains dedicated to elevating patient voices and fostering community engagement. #ForFCS

PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) marks Rare Disease Day with the expansion of the We’ll Get There Soon campaign, initially launched for healthcare providers in November 2024. This new phase introduces a dedicated educational website, www.LowerMyTGs.com, and the Spotlight on FCS white paper, which provides essential resources and support for those affected by Familial Chylomicronemia Syndrome (FCS).

The Power of Knowing: Living with Familial Chylomicronemia Syndrome (FCS) http://www.lowermytgs.com/ (Graphic: Business Wire)

The Power of Knowing: Living with Familial Chylomicronemia Syndrome (FCS) http://www.lowermytgs.com/ (Graphic: Business Wire)

FCS is an ultra-rare genetic disorder that significantly impairs the body's ability to process triglycerides, leading to dangerously high triglyceride (TG) levels — typically exceeding 880 mg/dL — and increasing the risk of life-threatening complications, such as acute pancreatitis. Because of this risk, expert guidelines recommend maintaining triglyceride levels below 500 mg/dL. FCS remains widely underdiagnosed and is estimated to affect between 1 and 13 people per million globally. To address this gap, Arrowhead’s new initiatives aim to amplify patient voices, foster community, and provide accessible educational tools.

Arrowhead’s new patient website serves as a comprehensive resource for those affected by FCS and extremely high triglycerides. It provides educational content, patient testimonials, and opportunities for community connection. Additionally, the Spotlight on FCS white paper, developed with engagement and support of the FCS Foundation, Action FCS, and FH Europe Foundation, captures firsthand patient experiences, highlighting key challenges in disease management and aiming to drive greater awareness and action.

One such patient testimonial can be found on the patient website here.

“In recognition of Rare Disease Day, we prioritize giving the FCS community a voice. Arrowhead’s Spotlight on FCS white paper, which highlights what life looks like for those living with this ultra-rare condition, is a novel look at how these individuals are navigating life with FCS,” said Lindsey Sutton, co-founder of the FCS Foundation. “This paper is a major contribution to the community as it sheds a light on the unmet needs patients face with their disease from their firsthand experiences.”

Andy Davis, Senior Vice President, Global Cardiometabolic Franchise at Arrowhead, added, “For nearly two decades, Arrowhead has been dedicated to advancing RNAi-based therapies to address challenging diseases. These new resources reaffirm our commitment to the FCS community by enhancing awareness, support, and access to information through the expansion of the We’ll Get There Soon campaign.”

Explore the new resources at www.LowerMyTGs.com.

#ForFCS #RareDiseaseDay #WellGetThereSoon

About Familial Chylomicronemia Syndrome

Familial chylomicronemia syndrome (FCS) is a severe and rare disease often caused by various monogenic mutations. FCS leads to extremely high triglyceride (TG) levels, typically over 880 mg/dL. Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, there are limited therapeutic options to adequately treat FCS.

About the FCS Foundation

Established in 2016 by two disease advocates directly impacted by FCS, the FCS Foundation connects the FCS community and provides resources and support for those with FCS. The Foundation hosts an annual Patient & Caregiver Meeting and works year-round to raise awareness and advocate for FCS. The Foundation operates as an all-volunteer organization alongside a medical advisory board of specialists working to advance future treatments. In Spring 2025, the Foundation will have expanded patient resources available for the community. For more information about the Foundation's mission and initiatives, please visit their official website at livingwithfcs.org.

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

For more information, please visit www.arrowheadpharma.com, or follow us on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.

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Source: Arrowhead Pharmaceuticals, Inc.

Arrowhead Pharmaceuticals, Inc.

Vince Anzalone, CFA

626-304-3400

ir@arrowheadpharma.com

Investors:

LifeSci Advisors, LLC

Brian Ritchie

212-915-2578

britchie@lifesciadvisors.com

Media:

LifeSci Communications, LLC

Kendy Guarinoni, Ph.D.

724-910-9389

kguarinoni@lifescicomms.com

Source: Arrowhead Pharmaceuticals, Inc.

FAQ

What is the prevalence rate of Familial Chylomicronemia Syndrome (FCS) according to ARWR's release?

According to Arrowhead Pharmaceuticals, FCS affects between 1 and 13 people per million globally.

What are the dangerous triglyceride levels associated with FCS mentioned in ARWR's announcement?

FCS patients typically have triglyceride levels exceeding 880 mg/dL, while expert guidelines recommend maintaining levels below 500 mg/dL.

What new resources has ARWR launched for FCS patients in 2025?

Arrowhead launched www.LowerMyTGs.com, a dedicated educational website, and released the 'Spotlight on FCS' white paper.

Which organizations collaborated with ARWR on the FCS patient resources?

The FCS Foundation, Action FCS, and FH Europe Foundation collaborated with Arrowhead on developing these resources.

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