Rising Sirturo Resistance Threatens TB Treatment Progress, Experts Call for New Therapies
• As Sirturo (bedaquiline) resistance emerges in multiple countries including South Africa and China, experts emphasize the urgent need for new tuberculosis therapies to combat multi-drug resistant TB.
• Despite being the world's top infectious killer with 1.5 million annual deaths, TB research remains severely underfunded, receiving only one-fifth of the $5 billion annual target in 2022.
• Initiatives like UNITE4TB are launching clinical trials to develop shorter treatment regimens and combat resistance, though funding cuts from the US threaten to further disrupt global TB response efforts.
FDA Approves Spravato (Esketamine) Nasal Spray as Standalone Treatment for Treatment-Resistant Depression
• The FDA has approved Spravato (esketamine) nasal spray as a monotherapy for adults with treatment-resistant depression (TRD).
• Spravato demonstrated rapid and superior improvement in depressive symptoms compared to placebo in clinical trials.
• The approval was based on a study where 22.5% of patients achieved remission with Spravato compared to 7.6% with placebo.
• Due to risks, Spravato is only available through a restricted program under the supervision of a healthcare provider.
Johnson & Johnson Seeks FDA Approval for Tremfya to Treat Pediatric Psoriasis and Arthritis
• Johnson & Johnson has submitted sBLAs to the FDA for Tremfya (guselkumab) to treat moderate-to-severe plaque psoriasis in children aged 6 and older.
• The submission also seeks approval for Tremfya to treat active juvenile psoriatic arthritis in children aged 5 and older.
• The sBLAs are based on data from the Phase 3 PROTOSTAR study and pharmacokinetic data from adult studies VOYAGE 1 and 2, and DISCOVER 1 and 2.
• Tremfya, an IL-23 inhibitor, is already approved for adults with plaque psoriasis, psoriatic arthritis, and ulcerative colitis.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
European Commission Approves Darzalex SC with VRd for Newly Diagnosed Multiple Myeloma
• The European Commission has approved Darzalex subcutaneous formulation (SC) in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for newly diagnosed multiple myeloma (NDMM) patients eligible for autologous stem cell transplant (ASCT).
• The approval is based on the Phase 3 PERSEUS study, which demonstrated a 58% reduction in the risk of disease progression or death compared to VRd alone.
• The Darzalex regimen led to deeper responses, with a minimal residual disease (MRD) negativity rate of 75.2%.
• This approval marks a significant advancement in frontline therapy, aiming to transform outcomes and establish new standards of care for eligible NDMM patients.
Enterprise Therapeutics Appoints Annabella Amatulli as Head of Regulatory Affairs
• Enterprise Therapeutics has appointed Annabella Amatulli as Head of Regulatory Affairs to bolster its strategic leadership team.
• Amatulli will spearhead regulatory strategy, focusing on the Phase 2 clinical trial of ETD001 for cystic fibrosis (CF) patients not benefiting from CFTR modulators.
• Her extensive experience includes securing Orphan Drug Designation and Breakthrough Therapy approvals from EMA, FDA, MHRA, and NMPA.
• Amatulli's role will also encompass defining regulatory pathways for Enterprise’s pipeline of novel low molecular weight compounds.
Nipocalimab Shows Sustained Disease Control in Adolescents with Generalized Myasthenia Gravis
• Nipocalimab, combined with standard of care, demonstrates sustained disease control in adolescents (12-17 years) with generalized myasthenia gravis (gMG) over 24 weeks.
• The Phase 2/3 Vibrance-MG study shows a significant reduction in immunoglobulin G (IgG) levels and improvements in MG-ADL and QMG scores.
• Nipocalimab is well-tolerated, with no serious adverse events reported, offering a potential new treatment option for adolescents with gMG.
• Johnson & Johnson has submitted applications to the FDA and EMA seeking approval for nipocalimab for the treatment of gMG.
European Study Reveals Harmful Impact of Delayed Myasthenia Gravis Diagnosis
• A new European study finds patients with myasthenia gravis wait an average of 363 days for accurate diagnosis, with over 27% experiencing delays exceeding one year.
• Patients with diagnostic delays over one year showed higher rates of MGFA class III disease, increased fatigue, anxiety, and depression compared to those diagnosed within a year.
• The study spanning five European countries reveals 69.2% of patients with extended delays received incorrect initial diagnoses, highlighting urgent need for improved diagnostic strategies.
NICE Declines NHS Coverage of Spravato for Treatment-Resistant Depression Citing Cost and Clinical Concerns
• The UK's healthcare cost-effectiveness agency NICE has rejected Johnson & Johnson's nasal spray antidepressant Spravato for NHS funding, citing concerns over its £10,500 per course price tag and clinical evidence gaps.
• NICE highlighted uncertainties about Spravato's long-term efficacy maintenance and lack of comparative data against existing treatments like ECT and psychological therapies for treatment-resistant depression.
• Janssen-Cilag, J&J's pharmaceutical unit, criticized the decision, arguing it could deny access to a needed treatment option for patients with treatment-resistant major depressive disorder, affecting up to 30% of depression cases.
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