Aegerion Therapeutics

Aegerion Pharmaceuticals is a biopharmaceutical company that specializes in the development and commercialization of innovative therapies for patients with rare and orphan diseases. The company's portfolio is anchored by two primary commercial products: Juxtapid (lomitapide), an oral small molecule inhibitor of microsomal triglyceride transfer protein for homozygous familial hypercholesterolemia, and Myalept (metreleptin), a recombinant analog of human leptin used to treat complications of leptin deficiency in patients with generalized lipodystrophy. Following a period of financial restructuring and significant legal settlements related to marketing practices, Aegerion filed for Chapter 11 bankruptcy in 2019 and was subsequently acquired by Amryt Pharma. The company's operations are now integrated into Chiesi Farmaceutici following Chiesi's acquisition of Amryt in 2023. Aegerion remains a notable example of a company focused on high-value, ultra-orphan metabolic indications.