There is a rise in the number and spread of rare disorders. Due to their growing presence and risk, various pharmaceutical firms are increasingly working towards the discovery and development of treatments.  

One such rare-disease treating drug is soon expected to hit the market as the U.S. Food and Drug Administration, an federal government body responsible for food and drugs inspection and consumer protection, reportedly announced that it has approved Scenesse (afamelanotide) in order to increase the pain-free light exposure among adults that have been diagnosed with erythropoietic protoporphyria (EPP), a rare form of porphyria that causes phototoxic reactions that damage the skin of the patients.

Erythropoietic protoporphyria is a very rare disorder caused due to mutations that leads to impaired activity of the ferrochelatase, which is an enzyme that is involved in heme production. Heme is a vital component in the hemoglobin, molecule that carries oxygen in the red blood cells.

Scenesse has already obtained a marketing approval earlier in Europe; the drug is delivered through an under-the-skin implant for treating the rare genetic condition.

Scenesse’s efficacy was confirmed in two parallel clinical group trials in patients suffering with erythropoietic protoporphyria, who were given Scenesse or placebo form of an implant that administered either subcutaneously after every two months.

There were total of 93 subjects enrolled in the first clinical trial of which 48 received Scenesse and were further observed for 180 days. The second clinical study registered 74 patients, of which 38 received Scenesse and were further observed for 270 days.

Director of FDA’s Center for Drug Evaluation and Research Office of Drug Evaluation III, Julie Beitz, M.D. stated that for patients diagnosed with the rare disorder, erythropoietic protoporphyria, exposure to light may be highly painful. Before today’s approval, the pharmaceutical sector did not have any FDA-approved therapies to help patients with erythropoietic protoporphyria to increase their exposure to light.

Beitz added that Scenesse’s approval is an example of the ongoing commitment of FDA to impel industry innovation of treatment options for rare diseases and collaborate with drug developers in order to develop new, promising therapies and make them available for patient treatment as efficiently and safely as possible.  

Priority Review designation was granted to the application by the FDA. Furthermore, Orphan Drug Designation (ODD) was also received by Scenesse, whose approval was granted by the FDA to Clinuvel.

Source credit: https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-increase-pain-free-light-exposure-patients-rare-disorder