Janssen Submits Light Chain Amyloidosis Treatment to FDA

Halozyme Therapeutics announced its collaborator Janssen Biotech has submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) seeking approval of DARZALEX FASPRO™ (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab utilizing Halozyme's ENHANZE® technology, for the treatment of patients with light chain (AL) amyloidosis, a rare and potentially fatal disease for which there are no currently approved therapies. The sBLA is supported by positive results from the Phase 3 ANDROMEDA study. Janssen's ANDROMEDA study evaluated subcutaneous daratumumab in combination with bortezomib, cyclophosphamide, and dexamethasone (D-VCd) compared to VCd alone and met its primary endpoint of overall hematologic complete response rate.

"We are excited that DARZALEX FASPROTM utilizing our ENHANZE® technology has the potential to be approved in a new indication," said Dr. Helen Torley, president and chief executive officer. "Patients diagnosed with AL amyloidosis currently have no approved therapeutic options and we are therefore pleased that a new treatment for the disease may soon be available."

The sBLA is being reviewed under the FDA Real-Time Oncology Review (RTOR) program, which allows data for certain applications to be reviewed before the applicant formally submits the complete application. The RTOR program aims to explore a more efficient review process to help ensure treatments are available as soon as possible for patients. Selection into the RTOR program does not guarantee or influence approvability of the supplemental application.

The submission is also being reviewed under Project Orbis, an initiative of the FDA Oncology Center of Excellence, which provides a framework for concurrent submission and review of oncology medicine applications among international regulatory agencies.