Company: Amylin Pharmaceuticals
Liaison(s): Ted Slocomb
Amylin Pharmaceuticals is a world leader in providing therapies for diabetes, obesity, and related metabolic disorders. Founded in 1987 and based in San Diego, Amylin currently has three drugs on the market. Symlin® is a drug for patients with Type 1 and Type 2 diabetes while Byetta® is indicated to treat Type 2 diabetes. The recently approved drug Bydureon® is also indicated for the treatment of Type 2 diabetes, and has the novel advantage of only requiring once a week dosing. Amylin is in the process of launching a new drug for the treatment of the ultra-rare disorder lipodystrophy. This disease is characterized by a lack of body fat and has severe metabolic consequences; one of the primary reasons for this is the loss of the neurohormone leptin which is produced by adipose cells. The drug Metreleptin is an analog of leptin and has been shown to significantly improve health outcomes relating to diabetes and hypertriglyceridemia in patients suffering from lipodystrophy. Due to the rarity of the disease and difficulty of diagnosis, Amylin is investigating strategies to discover undiagnosed patients who may benefit from Metreleptin. The role of the Amylin TMP team was to assist in the launch of Metreleptin by investigating the genetic testing landscape and the use of genetic testing for diagnosis of lipodystrophy. Pursuant to this goal, the team performed exhaustive case studies into other rare diseases as a way of comparing current strategies, developed a database of labs performing genetic testing for lipodystrophy genes, and carried out extensive research into newborn screening, genetic counseling, genetic testing reimbursement, and regulatory issues. Finally, the Amylin TMP team provided a prioritized set of strategic alternatives for integrating genetic testing into the overall Metreleptin launch plan.