- The U.S. Food and Drug Administration (FDA) granted orphan drug designation to Amryt's ( NASDAQ: AMYT ) Mycapssa to treat carcinoid syndrome (CS).
- CS occurs when a rare cancerous tumor called carcinoid tumor releases certain chemicals in the blood, causing several symptoms. According to the company CS is the most common functional syndrome associated with neuroendocrine tumours (NETs).
- Mycapssa (oral octreotide) is approved by the FDA as a long-term maintenance therapy for certain patients with acromegaly, a hormonal disorder which occurs when the pituitary gland produces too much growth hormone during adulthood.
- Amryt noted that its TPE platform enables the oral delivery of the octreotide molecule which is otherwise delivered as an injectable.
- The FDA grants orphan drug status to therapies that treat or prevent rare diseases that affect fewer than 200K people in the U.S. The designation provides certain incentives, including seven years of market exclusivity, if approved.
- AMYT +11.19% to $8.25 premarket July 14.
For further details see:
Amryt stock rises 11% on FDA orphan drug status for Mycapssa for rare tumor condition