Pipeline Report

New AcariaHealth Pipeline Report - October 2024

10/31/2024

Three FDA approvals occurred during this past quarter that bear mentioning. The first was for TECELRA (afamitresgene autoleucel), the first T-cell receptor immunotherapy that targets synovial sarcoma – a rare soft tissue sarcoma – becoming only the second cell therapy to be approved for a solid cancer. The next two are AQNEURSA (levacetylleucine) and MIPLYFFA (arimoclomol), both FDA-approved for the treatment of neurological manifestations of Niemann-Pick disease type C, an ultra-rare genetic disorder with no previously approved therapies. The joint approvals are a reflection of the FDA’s latest efforts to address the extensive unmet need in the rare disease space.

On the horizon are three more potential FDA approvals for cell and gene therapies. AUTO1 (obecabtagene autoleucel) is T-cell therapy that aims to provide better tolerability than other available T-cell alternatives for acute lymphoblastic anemia, representing an important advance in the treatment of this hematologic cancer. Additionally, an FDA review date has been secured for gene therapy UPSTAZA (eladocagene exuparvovec) which would be the first FDA-approved therapy for the treatment of AADC deficiency, an ultra-rare disorder which affects the nervous system and has wide-ranging effects on motor and cognitive function. And finally, NT-501 (revakinagene taroretcel) is now under FDA review for the treatment of macular telangiectasia type 2, a genetic condition resulting in loss of vision. By year’s end we should know whether these three progressive therapies will become available to provide their promised therapeutic benefits for the intended populations.

Alan R. Smith, MD

Vice President, Medical Director

Our quarterly publication is developed by our Clinical Pharmacy Drug Information team to provide additional drug pipeline information and insights to help health care leaders prepare for shifts in prescription drug management. Learn more by accessing the complete AcariaHealth October 2024 Pipeline Report (PDF).