Arrowhead Pharmaceuticals Launches FCS Patient Resources on Rare Disease Day
Arrowhead Pharmaceuticals Expands “We’ll Get There Soon” Campaign with New Resources for Familial Chylomicronemia Syndrome on Rare Disease Day
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) is marking Rare Disease Day with a significant expansion of its “We’ll Get There Soon” campaign, originally launched in November 2024 to support healthcare providers. This new phase introduces a patient-centric approach, aiming to provide critical educational resources and advocacy for individuals affected by Familial Chylomicronemia Syndrome (FCS). Central to this initiative is the launch of a dedicated educational website, www.LowerMyTGs.com, and the release of the Spotlight on FCS white paper, which offers vital information on FCS, its challenges, and the patient experience.
Addressing an Unmet Need in the FCS Community
FCS is an ultra-rare genetic disorder that severely impairs the body’s ability to break down triglycerides. This leads to extremely elevated triglyceride (TG) levels, often exceeding 880 mg/dL, which significantly increases the risk of life-threatening complications such as acute pancreatitis. Given this risk, expert guidelines recommend keeping triglyceride levels below 500 mg/dL. However, due to its rarity, FCS remains widely underdiagnosed, with estimates suggesting it affects between 1 and 13 people per million globally.
The symptoms of FCS, including persistent abdominal pain, fatigue, cognitive difficulties, and an increased risk of pancreatitis, can be life-altering. Many patients struggle for years before receiving a correct diagnosis, often misattributed to other lipid disorders. This diagnostic challenge underscores the importance of increased awareness and access to reliable educational resources.
Arrowhead Pharmaceuticals recognizes this gap and aims to empower both patients and the broader medical community with targeted initiatives to enhance understanding, early diagnosis, and better disease management.
Launch of New Patient Resources
To support individuals living with FCS, Arrowhead Pharmaceuticals has introduced a new educational website, www.LowerMyTGs.com. The platform provides a comprehensive resource hub, offering:
- Educational Content – Clear, accessible information about FCS, its symptoms, and the underlying genetic causes.
- Patient Testimonials – Firsthand stories from individuals navigating life with FCS, highlighting the real-world challenges and strategies for disease management.
- Community Support – Opportunities for patients to connect, share experiences, and find support networks.
- Updates on Advances in Research – Information on ongoing clinical studies and emerging therapeutic approaches in the FCS space.
Additionally, the Spotlight on FCS white paper, developed in collaboration with the FCS Foundation, Action FCS, and the FH Europe Foundation, provides an in-depth look into the experiences of FCS patients. By capturing patient perspectives, the report aims to shed light on the daily struggles faced by those living with the condition and the urgent need for greater awareness and medical intervention.
Giving a Voice to the FCS Community
For those living with FCS, the Spotlight on FCS white paper is a groundbreaking resource. It captures the voices of patients, shedding light on the personal and medical challenges associated with this condition. The paper underscores the need for improved diagnostic strategies, better disease management, and stronger advocacy efforts to drive change within the rare disease community.
Lindsey Sutton, co-founder of the FCS Foundation, emphasized the significance of this initiative:
“In recognition of Rare Disease Day, we prioritize giving the FCS community a voice. Arrowhead’s Spotlight on FCS white paper highlights what life looks like for those living with this ultra-rare condition. It provides a novel look at how these individuals navigate life with FCS. This paper is a major contribution to the community as it sheds light on the unmet needs patients face, directly from their firsthand experiences.”
The report aims to educate not only patients and caregivers but also healthcare professionals who may be unfamiliar with the complexities of diagnosing and managing FCS. Through this initiative, Arrowhead Pharmaceuticals is strengthening the bridge between scientific research and patient advocacy, ensuring that the patient perspective is at the heart of its efforts.
Arrowhead’s Commitment to Rare Disease Innovation
Arrowhead Pharmaceuticals has been at the forefront of RNA interference (RNAi)-based therapies for nearly two decades, focusing on developing treatments for challenging and underserved conditions. The company’s work in lipid metabolism and rare diseases has positioned it as a leader in innovative approaches to treating genetic disorders like FCS.
Andy Davis, Senior Vice President of Arrowhead’s Global Cardiometabolic Franchise, reaffirmed the company’s dedication to the FCS community:
“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.”
Through continuous research and engagement with the rare disease community, Arrowhead aims to not only develop groundbreaking treatments but also advocate for policy changes that improve the standard of care for FCS patients.
The Importance of Raising Awareness on Rare Disease Day
Rare Disease Day, observed annually on the last day of February, is a global initiative to raise awareness about rare diseases and advocate for better support systems for affected individuals. For those living with conditions like FCS, Rare Disease Day serves as a powerful reminder of the importance of research, patient education, and policy advocacy.
By launching the next phase of the We’ll Get There Soon campaign on Rare Disease Day, Arrowhead Pharmaceuticals is emphasizing its commitment to providing meaningful solutions for the rare disease community. The company hopes that these new resources will not only support patients in their journey but also spark broader conversations about the need for continued investment in rare disease research and treatment development.
