Rare Diseases

Led by Alexion, AstraZeneca’s Rare Disease group, we are dedicated to transforming the lives of people affected by rare and orphan conditions. By listening to the patient voice and collaborating with specialised researchers and caregivers, we accelerate the development of highly targeted therapies to improve health outcomes across the Swiss rare disease community.

Rare diseases by the numbers

~500,000 people

across Switzerland live with a rare disease, roughly 75% of whom are children¹’²

7,000+

distinct rare conditions are recognised worldwide, presenting complex diagnostic challenges1,2

~95%

of known conditions still lack an approved therapeutic option1

Our Philosophy

Every day, we follow the science to transform the lives of people living with rare diseases. Our work in Switzerland is guided by five core pillars:

Pioneers in Science

Advancing the next wave of rare disease research to discover transformative therapies for high-unmet-need conditions.

Patient-Focused Innovators

Embedding patient and caregiver insights into every stage of development to ensure therapies reflect lived experiences.

Advancing our Global Footprint

Expanding access to clinical trials, specialised knowledge, and innovative treatments across Switzerland and worldwide.

Championing Health Equity

Partnering with healthcare providers and patient organisations to accelerate diagnosis and ensure fair access to care.

Purpose-Driven People

Uniting specialised teams dedicated to delivering life-changing outcomes for patients, families, and health systems.

Rare diseases supported in Switzerland

Atypical Haemolytic Uraemic Syndrome (aHUS)

Generalised Myasthenia Gravis (gMG)

Hypophosphatasia (HPP)

Lysosomal Acid Lipase Deficiency (LAL-D)

Neurofibromatosis Type 1 (NF1)

Neuromyelitis Optica Spectrum Disorder (NMOSD)

Paroxysmal Nocturnal Haemoglobinuria (PNH)

Find more information here

Our pipeline for Rare Diseases

Our pipeline is dedicated to pioneering for conditions with significant unmet medical need. Building on a legacy of translating complex disease biology into innovative therapies, we advance next-generation research across novel therapeutic modalities.

Through clinical trial collaborations and a science-led approach, we focus on delivering potential first-in-class and best-in-class treatments to transform care for rare disease communities in Switzerland and worldwide.

References

1. Rare Disease Action Forum. https://rda-forum.org/rare-diseases/ (accessed: 09/2026).

2. Schweizer Register für seltene Krankheiten (SRSK). https://www.raredisease.ch/ (accessed: 09/2026).

Veeva ID: CH-13774
Date of preparation: October 2026