BACKGROUND Company: Mid-sized pharmaceutical company developing compounds to treat rare diseases Therapeutic area: Central Nervous System (CNS) Indication: Dravet Syndrome Phase: 3 CHALLENGE The sponsor was involved in a set of clinical studies focusing on a new therapy to treat Dravet syndrome, a rare and catastrophic form of intractable epilepsy that begins in infancy. The new compound had a history of producing cardiovascular adverse effects including valvulopathy (morbidity affecting the valves of the heart) and pulmonary hypertension. As the studies included both adult and pediatric patients, the sponsor encountered increased regulatory scrutiny. For the pediatric cohort in particular, the FDA insisted on documentation and notification of any instance — no matter how small — o

Join for free to read