We specialize in bringing orphan and ultra-orphan drugs to market in the UK and EU. Our team ensures that therapies meet all regulatory and safety standards, supporting approvals, post-marketing surveillance, and access frameworks.
Orphan Drug Commercialization
Our commercialization capabilities include:
Regulatory submissions and post-approval compliance (MHRA, EMA)
Market introduction strategies tailored to niche indications
Cross-border launch planning and lifecycle management
Commercial supply readiness and medical education at launch
With a focus on unmet clinical needs, we ensure each product launch is built around real patient impact.
Orphan Drug Commercialization at Aegerion Pharmaceuticals UK
At Aegerion Pharmaceuticals UK, our mission is to transform the lives of patients living with rare and underserved diseases. We specialize in the development and commercialization of orphan drugs—high-impact therapies designed for conditions that affect small patient populations, yet carry a significant burden on quality of life.
Our Commitment to Rare Disease Communities
Orphan drug commercialization requires more than innovation—it demands a deep understanding of patient needs, healthcare system dynamics, and the nuances of rare disease diagnosis and treatment. At Aegerion, we are dedicated to delivering meaningful therapies where few or none exist, with a commitment to accessibility, education, and long-term patient support.
Our Approach to Commercialization
1. Patient-Centered Strategy
We begin with the patient. Our commercialization model is designed around early engagement with patient communities, clinicians, and advocacy groups to ensure that our therapies are responsive to real-world needs.
Focused outreach to specialists and treatment centers of excellence
Collaboration with advocacy organizations to raise disease awareness
Tailored support programs to guide patients throughout their treatment journey
2. Navigating Complex Markets
Rare diseases present unique market challenges—from diagnosis delays to access barriers. Aegerion addresses these with a high-touch, data-driven approach.
Specialist field teams work closely with providers to support disease education and therapy adoption
Health economics and outcomes research (HEOR) teams generate robust value evidence to support reimbursement
Global pricing and market access strategies that reflect both the innovation and value of our therapies
3. Accelerated Access Through Regulatory Excellence
With experience navigating regulatory frameworks in the UK, EU, and globally, Aegerion leverages orphan drug designations and special pathways to bring therapies to market faster.
Engagement with regulatory bodies from early development through launch
Utilization of incentives such as market exclusivity and fee reductions
Adaptive development models aligned with patient and regulator needs
Beyond Launch: Sustaining Access and Value
Post-Market Commitment
Our responsibility to patients continues well beyond commercialization.
Real-world evidence generation to ensure ongoing safety and efficacy
Pharmacovigilance excellence to meet the highest standards of patient safety
Lifecycle management to explore new indications and treatment opportunities
Global Reach, Local Expertise
Aegerion Pharmaceuticals UK operates as part of a global network with deep local insight. This ensures that we can tailor our commercialization efforts to specific healthcare environments while maintaining the integrity and impact of our science.
Impact in Action
We’ve successfully brought therapies such as lomitapide to market, supporting patients with rare lipid disorders through:
Personalised patient engagement programs
Education for healthcare professionals on early diagnosis
A dedicated access and reimbursement support infrastructure
Partnering for the Future of Rare Disease Care
At Aegerion Pharmaceuticals UK, we’re more than a company—we’re a partner in rare disease care. Through scientific innovation, collaborative engagement, and unwavering commitment, we aim to bring hope to those who have long been overlooked by traditional pharmaceutical models.
If you are a healthcare provider, policymaker, or patient group interested in learning more about our work or partnering with us, please get in touch with our team.
Patient Access & Support Programs
Removing Barriers Between Patients and Life-Changing Treatments
For patients with rare diseases, access can be the single most critical component of care. Aegerion provides a range of programs and services designed to reduce delays, improve adherence, and help patients and families navigate the healthcare system.
