TRENDING TOPICS

First Topical Gene Therapy Approved For Deb

The FDA has approved beremagene geperpavec-svdt (Vyjuvek™), an HSV-1 vector-based gene therapy, for treatment of wounds in patients ≥ 6 months of age with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. Vyjuvek works by increasing production and secretion of the COL7 protein, resulting in improved wound healing. The product is available as a 5 x 109 PFU/mL biological suspension in a single-use vial, which is mixed into an excipient gel prior to administration. Vyjuvek is applied topically to the wound(s) once weekly by an HCP until wound closure. The recommended dosage per wound ranges from 4 x 108 PFU (0.2 mL) to 1.2 x 109 PFU (0.6 mL) depending on wound size. The maximum weekly dosage is 1.6 x 109 PFU (0.8 mL) for patients ages 6 months to < 3 years and 3.2 x 109 PFU (1.6 mL) for patients ≥ 3 years of age. Application to all wounds may not be possible at each treatment visit, and treatment should continue until wound closure before new wounds are selected for treatment. Previously treated wounds that have re-opened should be prioritized. Vyjuvek carries a warning for accidental exposure; though the drug will not replicate or integrate into the genetic material of the subject’s cells, HCPs should follow universal biohazard precautions when handling the medication.

The efficacy and safety of Vyjuvek were evaluated in an intra-patient randomized, double-blind, placebocontrolled trial, which included 31 patients with DEB. All study participants were ≥ 6 months of age with clinical manifestations of DEB and genetically confirmed COL7A1 gene mutation(s). The age of study participants ranged from 1 year to 44 years (mean 17 years), and the size of treated wounds ranged from 2 cm2 to 57 cm2. Two wounds were identified in each subject; one wound was treated with topical Vyjuvek gel and the other was treated with placebo once weekly for 26 weeks. The primary efficacy endpoint was the difference in proportion of complete (100%) wound closure after 6 months of treatment, confirmed at 2 consecutive visits 2 weeks apart. After 6 months, 67% of wounds had completely closed with Vyjuvek therapy compared to 22% of those treated with placebo, for a treatment difference of 46% (95% CI, 24% to 68%, p=0.002).

Weight Management Corner

An FDA Drug Safety Communication has been released regarding ADR reports with use of compounded semaglutide products that may not contain the same active ingredient as FDA-approved semaglutide (Ozempic®, Rybelsus® , Wegovy®) and instead may contain salt formulations (e.g., semaglutide sodium, semaglutide acetate). The agency recommends patients avoid compounded semaglutide if an approved drug is available. Novo Nordisk issued a notice regarding the sale of a counterfeit semaglutide (Ozempic) pen containing insulin glargine. It is recommended that pharmacies purchase product through an authorized distributor and that patients check the product label prior to use.

Biosimilar Notables

Adalimumab-aaty (Yuflyma®), a biosimilar to adalimumab (Humira®), has been approved for treatment of RA, JIA, PsA, AS, CD, UC, PsO, and HS. The product will be available as a high-concentration, citrate-free 40 mg/0.4 mL singledose prefilled autoinjector and syringe. The FDA has approved a new 40 mg/0.8 mL single-dose prefilled autoinjector pen presentation for the interchangeable Humira biosimilar adalimumab-adbm (Cyltezo®).

Behavioral Health Updates

The first nalmefene nasal spray, Opvee®, received approval for emergency treatment of known or suspected overdose induced by natural or synthetic opioids in patients ≥ 12 years of age, as manifested by respiratory and/or CNS depression. Opvee spray contains nalmefene 2.7 mg (0.1 mL). The recommended dosage is 1 spray intranasally with additional doses given every 2 to 5 minutes, if needed, until emergency assistance arrives. An ER buprenorphine injection, Brixadi™, has been approved for treatment of moderate to severe opioid use disorder in patients who have initiated treatment with a single dose of transmucosal buprenorphine or who are already being treated with buprenorphine. This SC injection is administered by an HCP weekly or monthly, with dosage and frequency depending on prior buprenorphine treatment. Brixadi is a CIII controlled substance and requires REMS certification. Escitalopram’s (Lexapro®) indication for GAD has been expanded to include patients 7 to 17 years of age. The initial dose for pediatric patients for GAD is 10 mg orally once daily, with a maximum dose of 20 mg daily.