CDER-Approved NDA for SCENESSE® (afamelanotide 16mg)

On October 8, 2019, the FDA granted approval to Clinuvel Pharmaceuticals Limited’s SCENESSE® (afamelanotide 16mg) implant, to prevent painful skin damage from the sun in adult patients with a history of a rare metabolic disorder—phototoxic reactions from erythropoietic protoporphyria (EPP). Key findings from the FDA’s Multi-Discipline Review:

• While the real-world evidence (RWE) didn’t establish safety or effectiveness of afamelanotide for the FDA, it supported the safety information obtained from the clinical trials.
• A randomized, placebo-controlled study (CUV039) provided evidence of efficacy.
• In the FDA’s review of the safety database, it included all randomized subjects from patients in phase 3 trials (CUV029, CUV030, and CUV039) who received at least one study implant. “Therefore, the safety database presented by the Applicant is sufficient [for the FDA] to characterize the safety of afamelanotide...”

The Applicant also submitted the EMA post-marketing exposure data (CUV-PASS-001/CUV-PASS-002), which included 323 patients treated with afamelanotide under Compassionate Use and Special Access programs. The post-authorisation safety studies (PASS), which include patients with up to 10 years of afamelanotide exposure, did not identify additional safety concerns for afamelanotide. “Thus, [the FDA does] not expect a change in the favorable risk/benefit assessment or increased risk with administration of afamelanotide implant in the post-market setting.”

• Clinuvel’s corporate annual report showed that the safety profile of SCENESSE, based on the PASS, was unchanged compared to the official product information.

The FDA granted this application Priority Review designation and Orphan Drug designation. ‍