New restrictions on Sarepta gene therapy
The FDA is limiting the use of Sarepta’s Elevidys gene by adding a boxed warning for acute liver injury and narrowing the gene therapy’s indication. Elevidys is now approved only for ambulatory patients aged four years and older with Duchenne muscular dystrophy bearing a confirmed mutation. The non-ambulatory indication has been removed following reports of fatal acute liver failure in this group.
The updated limitations of use advise that Elevidys should not be administered to individuals with pre-existing liver impairment, recent vaccination, or recent or active infection. The revised safety information also introduces enhanced monitoring requirements, including weekly liver function tests for at least three months after infusion and a recommendation for patients to remain near a medical facility for at least two months post-treatment.
As part of the regulatory action, FDA has required a post-marketing observational study of approximately 200 patients to further evaluate the risk of serious liver injury over a minimum of 12 months.
FDA mulls new plausible mechanism pathway for personalized therapies
Writing in The New England Journal of Medicine, the FDA’s Vinay Prasad and Martin Makary have outlined plans for a new regulatory approval pathway for personalized medicines. The “plausible mechanism” pathway is intended for highly individualized or small-population therapies where traditional clinical trial designs may not be feasible. Prasad and Makary believe the pathway could be applied in diseases with a clearly defined biological cause where therapies are designed to act directly on that causal mechanism, and in conditions with well-characterized natural histories that allow treatment effects to be assessed without large comparator groups. The framework relies on evidence that a therapy engages its intended target and produces clinically meaningful changes that cannot reasonably be attributed to spontaneous fluctuations in disease course. The FDA indicates that multiple successful cases could support broader authorization for similar products built on the same platform, with post-marketing studies required to monitor long-term safety and durability.
Non-viral vell therapy manufacturing
Kytopen and Excellos will explore a collaboration to advance non-viral cell therapy manufacturing technologies. The initiative will assess how Kytopen’s continuous-flow cellular engineering platform, Flowfect Tx, aligns with Excellos’s donor-to-dose manufacturing model and cell-characterization capabilities. The two organizations intend to evaluate whether combining these technologies can improve process consistency, scalability and overall cell quality in the development of next-generation cell-based therapies. The initial phase of the initiative will focus on identifying scientific and operational synergies, with the possibility of further cooperation as development priorities converge.
Sandoz and EirGenix in biosimilar agreement
A global license agreement with EirGenix will grants Sandoz exclusive rights to commercialize a proposed biosimilar of pertuzumab, indicated for HER2-positive early and metastatic breast cancer. The agreement is milestone-based and covers up to $152 million in payments, including an upfront component, with EirGenix responsible for development, manufacturing and supply of the biosimilar. Under the terms, Sandoz holds worldwide rights, excluding certain Asian territories. The reference medicine pertuzumab is reported to hold global sales of approximately $4.1 billion. The biosimilar has completed a human pharmacokinetic similarity study, and the agreement builds on an existing collaboration between the two companies for a trastuzumab biosimilar.
New gene therapy approval in Europe
The European Medicines Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion recommending the first gene therapy for the treatment of Wiskott‑Aldrich syndrome, a rare inherited immunodeficiency. Waskyra (etuvetidigene autotemcel) was developed by Fondazione Telethon ETS in coordination with the San Raffaele‑Telethon Institute for Gene Therapy in Milan. The gene therapy is an autologous CD34⁺ haematopoietic stem‐cell product transduced ex-vivo with a lentiviral vector encoding the human WAS gene, aiming to provide a one-time treatment for patients. The recommendation will now be forwarded to the European Commission for final authorisation, after which Waskyra could become the first gene therapy approved for this condition in Europe.
Positive results in Novartis phase III trial for malaria treatment
Novartis reported that its phase III trial of KLU156 (ganaplacide/lumefantrine, marketed internally as “GanLum”) met primary endpoint in the treatment of acute, uncomplicated malaria. The KALUMA study, conducted across 34 sites in 12 African countries and involving approximately 1,688 adults and children, compared this new regimen against the current standard of care, artemether-lumefantrine (Coartem). The PCR-corrected cure rate at day 29 was 97.4 per cent for GanLum versus 94.0 per cent for the comparator. Beyond the primary endpoint, data showed high activity of GanLum against parasite strains associated with partial drug resistance and a rapid response against mature gametocytes, the sexual stage of the parasite responsible for transmission. GanLum consists of a novel non-artemisinin compound, ganaplacide, developed by Novartis in collaboration with the non-profit Medicines for Malaria Venture (MMV), paired with a new once-daily formulation of lumefantrine. With plans underway to seek regulatory approval, Novartis states this could represent the first significant innovation in malaria treatment since the late 1990s.
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