Vyjuvek Label Update Broadens Access for Newborns and At-Home Use
On Sept. 15, 2025, the US Food and Drug Administration (FDA) approved label updates for Vyjuvek® (beremagene geperpavec-svdt), Krystal Biotech’s redoseable, topical gene therapy for dystrophic epidermolysis bullosa (DEB). It uses a herpes simplex virus type 1 vector, delivering functional COL7A1 genes to promote collagen VII production, addressing the root cause of DEB. The update expands eligibility to include patients from birth, previously age-restricted to older than six months of age, and allows self-application by patients or caregivers, eliminating the requirement for healthcare professional administration. The expanded label also introduces greater flexibility in wound care, allowing dressing removal during the next scheduled change rather than waiting 24 hours. These changes are supported by real-world data and an open-label extension study, which confirms Vyjuvek’s long-term safety and efficacy across all age groups. The recommended application is once weekly to DEB wounds. Patients and caregivers are advised to follow strict hygiene protocols during dressing changes. Here is the updated prescribing information.
First Intranasal Loop Diuretic, Enbumyst, Approved
The FDA approved Enbumyst™ (bumetanide nasal spray) from Corstasis Therapeutics on Sept. 12, 2025, for the treatment of edema associated with congestive heart failure, liver disease and kidney disease, including nephrotic syndrome, in adults. Enbumyst is the first intranasal loop diuretic approved in the US. Approval is based on clinical studies demonstrating rapid absorption, predictable diuretic response and comparable efficacy to intravenous (IV) bumetanide, with fewer treatment-related adverse events than oral formulations. The recommended dose is one 0.5mg spray daily, with dosing adjusted based on clinical response up to 2mg per day. It is not intended for chronic use and patients should be transitioned to oral diuretics as soon as practical, with approximate substitution ratios of 1:40 for oral furosemide and 1:20 for intravenous furosemide. Here is the prescribing information.
More Denosumab Biosimilars Approved
On Sept. 17, 2025, the FDA approved Biocon Biologics’ biosimilars Bosaya™ (denosumab-kyqq 60mg/mL injection) and Aukelso™ (denosumab-kyqq 120mg/1.7mL) as alternatives to Amgen’s Prolia® (denosumab) and Xgeva® (denosumab), respectively. Bosaya is indicated for the treatment of osteoporosis for those at high risk for fracture in certain populations like men, post-menopausal women and those taking glucocorticoids or certain drugs for prostate and breast cancer. It is administered by a healthcare provider as a 60mg subcutaneous (SC) injection once every six months. A boxed warning and a risk evaluation and mitigation strategy (REMS) caution that using Bosaya may increase the risk of severe hypocalcemia (low levels of calcium in the blood) in patients with chronic kidney disease (CKD). Aukelso is indicated to prevent skeletal-related events for some patients who have multiple myeloma, bone metastases from solid tumors, giant cell tumors of the bone or hypercalcemia of malignancy. After loading doses, it is administered SC at 120mg once every month by a healthcare provider. Bosaya and Aukelso were granted interchangeability, effective after exclusivity for Jubbonti® (denosumab-bbdz – Sandoz) and Wyost® (denosumab-bbdz – Sandoz) expires on Oct. 29, 2025. Jubbonti, the first biosimilar to Prolia, and Wyost, the first biosimilar to Xgeva, launched on May 31, 2025. Other biosimilars are expected to launch this year. Bosaya and Aukelso prescribing information can be found here.
Opzelura Approved for Pediatric Eczema Incyte’s Opzelura® (ruxolitinib) cream 1.5% approval was expanded on Sept. 18, 2025, to include children ages two years to 11 years for the treatment of mild to moderate atopic dermatitis (AD). Previously approved for AD patients 12 years and older, Opzelura is now the first topical Janus kinase (JAK) inhibitor approved for pediatric AD. It is also indicated for nonsegmental vitiligo in patients over 12 years of age. Use is indicated for short-term and non-continuous chronic treatment in non-immunocompromised children and adults whose disease is inadequately controlled with topical prescription therapies or when those therapies are not advisable. According to the National Eczema Association, approximately 9.6 million children in the US are affected by eczema and according to Incyte, 2-3 million of these cases are aged two years through 11 years. Opzelura is a steroid-free cream with a recommended dose of twice-daily application in a thin layer to the affected areas. Use should be limited to no more than one-fifth of the total body surface area. For children, the total application should not exceed 60 grams (one tube) over a period of two weeks. For adults, total application should not exceed 60 grams over one week or one 100 gram tube over two weeks. Treatment should be discontinued once symptoms resolve. It is not intended for continual use. Opzelura should not be used at the same time as an oral JAK inhibitor, such as Xeljanz® (tofacitinib); a biologic therapy such as Dupixent® (dupilumab) or an immunosuppressant, such as cyclosporine. Its labeling carries the same boxed warnings as oral JAK inhibitors used to treat inflammatory conditions. They outline increased risks of blood clots, cancer and serious infections that may be associated with the use of a JAK inhibitor. Patients taking or using one of them also should be screened for tuberculosis (TB) before starting treatment and periodically while therapy continues. The updated prescribing information may be found here.