New Steqeyma Formulation for Pediatric Dosing

The US Food and Drug Administration (FDA) granted approval on June 13, 2025, for a new presentation of Celltrion’s Steqeyma^® (ustekinumab-stba), a biosimilar to Janssen Pharmaceutical’s Stelara^® (ustekinumab). This new presentation, a 45mg/0.5mL single-dose vial for subcutaneous (SC) injection, is approved for pediatric patients ages six to 17 years, weighing under 60kg, who have plaque psoriasis (PsO) or psoriatic arthritis (PsA). In December 2024, the FDA approved Steqeyma in 45mg/0.5mL and 90mg/mL solutions in single-dose prefilled syringes for SC injection, and 130mg/26mL single-dose vials for intravenous (IV) infusion, for adult and pediatric patients aged six years and older with PsO and PsA, as well as adult patients with Crohn’s disease and ulcerative colitis. This approval expands dosing flexibility and matches all dosage forms and strengths of the reference product. Updated prescribing information will be available on the Drugs@FDA drug database at www.fda.gov.

Elevidys Use Suspended in Non-Ambulatory Patients After Second Patient Death

On June 15, 2025, Sarepta Therapeutics announced steps to enhance the safety of Elevidys^® (delandistrogene moxeparvovec-rokl) for non-ambulatory Duchenne muscular dystrophy (DMD) patients following two cases of acute liver failure resulting in death. Sarepta is working on an improved immunosuppressive regimen and has halted shipments of Elevidys for non-ambulatory patients. Elevidys, the only gene therapy for DMD, carries a known risk of liver damage with Sarepta reporting severe liver function test (LFT) elevations in approximately 30% of DMD patients. All patients are treated with a corticosteroid beginning one day before the infusion and lasting for at least 60 days after it. Sarepta is currently investigating the addition of sirolimus prior to treatment, to help control liver enzyme elevations. Sarepta has also paused the confirmatory clinical trial required for converting the accelerated approval into a traditional FDA approval for non-ambulatory patients to possibly incorporate this new regimen. Full prescribing information is available here.

Follicular Lymphoma Indication for Monjuvi

Incyte’s Monjuvi^® (tafasitamab-cxix) was approved by the FDA in combination with rituximab and lenalidomide for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) on June 18, 2025. A slowly progressing form of non-Hodgkin lymphoma (NHL), FL affects about 15,000 new patients in the US each year, mostly among older adults. Although relatively easy to treat, FL frequently develops resistance to treatment and therefore relapses. This is the first CD19- and CD20-targeted immunotherapy combination for FL. Approval is based on the phase III trial, which showed significantly improved progression-free survival (PFS) with Monjuvi compared to placebo. Initially approved in July 2020, Monjuvi combined with lenalidomide was FDA-approved for treating adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and labeling includes warning and precautions for infusion-related reactions, myelosuppression, and infections. The recommended dose for FL is 12mg/kg as an IV infusion for a maximum of 12 cycles. Approval was granted under Project Orbis, an FDA initiative under the Oncology Center of Excellence that speeds up approvals for drugs that may fill critical unmet needs. Here is the updated prescribing information.

FDA Adds New Warning to Transderm Scōp Antinausea Patch

On June 18, 2025, the FDA issued a warning that Baxter Healthcare’s Transderm Scōp^® (scopolamine transdermal system/generics) patch can increase body temperature and cause heat-related complications, leading to hospitalization or death, particularly in children under 17 years and adults over 60 years of age. Transdermal scopolamine is used to prevent nausea and vomiting induced by motion or by surgery. The FDA required updates to the prescribing information to include warnings about this risk. Hyperthermia often occurs within 72 hours of first application. Patients should remove the patch if they experience increased body temperature or reduced sweating and contact their healthcare professional. Updated prescribing information is available here.

First Treatment for Alkaptonuria Approved

Cycle Pharmaceuticals announced that a new formulation of nitisinone, Harliku^™ was approved by the FDA on June 19, 2025, for the reduction of urine homogentisic acid (HGA) in adult patients with alkaptonuria (AKU). AKU is an ultra-rare genetic metabolic disorder caused by an enzyme deficiency that leads to the accumulation of HGA in the body. This causes darkening of the urine and connective tissues, as well as joint and spine problems like osteoarthritis and requiring large joint replacements, and complications in the kidneys and heart over time. It is estimated less than 300 Americans are living with this disorder. A hydroxyphenyl-pyruvate dioxygenase inhibitor, Harliku is a new formulation of nitisinone. Orfadin^® (nitisinone – SOBI) capsules were FDA approved as an orphan drug to treat hereditary tyrosinemia type-1 (HT-1) in 2002. In 2017, bioequivalent tablets to Orfadin capsules were approved, Nityr^® (nitisinone – Cycle Pharmaceuticals). The recommended dose of Harliku is 2mg orally once daily. Prescribing information can be found here.

Bullous Pemphigoid Indication for Dupixent

On June 20, 2025, the FDA granted another indication to Sanofi/Regeneron’s Dupixent^® (dupilumab) for treating adult patients with bullous pemphigoid (BP). This is the first FDA-approved drug to treat BP, a chronic, relapsing disease that causes intense itching, blisters, red skin, and painful lesions most often observed in elderly patients. Patients face increased infection risk, and the widespread blisters and rash can bleed, scab, and affect daily functioning. About 27,000 adults in the US have BP that is not controlled by systemic corticosteroids. This approval is based on the pivotal ADEPT phase II/III study showing Dupixent significantly improved sustained disease remission, reduced itch, and decreased oral corticosteroid use compared to placebo. The recommended dose for treating BP is an initial SC loading dose of 600mg (two 300mg injections) followed by 300mg every other week in combination with tapering a course of oral corticosteroids. Dupixent also has indications for treating atopic dermatitis, eosinophilic esophagitis (EoE), chronic obstructive pulmonary disease (COPD), certain types of asthma, chronic rhinosinusitis with nasal polyposis, and prurigo nodularis. Updated prescribing information can be found here.

Recall

Sodium Chloride IV Bags Recalled

On June 17, 2025, B. Braun Medical Inc. issued a recall for 0.9% (Sodium Chloride) NACL INJ USP 500mL due to potential fluid leakage from small pinholes in the IV bags. The FDA-approved use for 0.9% NACL INJ USP 500mL is for IV administration to provide fluid and electrolyte replenishment, and it can also be used to prepare other sterile drugs, including chemotherapy and other hazardous drugs. The leakage may pose risks such as falls, slips, treatment delays, exposure to hazardous drugs, and potential bloodstream infections, particularly in vulnerable patients. For more information about the recall, see here.