Osiris and FDA Reach Agreement on Submission of First Marketing Application for a Stem Cell Product
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Osiris Therapeutics, Inc. has held a successful pre-Biologics License Application (pre-BLA) meeting for Prochymal used in the treatment of graft-versus-host disease (GvHD). A BLA is a comprehensive regulatory submission prepared by a biologic drug's sponsor to obtain full marketing approval from the Food and Drug Administration (FDA).
As an outcome of the meeting between Osiris and FDA, the parties finalized the content and timing of what is expected to be the first BLA submission for a stem cell product.
"Agreement on the timing and content of the BLA marks an important milestone in our quest to make Prochymal the world's first fully approved stem cell therapy," said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris.
"We thank FDA for their significant efforts over the past several years as we have forged a highly productive partnership in this new and exciting area of medicine. Having proactively addressed issues critical to a successful review, we have created a clear understanding of the regulatory path to market approval."
A pre-BLA meeting is a mechanism by which FDA and the sponsor can discuss the application in advance of submission to avoid potential pitfalls in review process.
In the Prochymal pre-BLA meeting there were three main areas of discussion. The first was the type and extent of safety and efficacy data that would be included. The second was manufacturing and quality data that would comprise the submission. The third was the process and format of the submission itself. Prior to the meeting, Osiris submitted a comprehensive outline of the proposed content and structure of the BLA.
Key results of the pre-BLA meeting are below:
• FDA agreed with the proposed content and structure of the BLA for Prochymal for the treatment of GvHD.
The primary endpoint of the Phase III trial that will be reviewed by FDA for approval was confirmed to be GvHD Complete Response (CR). A CR is complete remission of the disease.
• FDA indicated that the statistical analysis plan was appropriate.
The overall safety database (Integrated Summary of Safety) of Prochymal is sufficient in scope for the indication. No additional clinical data beyond what was presented is anticipated to be necessary for the BLA.
• Agreement was reached on BLA requirements for Chemistry, Manufacturing and Controls (CMC) data.
The FDA concurred with the proposed product stability and process validation plan for submission and provided guidance on how to present the data for ease of review.
• Agreement was reached on the timing and content of the BLA in rolling submission format. The rolling submission is an FDA provision available to drug candidates that have received Fast Track designation, which allows for completed sections of a BLA to be submitted on an ongoing basis. It can facilitate the process by allowing FDA to complete review of sections as soon as they are available. It is anticipated that the nonclinical sections, such as toxicology, will be the first submitted for review.
The BLA will be in the electronic Common Technical Document (eCTD) format. The eCTD format facilitates the review of the BLA and allows parallel submission of the dossier in other territories.
At the time of submission, Osiris will also submit the request for Priority Review of the Prochymal BLA. Investigational drugs with Fast Track designation are eligible for consideration for Priority Review, which provides for an accelerated six month application review by FDA.
As an outcome of the meeting between Osiris and FDA, the parties finalized the content and timing of what is expected to be the first BLA submission for a stem cell product.
"Agreement on the timing and content of the BLA marks an important milestone in our quest to make Prochymal the world's first fully approved stem cell therapy," said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris.
"We thank FDA for their significant efforts over the past several years as we have forged a highly productive partnership in this new and exciting area of medicine. Having proactively addressed issues critical to a successful review, we have created a clear understanding of the regulatory path to market approval."
A pre-BLA meeting is a mechanism by which FDA and the sponsor can discuss the application in advance of submission to avoid potential pitfalls in review process.
In the Prochymal pre-BLA meeting there were three main areas of discussion. The first was the type and extent of safety and efficacy data that would be included. The second was manufacturing and quality data that would comprise the submission. The third was the process and format of the submission itself. Prior to the meeting, Osiris submitted a comprehensive outline of the proposed content and structure of the BLA.
Key results of the pre-BLA meeting are below:
• FDA agreed with the proposed content and structure of the BLA for Prochymal for the treatment of GvHD.
The primary endpoint of the Phase III trial that will be reviewed by FDA for approval was confirmed to be GvHD Complete Response (CR). A CR is complete remission of the disease.
• FDA indicated that the statistical analysis plan was appropriate.
The overall safety database (Integrated Summary of Safety) of Prochymal is sufficient in scope for the indication. No additional clinical data beyond what was presented is anticipated to be necessary for the BLA.
• Agreement was reached on BLA requirements for Chemistry, Manufacturing and Controls (CMC) data.
The FDA concurred with the proposed product stability and process validation plan for submission and provided guidance on how to present the data for ease of review.
• Agreement was reached on the timing and content of the BLA in rolling submission format. The rolling submission is an FDA provision available to drug candidates that have received Fast Track designation, which allows for completed sections of a BLA to be submitted on an ongoing basis. It can facilitate the process by allowing FDA to complete review of sections as soon as they are available. It is anticipated that the nonclinical sections, such as toxicology, will be the first submitted for review.
The BLA will be in the electronic Common Technical Document (eCTD) format. The eCTD format facilitates the review of the BLA and allows parallel submission of the dossier in other territories.
At the time of submission, Osiris will also submit the request for Priority Review of the Prochymal BLA. Investigational drugs with Fast Track designation are eligible for consideration for Priority Review, which provides for an accelerated six month application review by FDA.
