October Regulatory Outlook

Only one investigational drug has an October 2019 PDUFA Date, Clinuvel Pharmaceutical’s afamelanotide (Scenesse). The FDA has given afamelanotide an Orphan Drug Priority Designation and its PDUFA Date is October 6, 2019. Afamelanotide is being developed as a treatment for erythropoietic protoporphyria. The drug is a melanocyte-stimulating hormone delivered via subcutaneous implant.  Afamelanotide was approved in the EU in 2015. The FDA delayed the PDUFA date of afamelanotide for three months to allow more time for a full review of the NDA. In a 9-month, 74 patient, Phase III trial, patients treated with afamelanotide tolerated six hours of direct exposure to sunlight without pain compared to 0.8 hours with placebo in European patients with erythropoietic protoporphyria. In a 6-month, 94 patient, Phase III trial, patients treated with afamelanotide tolerated 69.4 hours of direct exposure to sunlight without pain compared to 40.8 hours with placebo in U.S. patients with erythropoietic protoporphyria. (N Engl J Med. 2015 Jul 2;373(1):48-59). There are no other late stage investigational drugs in our knowledgebase indicated for erythropoietic protoporphyria.
 
There is one investigational drug with a November 2, 2019 PDUFA Date, RedHill Biopharma’s combination of amoxicillin, omeprazole, rifabutin (Talcia). It is an oral combination product that has Fast Track and Qualified Infectious Disease designations for the eradication of H. pylori. The results of the Phase III ERADICATE Hp2 study have not been published, but Redhill announced that in the 455 patient trial, the combination of amoxicillin, omeprazole and rifabutin demonstrated an 84% eradication of H. pylori compared to 58% eradication with amoxicillin and omeprazole in dyspepsia patients with confirmed H. pylori infection.