Sarepta Therapeutics, Inc. announced that the rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the U.S. Food & Drug Administration. In keeping with FDA's pursuit of science-driven efficiency, the platform technology designation program is a critical tool that will help support rare disease therapies by streamlining drug development, manufacturing, and review processes for drug product applications by incorporating data from designated technologies. To be eligible for this designation, the technology must be a well-understanding and reproducible technology - such as a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of any such technologies - and can support the development of multiple drugs or biologics.
A platform is also eligible for consideration if it is incorporated in an approved drug and preliminary evidence demonstrates that the technology has the potential to be incorporated in or utilized by more than one drug without an adverse effect on quality, manufacturing, or safety, including using the same manufacturing process. Once granted, the designation enables sponsors to leverage prior data from the platform to support investigational new drug applications (INDs), new drug applications (NDAs) or biologic license applications (BLAs). SRP-9003 (bidridistrogene xebOParvovec) is an investigational gene therapy designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat neuromuscular diseases.
SRP-9003 is intended to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to robustly express in the heart which is critically important for patients with limb-girdle muscle dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications the company use to assess particular safety or efficacy parameters may yield different statistical results; success in clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; may not be able to comply with all FDA requests in a timely manner or at all; products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; the possible impact of regulatory decisions by, and any halts imposed by, regulatory agencies on business; and those risks identified under the heading "Risk Factors" in most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which are encouraged to review.
Any of the foregoing risks could materially and adversely affect the Company's business, results of operations, and the Company's operations, and are expected to review.
