Orphan drug designation entitles Atlanta-based Tikvah to seven years of market exclusivity for phenylbutyrate for the treatment of patients with spinal muscular atrophy (SMA). Additional incentives for orphan drug development include tax credits related to certain development expenses, an exemption from the FDA user fee and FDA assistance in clinical trial design.
SMA, the leading genetic killer of children under the age of two, is a group of inherited and often fatal diseases that destroy the nerves controlling voluntary muscle movement, which affects crawling, walking, head and neck control, and even swallowing. There currently are no approved therapies for the treatment of SMA.