FDA Approves Updated Elevidys Label with Boxed Warning and Restricted Indication

On Nov. 14, 2025, the   US Food and Drug Administration (FDA) updated labeling for Sarepta Therapeutics’ Elevidys^® (delandistrogene moxeparvovec-rokl), an AAVrh74-based gene therapy for Duchenne muscular dystrophy (DMD), following reports of fatal cases of acute liver failure in non-ambulatory patients. The revised label adds a boxed warning, the FDA’s most serious safety warning, for acute liver failure and restricts use to ambulatory patients age four years and older who have a confirmed DMD mutation, meaning the label removes the non-ambulatory indication. It also introduces new limitations of use for patients with liver impairment, recent infections or vaccinations, calls for weekly liver function tests for three months and cardiac monitoring for one month after infusion, along with staying near an appropriate medical facility for at least two months post-infusion. The FDA is also adding a new patient medication guide to help inform patients and caregivers of the risks related to therapy. The FDA is also requiring a post marketing study of about 200 patients for at least 12 months to further assess liver safety. The label changes follow two reports of fatal acute liver failure in non-ambulatory pediatric patients treated with Elevidys and an additional serious case involving complications such as bowel ischemia and portal hypertension. Sarepta voluntarily paused dosing for non-ambulatory patients earlier this year and is now planning a study of an enhanced sirolimus regimen to mitigate liver risk and potentially restore access for that population. Here is the prescribing information and new Medication Guide.

Sandoz Launches Tyruko, First FDA-Approved Biosimilar for Multiple Sclerosis

On Nov. 17, 2025, Sandoz announced the US launch of Tyruko^® (natalizumab-sztn) injection, the first biosimilar for Tysabri^® (natalizumab – Biogen). Tyruko was initially approved on Aug. 24, 2023, for the treatment of relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome, relapsing-remitting MS and active secondary progressive disease, as well as Crohn’s disease in adults.Developed by Polpharma Biologics, Tyruko shares the same intravenous (IV) dosing regimen and carries a boxed warning for progressive multifocal leukoencephalopathy (PML). It is distributed under a Risk Evaluation and Mitigation Strategy (REMS) program to manage this risk. Approval was based on phase I and phase III studies showing equivalent efficacy, safety and immunogenicity to the reference product. The launch came after delays tied to patent litigation and the need for FDA clearance of a John Cunningham virus antibody test required under the REMS program, which created uncertainty around the timing of market entry. In 2024, Tysabri’s total sales amounted to about $1.53 billion. Full prescribing information is available here.

Epkinly Combination Approved for Relapsed/Refractory Follicular Lymphoma

The FDA approved AbbVie’s Epkinly^® (epcoritamab-bysp), in combination with rituximab and lenalidomide for the treatment of adult patients who have relapsed or refractory follicular lymphoma (FL) after at least one prior systemic therapy on Nov. 18, 2025. Epkinly is a bispecific CD20-directed CD3 T-cell engager that is administered as a subcutaneous (SC) injection by a healthcare professional. FL is a slow-growing type of non-Hodgkin lymphoma, affecting about 15,000 Americans annually. It is considered incurable with current treatments and can relapse or transform into a more aggressive diffuse large B-cell lymphoma (DLBCL). Epkinly was first approved in May 2023 for treating adults with DLBCL after two or more lines of systemic therapy. Approval was based on the phase III EPCORE FL-1 trial, which reduced the risk of progression or death by 79% (p<0.0001) and had an overall response rate of 89%, including 74% complete responses, compared with rituximab plus lenalidomide alone. The recommended regimen follows a fixed-duration schedule with step-up dosing to mitigate cytokine release syndrome (CRS), which occurred in 24% of patients. The prescribing information includes a boxed warning for CRS and immune effector cell-associated neurotoxicity syndrome (ICANS), along with precautions for infections, cytopenia and embryo-fetal toxicity. The FDA also granted traditional approval to Epkinly as monotherapy for relapsed or refractory FL after two or more lines of systemic therapy on Nov. 18, 2025, for which accelerated approval was granted in 2024. Here is the updated prescribing information.

FDA Expands THROMBATE III Indication to Include Pediatric Patients

On Nov. 18, 2025, the FDA approved an expanded indication for Grifols’ THROMBATE III^® (antithrombin III [human]), a plasma-derived concentrate, to include pediatric patients who have hereditary antithrombin deficiency (hATd), in addition to adults. THROMBATE III is the only antithrombin concentrate approved for both adult and pediatric patients. Hereditary antithrombin deficiency is an underdiagnosed clotting disorder that could affect up to 700,000 Americans, with most patients facing an 85% chance of experiencing at least one blood clot by age 50. The therapy, administered intravenously (IV), helps restore antithrombin levels to reduce the risk of abnormal blood clots. Approval was based on data extrapolation from two adult clinical trials, supported by decades of safe use in adults, confirming that THROMBATE III can be safely and effectively used in children. The dose is tailored to maintain antithrombin levels between 80% and 120% of normal, with the full amount infused over 10 to 20 minutes and adjusted based on patient response. Here is the updated prescribing information.

Koselugo’s NF1 Indication Expanded to Adults

The FDA approved AstraZeneca’s Koselugo^® (selumetinib) oral capsules for the treatment of adult patients who have neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN) on Nov. 19, 2025. This expands Koselugo’s prior pediatric indication and joins SpringWorks Therapeutics’ Gomekli^™ (mirdametinib) as a treatment for adults who have symptomatic NF1-PN that is unable to be removed by surgery. Koselugo is a mitogen-activated protein kinase (MEK) 1/2 inhibitor that targets the RAS/mitogen activated protein kinase (MAPK) pathway, which is overactive in NF1. Approval was based on results from a clinical trial showing a confirmed overall response rate (ORR) of 20% versus 5% for placebo, with 86% of responders maintaining benefit for at least six months. The recommended dose is 25mg/m² orally twice daily until disease progression or unacceptable toxicity. Prescribing information includes warnings for left ventricular dysfunction, ocular and gastrointestinal toxicity, skin reactions, elevated creatine phosphokinase, increased vitamin E levels, bleeding risk and embryo-fetal toxicity. The updated prescribing information may be found here.

FDA Grants Traditional Approval for Darzalex Faspro in AL Amyloidosis

The FDA granted traditional approval to Janssen Biotech’s Darzalex Faspro^® (daratumumab and hyaluronidase-fihj) injection, in combination with bortezomib, cyclophosphamide and dexamethasone, for the treatment of newly diagnosed light chain (AL) amyloidosis in adults. The drug received accelerated approval for this indication in 2021. Traditional approval was based on an open-label, randomized study demonstrating a hematologic complete response rate of 42.1% for the Darzalex Faspro regimen versus 13.5% for the control arm (p<0.0001). Full prescribing information is available here.

Imdelltra for Extensive-Stage Small Cell Lung Cancer Gets Traditional Approval

On Nov. 19, 2025, the FDA granted traditional approval to Amgen’s Imdelltra^® (tarlatamab-dlle), a bispecific delta-like ligand 3 (DLL3)-directed CD3 T-cell engager, for the treatment of adults who have extensive-stage small cell lung cancer (ES-SCLC) who experienced disease progression on or after platinum-based chemotherapy. Imdelltra received accelerated approval for this indication in May 2024. Traditional approval is based on results from the phase III trial, DeLLphi-304, which showed a 40% reduction in risk of death versus standard chemotherapy. It extended median overall survival by 13.6 months compared with 8.3 months for standard chemotherapy in patients with ES-SCLC after platinum-based treatment, with significant gains in progression-free survival and symptom relief. The National Comprehensive Cancer Network^® (NCCN^®) Clinical Practice Guidelines in Oncology (NCCN Guidelines^®) were recently updated to include Imdelltra as a category 1 preferred option. Here is the prescribing information.

More Denosumab Biosimilars Approved

On Nov. 20, 2025, the FDA approved Accord BioPharma’s Osvyrti^® (denosumab-desu) and Jubereq^® (denosumab-desu), biosimilars to Prolia^® and Xgeva^® respectively, for the treatment of osteoporosis and skeletal-related events in cancer patients. Osvyrti 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 SC injection once every six months. Jubereq 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 as a 120mg SC injection once monthly by a healthcare provider. Jubbonti^® (denosumab-bbdz – Sandoz), the first biosimilar to Prolia, and Wyost^® (denosumab-bbdz – Sandoz), the first biosimilar to Xgeva, launched on May 31, 2025. Other biosimilars are expected to launch this year and Accord BioPharma states Osvyrti and Jubereq will launch in 2026. Here is the prescribing information. 

Two New Approvals for High Dose Eylea

The FDA granted Regeneron’s Eylea^® HD (aflibercept injection 8mg) two new indications on Nov. 19, 2025. First, for the treatment of patients with macular edema following retinal vein occlusion (RVO) with up to every eight-week dosing after an initial monthly dosing period. The FDA also approved an every four-week (monthly) dosing option for some patients who may benefit from this dosing schedule across approved indications: wet age-related macular degeneration (wAMD), diabetic macular edema (DME), diabetic retinopathy (DR) and RVO. Approval for RVO is supported by the phase III QUASAR study, which found that patients receiving high-dose Eylea reached a best-corrected visual acuity of 72.8 letters at 36 weeks. Those treated with the standard dose averaged 72 letters over the same period, meeting the trial’s goal of non-inferiority. This marks the first eight-week dosing option for RVO. The four-week dosing regimen for Eylea HD, applicable to all indications, is designed to support patients who experience a loss of vision improvement when treated at extended intervals. Clinical trials showed some patients failed to maintain response after moving to longer dosing schedules; those individuals may benefit from returning to monthly injections. The approval also aims to secure payer coverage for more frequent dosing. The safety profile remains consistent with the lower-dose formulation, with warnings for endophthalmitis and retinal detachment. A pre-filled syringe version of high-dose Eylea was rejected by the FDA last month, citing unresolved issues at the manufacturing facility. Regeneron plans to resubmit its application in January 2026, which will include an alternate manufacturing filler for the biologics license. The updated prescribing information is available here.