Fera Pharmaceuticals receives Orphan Drug Designation from FDA for sickle cell treatment
pharmafile | March 2, 2022 | News story | Research and Development |
The FDA has granted an Orphan Drug Designation for naproxcinod for the treatment of sickle cell disease, which affects an estimated 100,000 Americans.
Naproxcinod is a nitric oxide (NO)-donating naproxen. This combines the cyclooxygenase (COX) inhibitory activity of naproxen with that of nitric oxide developed by Nicox, and exlusively licensed to Fera in the US. Nicox has tested naproxcinod in over 2,700 patients in osteoarthritis, generating a significant package of clinical safety data which is available to support Fera’s development of naproxcinod, and an NDA submission for Ssickle cell disease.
Sickle cell disease is an inherited disorder which affects red blood cells, with a faulty version of haemoglobin causing normally oval-shaped red blood cells to become distorted. This can lead to symptoms such as pain, frequent infections, and anaemia. The rupture of these cells in the bloodstream can lead to inflammation, a reduction in NO, and a subsequent endothelial cell wall thickening, as well as platelet activation.
Michele Garufi, CEO and Chairman of Nicox, remarked on this development: “We congratulate Fera on achieving Orphan Drug Designation for naproxcinod, which is a very important step in being able to develop this molecule as a potential treatment for sickle cell disease. Fera has already carried out pre-clinical development work on naproxcinod in models of sickle cell disease, and the extensive clinical package already developed by Nicox positions the molecule – the first that was taken into clinical development by our Company – for an accelerated development.”
Lina Adams
