10 Years of commitment to treating rare diseases
10 June 2025
Pharmablue
Rare diseases: At the core of our mission
At PharmaBlue, we’ve spent the past decade supporting biotech innovators in bringing their rare disease therapies to patients in France—quickly, compliantly, and with care. As a licensed Exploitant, we specialize in managing Early Access Programs (EAPs) and post-authorization market entry, helping our partners navigate the complex French regulatory landscape.Our work spans 24 rare diseases across 10 therapeutic areas, including:
- Neurology
- Oncology
- Immunology
- Pneumology
- Cardiology
- Endocrinology
- Infectiology
- Rheumatology
- Hematology
- Dermatology
Why rare diseases matter
In France and across Europe, patients with rare diseases often rely on early access mechanisms to receive innovative therapies before full market authorization.*- 95% of these conditions still lack an approved treatment**
- 72% of rare diseases are genetic in origin, often manifesting in childhood.*
- 1 in 20 people will live with a rare disease at some point in their lives.*
- Many patients wait 5 to 7 years for an accurate diagnosis.
As we celebrate 10 years of impact, we remain committed to expanding access and improving outcomes for patients with rare diseases.
Did you like this article? Share on social networks:
