I have sent the following letter to Dr. Gottlieb at the FDA Please do the same (letter or email) so he is innundated by requests for further action by the FDA and not leave this as a settled matter. His email address is: CommissionerFDA@fda.hhs.gov<CommissionerFDA@fda.hhs.gov>
Dear Dr. Gottlieb,
Commissioner, FDA
The FDA has recently acted on a BLA by Prometic Life Sciences (PLI) for Ryplazim (Plasminogen) for an orphan drug designation for congenital plasminogen deficiency and had previously granted Priority Review Status, the Orphan Drug Designation and the Rare Pediatric Disease Designation for RYPLAZIM™ (plasminogen) for the treatment of congenital plasminogen deficiency. The FDA’s review of the BLA raised no issues regarding the clinical data for the accelerated approval. The FDA has, however, identified the need for Prometic to make a number of changes in the Chemistry, Manufacturing and Controls (CMC) section of its BLA. These changes require the implementation and validation of additional analytical assays and “in-process controls” in the manufacturing process of RYPLAZIM™ (plasminogen). While Prometic is expecting to complete said implementation and validation in April 2018, it will be necessary to manufacture additional RYPLAZIM™ (plasminogen) lots to support the implementation and validation of these process changes. No new PDUFA date has been issued. The current PDUFA date was April 14, 2018. No provisional approval was granted.
The Ryplazim has shown 100% efficacy in clinical trials with no safety concerns for a disease that has no other viable treatment. It was immoral to prevent those who suffer from this disease access to t his treatment and to be forced to wait for at least another year without treatment over a minor issue that is readily fixed and easily inspected. The FDA should have provided a provisional approval contingent on the appropriate and readily made CMC changes so the very sick with no other option would not continue to suffer for another year rather than gain access to a treatment that has shown to be 100% efficacious in the clinical trials.
Sir, I would look to you to step in and revise the decision to a provisional approval as soon as possible.
Sincerely