- Nivalis Therapeutics last week announced its cystic fibrosis (CF) drug, N9115, had been granted an orphan drug designation by the FDA, causing its shares to spike on Friday.
- Nivalis raised $88 million in its IPO back in June of last year.
- Orphan drug designations grant drugmakers seven-year marketing exclusivity provided the target disease affects less than 200,00 people in the U.S.
Treatment options for some CF patients improved dramatically with the approval of Vertex’s Kalydeco and its follow-up drug, Orkambi. However, Jon Congleton, president and CEO of Nivalis, has cited the unmet medical need in CF, which affects 70,000 patients worldwide.
These two drugs have been shown in clinical trials to significantly improve lung function in CF patients by up to 4%, while also decreasing pulmonary exacerbations by up to 39%. However, the drugs are expensive, although they work well in patients with the target genetic mutations.
Nivalis is now developing N9115 and testing it in a Phase 2 trial in conjunction with Orkambi. It hopes to gain fast track status for N9115 in addition to the orphan drug designation.