Pipeline Report

To wrap up 2024, the FDA approved several new treatment options for patients with rare diseases, offering hope to many who previously had no approved therapies available. KEBILIDI (eladocagene exuparvovec-tneq) gene therapy for aromatic L-amino acid decarboxylase deficiency, once launched, promises dramatic, disease-altering effect. Meanwhile TRYNGOLZA (olezarsen) provides an adjunctive option to significantly lower triglyceride levels beyond an extremely restrictive diet which was previously the only treatment modality available for patients with familial chylomicronemia syndrome, and CRENESSITY (crinecerfont) delivers on the prospect of being able to decrease usage of high doses of glucocorticoids in patients with congenital adrenal hyperplasia, allowing for fewer steroid-related long-term adverse effects.

The FDA approval of ZEPBOUND (tirzepatide) for obstructive sleep apnea highlights the emphasis on health on a larger scale as well. This approval of a medication that previously was only approved for chronic weight management, now for a weight-related medical condition, reflects the turning tide of opinion on the relationship between weight and comorbid conditions, and the trend towards more holistic treatment approaches.

PZ-CEL (prademagene zamikeracel) gene-corrected epidermal sheets are back under FDA review for the treatment of epidermolysis bullosa, targeting the population of patients with larger disease-affected areas who may not be candidates for therapy with the existing topical gene therapy alternative, VYJUVEK. And finally, the FDA continues its review of NT-501 (revakinagene taroretcel) cell therapy for the treatment of macular telangiectasia type 2, a genetic condition resulting in loss of vision. We expect approval of these potentially significant new therapies within the first few months of the new year.

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 January 2025 Pipeline Report (PDF).