Epilepsy improvements
UCB has announced the publication of the final analysis from an open-label extension (OLE) study evaluating the long-term safety and effectiveness of Fintepla (fenfluramine) in patients with Lennox-Gastaut syndrome (LGS). The study, published in Epilepsy & Behavior, demonstrates sustained seizure reduction and improved quality of life for patients and their caregivers.
The study included 247 participants from North America, Europe, and Australia. Over a median treatment period of one year, patients experienced a median 31 percent reduction in the frequency of seizures associated with falls (drop seizures), with improvements seen as early as Month 2 and sustained through the study’s end. Adults showed a 40 percent median reduction, while pediatric patients achieved a 27.6 percent reduction. Nearly 60 percent of patients were rated by caregivers and investigators as showing overall improvement.
Caregivers also reported better social interactions, reduced feelings of stigma, and lower anxiety and depression levels. Importantly, no new safety signals emerged, with the most common treatment-emergent adverse events being decreased appetite, fatigue, and nasopharyngitis. No cases of valvular heart disease or pulmonary arterial hypertension were observed.
Kelly G Knupp, Associate Professor of Pediatrics and Neurology at the CU Anschutz School of Medicine, and co-author of the paper, said, “These data reinforce the long-term safety and tolerability of fenfluramine in children and adults living with Lennox-Gastaut syndrome, a condition with a significant unmet medical need. The sustained reduction in seizure frequency, coupled with the meaningful improvements in quality of life for both patients and their families, underscores the importance of addressing not just the clinical but also the emotional and social challenges faced by these families every day.”
Amoxicillin analysis
A report compiled by Portugal-based research consultancy New Angle, and sponsored by Viatris, highlights Amoxicillin as a central example of the challenges facing off-patent antibiotics in Europe. The report notes that availability has fluctuated in recent years, with shortages driven by rising production costs, API concentration, and reduced manufacturer profitability. It also shows how 27 of 125 Amoxicillin products (21.6 percent) were withdrawn from the market, with shortages affecting 69 products. The analysis links these shortages to broader systemic issues, including production fragility and policy-driven price suppression. Amoxicillin, according to the report, exemplifies how economic and regulatory pressures are destabilizing Europe’s antibiotic supply chains.
Head of Europe and Canada at Viatris Artur Cwiok said, “Securing patient access to medicines requires strong and sustainable market policies that reflect the therapeutic value of off-patent medicines, fostering access and supply security for all European patients. We urge policymakers, payers, healthcare professionals and industry leaders to work together to implement reforms that prioritize patient access and supply resilience.”
Dermatitis development
The FDA has approved Arcutis Biotherapeutics’ Zoryve (roflumilast) cream for the treatment of mild to moderate atopic dermatitis in pediatric patients. The approval is supported by data from the INTEGUMENT-PED phase III trial and its long-term extension (INTEGUMENT-OLE). In the pediatric study of 652 children, 39.4 percent reached a 75 percent reduction in disease severity. 25.4 percent of treated children met the “vIGA-AD Success” endpoint versus 10.7 percent in the vehicle group. Safety profiles were favorable, with the most common side effects being upper respiratory infections, diarrhea, vomiting, rhinitis, conjunctivitis, and headache.
This marks the sixth FDA approval in the Zoryve portfolio within just over three years. Arcutis CEO Frank Watanabe said, “Zoryve cream was intentionally formulated to address the critical unmet need in atopic dermatitis care, and it provides a safe, effective alternative to steroid treatments, targeting the underlying inflammation that drives disease activity. Notably, Zoryve does not include ingredients known to compromise the skin barrier, which is especially important for young children with atopic dermatitis.”
Investigating iopofosine
The EMA has confirmed that Collectar Biosciences’ investigational therapeutic iopofosine I-131 is eligible for submission of a Conditional Marketing Authorization (CMA) as a treatment for patients with Waldenström macroglobulinemia (WM), a B-cell malignancy characterized by bone marrow infiltration with clonal lymphoplasmacytic cells that produce a monoclonal immunoglobulin M (IgM) that remains incurable with available treatments.
The decision follows scientific guidance from the EMA’s Scientific Advice Working Party (SAWP), which reviewed the safety, efficacy, manufacturing, and phase II clinical trial data. The SAWP confirmed that iopofosine meets eligibility criteria for a CMA filing in the proposed patient population (post-BTKi), although they do not determine whether the existing data are sufficient for approval.
Iopofosine is a novel radioconjugate using the company’s phospholipid ether platform. If authorized, the therapy could become commercially available across the 30 EMA member countries as early as 2027. CEO James Caruso said, “We are thrilled to take this important step toward bringing iopofosine I-131 to patients in Europe living with WM. With PRIME designation already in hand and feedback from the SAWP, we are rapidly proceeding toward a potential European approval and commercial availability in 2027.”