Amryt Shares Shoot Higher As FDA Grants Orphan Drug Status To Carcinoid Tumor Candidate

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Amryt’s (NASDAQ: AMYT) lead drug Mycapssa for the treatment of carcinoid syndrome.

Carcinoid syndrome (CS) is the most common functional syndrome associated with neuroendocrine tumours (NETs).

Mycapssa (oral octreotide) is approved by the FDA for long-term maintenance treatment in acromegaly patients who have responded to and tolerated injectable treatment with octreotide or lanreotide

Dr Joe Wiley, CEO of Amryt Pharma, said, "Today's news is another significant milestone achieved in our plan to develop Mycapssa® for patients affected by carcinoid syndrome associated with NET and we are excited at the potential for Mycapssa® and our TPE® technology to deliver an oral therapy option to NET patients in need.”

The company Plans to initiate Phase 3 study in patients with carcinoid syndrome early in 2023.  

FDA grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. It also provides benefits of seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

Price Action : Amryt Shares are trading around 8 percent higher at $8 on Thursday’s pre-market session.