Commercial stage Company focused on rare diseases with significant unmet needs. Endocrine franchise includes RECORLEV™, cortisol synthesis inhibitor in Ph 3 studies for endogenous Cushing's syndrome, and veldoreotide, next-gen somatostatin analog for acromegaly. Neuromuscular franchise includes KEVEYIS® approved for primary periodic paralysis. Company sold NA rights for MACRILEN™ to Novo Nordisk for $145 m upfront and tiered royalties in Oct 2018.
900 Northbrook Dr #200
Feasterville Trevose, PA 19053
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