Nivalis Therapeutics, Inc. announced that the FDA has granted orphan drug designation to its lead candidate, N91115, for the treatment of patients suffering from cystic fibrosis. The stock surged 15.5% on the news.
We note that orphan drug designation is granted to drugs capable of treating diseases that affect less than 200,000 people in the U.S. The status makes N91115 eligible for seven years of marketing exclusivity in the U.S., following an approval for cystic fibrosis. The designation also makes the company eligible for certain other benefits, including tax credits related to clinical trial expenses, exemption from the FDA-user fee and assistance from the FDA in clinical trial design.
Going ahead, the company plans to seek Fast Track status for N91115 in the U.S. The FDA grants fast track status to therapies that are considered to be capable of addressing unmet medical needs and have the potential to treat serious or life-threatening disease conditions in order to facilitate its development and expedite the review procedure.
Currently, N91115 is being evaluated in a 12-week, double-blind, randomized, placebo-controlled, parallel-group phase II study in adults suffering from cystic fibrosis who are homozygous for the F508del-CFTR mutation and are being treated with Vertex Pharmaceuticals Incorporated’s Orkambi, an approved therapy from cystic fibrosis. Data from the study should mature in the second half of 2016.
We are encouraged by the FDA granting orphan drug status to N91115. According to the Cystic Fibrosis Foundation, approximately 70,000 people suffer from cystic fibrosis worldwide.