Travere Therapeutics is determined to bring life-changing treatments, support, and hope to people with rare disease
We come together every day to help patients, families, and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop, and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow.
Travere Therapeutics is 450+ people with a unique understanding of rare disease who cultivate a culture of compassion and integrity together, and who possess a deep-rooted commitment to people with rare disease.
Many of our team members are rare disease patients, survivors and caregivers themselves. For us, advancing research, delivering life-changing therapies, and doing everything we can to support this community is personal. We know that time matters, and we know that the work that we do can make a difference. We know what hope looks like, but we also know what having no answer and no treatment feels like.
Travere Therapeutics is determined to bring life-changing treatments, support, and hope to people with rare disease – an area often overlooked. Together, we can create a better future for families affected by rare disease.
Our inspiration comes from people living with rare disease. Our decisions and actions are guided by the voices of patients and caregivers. Driven by the urgent need for treatments, we strive in all that we do to make a positive impact on rare patients’ lives and provide hope for a better future.
The path in rare disease is often uncharted. We rely on scientific excellence, grit, perseverance, integrity, bravery, and ambition to forge new paths and bring innovation to people living with rare disease.
We foster an environment of belonging, empowerment, and sharing. We embrace diverse perspectives to create shared understanding that inspires how we work to meet the needs of people living with rare disease.
Our best advancements come from the strength of our teamwork and collaboration with leading experts and the rare disease community. By working together, we can make a difference in their journey.
We are impatient for the patients we serve
We are driven to deliver safe and effective therapies to patients as quickly as possible. To do this, we are advancing a pipeline of potential first-in-class medicines targeting rare diseases with significant unmet needs. Travere recently announced FDA accelerated approval of a therapy for IgA nephropathy, a rare kidney disease (RKD) that is a leading cause of kidney failure due to glomerular disease. We are in late-stage clinical development for the treatment of focal segmental glomerulosclerosis (FSGS), an RKD with no approved treatments. We are evaluating what could potentially become the first disease-modifying therapy for people living with classical homocystinuria (HCU). Our clinical programs also include a Phase 3 study in cerebrotendinous xanthomatosis (CTX), an ultrarare and underdiagnosed leukodystrophy. In partnership with the National Institutes of Health (NIH) National Center for Advancing Translational Sciences (NCATS), and patient advocacy foundation Alagille Syndrome Alliance (ALGSA), we are engaged in preclinical research to identify and develop potentially novel therapeutics for Alagille syndrome (ALGS).
Rare disease presents challenges for accurate and timely diagnosis – costing rare families time, frustration, financial burden – as well as delay in starting any potential therapy. Newborn screening (NBS) is a critical public health tool for the diagnosis of many rare diseases, but rare families face too many challenges with our current newborn screening system. It takes too long to get a new condition approved for screening and even longer for screening to actually start in each state, leading to preventable deaths and disability. In 2021, we supported work on a groundbreaking Newborn Screening Modernization Study that outlined specific weaknesses in the system, and what could be done to change it.
Travere is supporting two transformative new programs in NBS led by leaders in NBS operating in North Carolina and California that can potentially disrupt NBS as we know it through whole genome sequencing (WGS). Early Check is a collaboration between the University of North Carolina (UNC), a National institutes of Health (NIH) Center of Excellence in NBS and WGS, and the North Carolina Department of Public Health; and BeginNGS operates out of Rady Children’s Hospital in San Diego, also an NIH Center of Excellence in NBS and WGS. We see how whole genome sequencing in NBS can potentially dramatically shorten the diagnostic odyssey, which is why we are supporting both initiatives which began in 2022.
80% of rare diseases have identified genetic origins. We are championing earlier diagnosis through genetic testing and investing in areas that extend beyond the scope of our own programs. Travere sponsors a genetic testing program through PreventionGenetics to help diagnose the cause of newborn cholestasis at no cost to qualifying patients. This program has provided thousands of families with answers and enabled them to seek care – typically for conditions outside of Travere’s therapeutic focus.
We are champions of telehealth to streamline optimal access to rare disease care. Travere is a founding sponsor of a telehealth model at Children’s National Rare Disease Institute (CNRDI) in Washington, D.C., to ensure that access to rare disease specialists is not dictated by zip code or mobility. Our work includes advocating for both state and federal policies that would permanently allow telehealth appointments for rare disease patients.
Travere is dedicated to improving access to care for traditionally underserved communities. We worked closely with the leaders of the Black Women’s Health imperative (BWHI) to spearhead the formation of a first-of-its-kind Rare Disease Diversity Coalition – a steering committee of influential, cross-sector leaders who share a passion and commitment to addressing the racial and socioeconomic disparities among people living with rare disease. In 2022, the coalition was focused on advancing initiatives ranging from cultural sensitivity training for providers and medical students, to developing clinical trial awareness and recruitment tools to better engage patients of color.
There are situations in which individuals may not be able to access our investigational products through our clinical trials due to eligibility requirements or geographic distance from a clinical trial site. Some may have a critical need for immediate care. We consider requests for special access to our investigational products through our compassionate use and expanded access programs.
We are focused on better understanding the experiences and challenges facing diverse populations and to elevating diversity and inclusion in every aspect of our research programs. For example, focal segmental glomerulosclerosis (FSGS) is 4 times more common in people of African ancestry. When compared with Caucasian patients, people with IgA nephropathy of Asian ancestry have a worse prognosis and are at an increased risk of progression to kidney failure.
Travere prioritizes investment and action that drives health equity and deeper understanding of diverse patient populations. We aim to spur collaborative engagement in the external community regarding the broader importance of diversity through our work with the Rare Disease Diversity Coalition and our support of the first-ever Diversity Inclusion Advocacy Fellowship at the EveryLife Foundation for Rare Diseases – to increase the representative diversity of patient voices and to engage low income and minority communities in advocacy.
We cannot ignore the cost of rare disease. We supported the Everylife Foundation for Rare Diseases’ Burden of Rare Disease Study (2021), a landmark study that sought to demonstrate the direct, indirect, and non-medical costs of rare disease. This study was foundational to the Governmental Accountability Office’s report (2021) examining the substantial cost of rare disease, a notable US Government-acknowledgement of the comprehensive burden that rare disease places on patients and families. With this evidence, we work alongside patient advocacy organizations to propose changes to federal policies that can improve the lives of rare patients and their families.
The experience of caregivers and care partners is a critical part of understanding rare disease. We support the National Alliance of Caregiving, including their groundbreaking report, Rare Disease Caregiving in America (2018), which gave voice to the millions of unpaid rare caregivers in the US. From workplace flexibility and the impact of caregiving on family life to the role of caregivers in the development of therapies, we recognize the need to treat caregiving as a public health issue. We also recognize that the need for respite care and how difficult it is to find medical nursing care that can step into the shoes of the caregiver. Travere established the Rare Caregiver Respite Program at National Organization for Rare Disorders (NORD), a program that provides rare caregivers much-needed respite.
We are committed to broad access for our US Food and Drug Administration (FDA)-approved therapies, and we provide personalized clinical support services through the Total Care Hub. Through the Hub, patients can get help with insurance verification, reimbursement options, and financial support for our therapies. Hub pharmacists and counselors can answer medication questions, and support people in the day-to-day management of their condition.
We are committed to incorporating the patient voice throughout the drug development process. The patient experience informs clinical concept, protocol development, and how we recruit and communicate with the rare disease community about our work. By including and engaging with people living with rare disease, we are better able to meet the unique needs of the patient communities we serve.