The FDA approved ZEVASKYN (prademagene zamikeracel) gene-corrected epidermal sheets for the treatment of epidermolysis bullosa in April, 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. ZEVASKYN’s approval marks a significant milestone in personalized regenerative medicine, though its high cost may limit widespread use. The FDA also approved two new therapies for two disease states where there are already multiple alternatives, but the newly approved therapies offer dosing regimen advantages. YEZTUGO (lenacapavir) is an alternative brand formulation of the already-approved SUNLENCA but is approved for pre-exposure prophylaxis of HIV-1 infection. YEZTUGO is a twice-yearly injection which is arguably more convenient than APRETUDE which is injected every two months and DESCOVY which is taken orally every day. EKTERLY (sebetralstat) also provides a new and more convenient oral option for the on-demand treatment of acute attacks of hereditary angioedema (HAE) which previously could only be treatedwith injectable therapies.

There are several potential gene therapy FDA approvals approaching 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 current availability of an enzyme-directed therapy for MPS II, a significant unmet need exists for those with severe neurologic manifestations of the disease – a need that could be met with a gene therapy such as RGX-121. Similarly, while symptom-directed therapy options exist for the treatment of WAS, these therapies are only supportive in nature and a hematopoietic stem cell transplant is the only curative treatment currently available. An FDA approval of etuvetidigene autotemcel would provide another potentially curative treatment alternative for WAS. And 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). The intended treatment population includes pediatric SMA patients from two to 18 years of age, a population for which the existing intravenous formulation of ZOLGENSMA is not indicated. If approved, intrathecal ZOLGENSMA would greatly expand the number of SMA patients eligible for gene therapy. Additionally, for SMA, another anticipated FDA approval is for SRK-015 (apitegromab), a muscle-directed therapy intended to be used as an adjunctive therapy to existing SMA therapies, further expanding the available treatment options for the rare disease.

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 July 2025 Pipeline Report (PDF).