Aegerion, now part of Amryt Pharma Plc, is a commercial-stage pharmaceutical company dedicated to developing and delivering innovative treatments for rare and orphan diseases. These are conditions that affect a small percentage of the population, often lacking effective treatments. Aegerion focuses on providing hope and improving the quality of life for patients suffering from these challenging conditions.

The company serves a specialized client base, primarily healthcare professionals and patients dealing with rare diseases. Two key conditions they address are Epidermolysis Bullosa (EB), a painful and incurable genetic skin disorder, and Homozygous Familial Hypercholesterolemia (HoFH), a life-threatening condition characterized by extremely high cholesterol levels.

Aegerion operates in the niche market of rare and orphan diseases, which, despite its small size, has significant unmet medical needs. This market is characterized by high demand for specialized treatments and often involves complex regulatory environments.

The business model of Aegerion revolves around research and development (R&D) of new drugs, obtaining regulatory approvals, and then commercializing these treatments. They make money by selling these specialized medications to healthcare providers and directly to patients. Additionally, they may receive funding and grants aimed at supporting rare disease research.

Aegerion's revenue streams are primarily driven by the sales of their approved drugs. They also invest in ongoing R&D to expand their product pipeline, ensuring a steady flow of innovative treatments to address other rare diseases.

In summary, Aegerion is a key player in the rare disease pharmaceutical sector, focusing on developing life-changing treatments for conditions that have limited or no current therapies. Their commitment to this underserved market positions them as a vital resource for healthcare professionals and patients alike.

Keywords: Rare diseases, orphan diseases, pharmaceuticals, Epidermolysis Bullosa, Homozygous Familial Hypercholesterolemia, innovative treatments, healthcare professionals, research and development, niche market, patient care.