Alfasigma Specialty Pharmaceuticals

Building a healthier tomorrow for people living with rare liver diseases

At Alfasigma, we are dedicated to advancing innovative medicines for people living with rare and serious liver diseases. Through science driven innovation, deep disease understanding, and close collaboration with the liver health community, we work to translate meaningful research into therapies that make a real difference in patients’ lives.

A specialty pharmaceutical company focused on patient impact

Alfasigma is committed to addressing some of today’s most challenging liver diseases. We focus on conditions where symptoms are underrecognized, burdensome, and life altering, and where targeted innovation has the potential to change the course of daily life.

Headquartered in Morristown, New Jersey, at 305 Madison Avenue, our US organization combines world class scientific expertise with a deep appreciation of the patient journey. This perspective informs every stage of our work, from discovery and development to education and community engagement.

Advancing science to restore quality of life

Rare liver diseases affect more than long term health. They can disrupt sleep, energy, emotional well being, and the ability to fully participate in everyday life. Many patients live with persistent symptoms that remain inadequately treated despite existing standards of care.1

At Alfasigma, we believe that addressing symptom burden is essential to comprehensive care. When quality of life improves, outcomes improve. Our mission is to develop therapies that target the biological drivers of disease and its most disruptive symptoms, helping patients regain comfort, function, and confidence.

Patient Centered

People living with rare and serious liver diseases are the reason behind every decision we make. We listen to patient and caregiver experiences and build resources, support, and education that help them navigate their journey with clarity and confidence.

Science Driven

Our research focuses on disease biology with the greatest potential for meaningful clinical impact. Drawing on decades of expertise in bile acid science, we pursue targeted approaches designed to address underlying mechanisms, not just observable symptoms.

Community Committed

We partner with healthcare professionals, advocacy organizations, and the broader liver health community to advance disease education, strengthen awareness of unmet need, and support access to appropriate care.

Rare and serious liver diseases

We are dedicated to building a healthier tomorrow for people living with chronic and serious liver conditions, particularly those with limited or no approved treatment options. Our approach is guided by scientific rigor, urgency, and a clear emphasis on patient experience and quality of life.

Disease Area Focus

Primary Biliary Cholangitis

Primary Biliary Cholangitis is a rare, progressive autoimmune liver disease characterized by gradual bile duct destruction. Many people living with PBC experience significant symptoms that affect daily functioning, long before advanced disease is evident.

One of the most burdensome symptoms is cholestatic pruritus, a persistent internal itch that up to eighty nine percent of patients experience during their disease course. This symptom can disrupt sleep, impair mental health, limit social engagement, and reduce overall quality of life.2

Our long standing commitment to the PBC community has shaped our understanding of both the disease and its profound human impact.

Treatment Focus

Cholestatic pruritus in adults with PBC

Cholestatic pruritus is more than an inconvenience. Patients describe persistent sensations that cannot be relieved by scratching and that interfere with sleep, concentration, relationships, and emotional well being.

Many adults with PBC remain inadequately treated. Addressing this unmet need is central to our therapeutic strategy and reflects our belief that symptom relief is a critical component of patient care.3

Pipeline Direction

Targeting high unmet need liver conditions

Beyond our current IBAT inhibitor program, we continue to explore bile acid biology across a range of liver diseases. Our pipeline strategy focuses on conditions where mechanism based intervention has the potential to alter disease trajectory and improve both clinical outcomes and everyday life for patients.

Experience guided by purpose

References

  1. 1.Levy C, Younossi ZM, Mayo MJ, et al. Assessment of pruritus in primary biliary cholangitis: Quality of life, productivity, and treatment experiences (ITCH‑E study). Liver International. 2025;45(9):e70276. doi:10.1111/liv.70276
  2. 2.Koc OM, Toussaint AK, Untas A, et al. Fatigue in people with primary biliary cholangitis: A position paper from the European Reference Network for Rare Liver Diseases. Lancet Gastroenterol Hepatol. 2026;11(1):71–86.
  3. 3.Levy C, Younossi ZM, Mayo MJ, et al. Assessment of pruritus in primary biliary cholangitis: quality of life, productivity, and treatment experiences (ITCH‑E study). Liver International. 2025;45(9):e70276. doi:10.1111/liv.70276