RE:RE:FDA Approval for Riplazym could also be Monday morning...Interesting comments from spark Therapeutics on the very recent approval of Luxturna by the FDA a month before the PDFUA date... particularly the part where the researcher says he’s never heard of any drug being approved before the date. Kind of throws water on the “Konday” theory considering the set date is two months away.... they also got the 13th voucher ever handed out.
The approval comes almost one month ahead of the scheduled Prescription Drug User Fee Act (PDUFA) date of January 12, 2018. “I’ve never heard of any drug being approved before it’s PDUFA date,” said Stephen R. Russell, M.D., of the University of Iowa, one of the lead researchers on the study in an exclusive interview with Rare Disease Report in November, “but I guess it could happen.” “The approval of Luxturna further opens the door to the potential of gene therapies,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Patients with biallelic RPE65 mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed.” It is suggested by the FDA that Luxturna be administered only to patients who have viable retinal cells as determined by the treating physicians, and treatment with the drug must be done separately in each eye on separate days, with at least 6 days between surgical procedures. The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear. Spark Therapeutics is receiving a Rare Pediatric Disease Priority Review Voucher, the 13th distributed by the FDA since the initiation of the program.