Pipeline Report

The fourth quarter of 2025 saw a wave of FDA approvals across specialty and rare disease categories, with a robust pipeline of high-cost therapies poised to reshape treatment paradigms in 2026. FORZYNITY (elamipretide) was approved for the ultra-rare disease Barth syndrome. This approval introduces a first-in-class treatment that addresses a critical unmet need, albeit with a significant price tag and a very small target population. Meanwhile, PAPZIMEOS (zopapogene imadenovec-drba) was approved for another rare condition, recurrent respiratory
papillomatosis. This one-time treatment may reduce the need for repeatedsurgeries, potentially offsetting long-term medical costs.

Several potential gene therapy FDA approvals are expected in the upcoming quarters, including RGX-121 (clemidsogene lanparvovec) for mucopolysaccharidosis type II (MPS II) and etuvetidigene autotemcel for Wiskott-Aldrich syndrome (WAS). Despite the availability of enzyme replacement therapy for MPS II, a significant unmet need remains for patients with severe neurologic manifestations—an unmet need that gene therapy such as RGX-121 could address. Similarly, while symptom-directed therapies exist for WAS, these treatments are only supportive, with hematopoietic stem cell transplant currently being the only curative option. FDA approval of etuvetidigene autotemcel would provide a potentially curative alternative for WAS.

Finally, in the area of gene therapies, the intrathecal formulation of ZOLGENSMA (onasemnogene abeparvovec-xioi) is expected to be approved in late 2025 or early 2026 for the treatment of spinal muscular atrophy (SMA). This formulation targets pediatric SMA patients aged two to eighteen, a population for which the existing intravenous formulation of ZOLGENSMA is not indicated. If approved, intrathecal ZOLGENSMA would significantly expand the number of SMA patients eligible for gene therapy.

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 2025 Pipeline Report (PDF).