Wave Life Sciences to Seek FDA Approval for Duchenne Muscular Dystrophy Drug Following Promising Phase 2 Results
• Wave Life Sciences will pursue FDA accelerated approval for WVE-N531, an exon 53 skipping therapy for Duchenne muscular dystrophy, following positive Phase 2 trial results showing increased dystrophin production.
• The experimental treatment demonstrated substantial improvements in muscle health with a statistically significant improvement in "time to rise" functional tests compared to historical controls through 48 weeks of treatment.
• If approved, WVE-N531 could offer a potential monthly dosing regimen and become a new therapeutic option for approximately 8-10% of Duchenne patients with specific genetic mutations amenable to exon 53 skipping.
FDA Signals Potential Traditional Approval Pathway for Genelux's Olvi-Vec in Platinum-Resistant Ovarian Cancer
• FDA indicates that Genelux's ongoing Phase 3 OnPrime/GOG-3076 trial could potentially support traditional approval for Olvi-Vec in platinum-resistant/refractory ovarian cancer without requiring a separate confirmatory study.
• The regulatory guidance represents a significant milestone for Genelux, potentially accelerating the approval timeline if the trial demonstrates clinically meaningful progression-free survival advantage without compromising overall survival.
• Topline safety and efficacy data from the registrational trial are expected in the first half of 2026, addressing an urgent unmet need for innovative treatments in this difficult-to-treat patient population.
Japan Approves Groundbreaking Viral Therapy Teserpaturev for Glioblastoma Treatment
• Japan's health ministry committee has approved Teserpaturev, the country's first virotherapy drug developed by Daiichi Sankyo, marking a significant breakthrough in cancer treatment.
• The novel therapy, which uses modified herpesvirus to selectively target and destroy brain cancer cells, demonstrated a remarkable 92.3% one-year survival rate in clinical trials.
• The treatment, designed for malignant glioma patients with recurring tumors, showed a median survival period of 20 months, substantially exceeding traditional treatment outcomes.
Sarepta Seeks Accelerated Approval for DMD Gene Therapy SRP-9001
• Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients.
• The filing is based on positive data from early-stage studies, showing improvements in clinical function and a consistent safety profile, while awaiting Phase 3 EMBARK results.
• SRP-9001, a one-time gene therapy, delivers a shortened dystrophin gene via an AAV vector, addressing the underlying genetic defect in DMD patients.
• If approved, SRP-9001 would offer a one-time treatment option for DMD, contrasting with Sarepta's existing chronic exon-skipping therapies.
Genelux Doses First Patient in Phase 2 Trial of Olvi-Vec for Recurrent NSCLC
• Genelux has dosed the first patient in a Phase 2 clinical trial (VIRO-25) evaluating Olvi-Vec for recurrent non-small cell lung cancer (NSCLC).
• The trial will assess Olvi-Vec's efficacy and safety in combination with platinum-doublet chemotherapy and an immune checkpoint inhibitor (ICI) versus docetaxel.
• Olvi-Vec is an oncolytic vaccinia virus being developed as a potential treatment for various cancers, demonstrating a manageable safety profile in prior studies.
• Interim data from the Phase 2 trial is expected in mid-2025, potentially offering a new systemic treatment option for NSCLC patients with limited options.
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