Tagged News
FDA Approves 18 New Personalized Medicines in 2024, Marking Significant Shift in Treatment Paradigm
- The FDA approved 18 new personalized medicines in 2024, representing 38% of all newly approved therapeutic molecular entities across multiple treatment areas including cancer and Alzheimer's disease.
- Six new gene and cell-based therapies for rare genetic diseases and cancers were authorized, alongside expanded indications for 11 diagnostic testing systems and the first-ever expanded indication for an approved gene therapy.
- Personalized medicines now constitute at least 25% of drug approvals for the past decade, a substantial increase from less than 10% ten years ago, demonstrating the healthcare system's shift away from one-size-fits-all approaches.
Tessera Therapeutics Reports Breakthrough Gene Editing Results for Multiple Genetic Diseases
- Tessera's RNA Gene Writer technology achieved 76% and 70% editing efficiency in hepatocytes for alpha-1 antitrypsin deficiency and phenylketonuria respectively, with high specificity and durability in non-human primate studies.
- Preclinical data for sickle cell disease demonstrated greater than 20% editing in long-term hematopoietic stem cells across multiple species, potentially reaching curative thresholds without requiring stem cell transplantation.
- The company's proprietary lipid nanoparticle delivery system showed high liver specificity with no off-target activity detected, advancing the potential for in vivo gene editing therapies for multiple genetic disorders.
Eisai's Remitoro Clears Post-Marketing Surveillance Requirements for T-Cell Lymphoma Treatment in Japan
- Japan's Ministry of Health has lifted the all-case surveillance requirement for Eisai's anticancer agent Remitoro after reviewing safety data from 111 patients and efficacy data from 85 patients with T-cell lymphoma.
- Remitoro, a fusion protein combining IL-2 and diphtheria toxin, demonstrated an overall response rate of 16.5% across both peripheral and cutaneous T-cell lymphoma patients in the post-marketing study.
- Common adverse reactions included elevated liver enzymes (22.5%), capillary leak syndrome (21.6%), and decreased platelet count (15.3%), confirming the safety profile in real-world clinical settings.
First Biologic Drug for Pulmonary Sarcoidosis in 50 Years Administered at Oxford Hospital
- Infliximab, a TNF-α inhibitor, has become the first new drug approved for pulmonary sarcoidosis in over 50 years, following NHS England commissioning with NICE support.
- The biologic therapy, administered to the first patient at Oxford's John Radcliffe Hospital, offers potential to reduce reliance on broad immunosuppressants that often cause significant side effects.
- Professor Ling-Pei Ho, who led the Oxford Sarcoidosis Service and chaired the initial NHS England working policy group, played a key role in securing the drug's approval for this rare condition affecting 1 in 10,000 UK residents.
China Approves First Domestically Developed Enzyme Replacement Therapy for Gaucher Disease
- CANbridge Pharmaceuticals has received NMPA approval for velaglucerase-beta (Gaurunning), China's first domestically developed enzyme replacement therapy for Type I and III Gaucher disease in patients aged 12 and above.
- The pivotal clinical trial demonstrated statistically significant reductions in spleen volume at both 60 U/kg (P<0.0001) and 30 U/kg (P<0.001) doses, meeting its primary efficacy endpoint.
- Developed in collaboration with WuXi Biologics, Gaurunning represents a breakthrough in rare disease treatment in China, potentially improving accessibility and affordability for the estimated 3,000 Chinese Gaucher disease patients.
NICE Extends Access to Brineura for CLN2 Batten Disease While Seeking Long-term Solution
- NICE has secured continued access to cerliponase alfa (Brineura) for current patients and those starting treatment before December 2025, despite not recommending it for routine NHS use due to cost concerns.
- The enzyme replacement therapy, costing over £500,000 per patient annually, has demonstrated effectiveness in slowing CLN2 progression in the short term, but lacks sufficient long-term efficacy data.
- NICE, NHS England, and manufacturer BioMarin continue negotiations to reach a sustainable pricing agreement that could extend access to all future patients with this rare, life-limiting condition.
Acquired Hemophilia A Pipeline Expands with 10+ Novel Therapies in Development
- Global Acquired Hemophilia A pipeline comprises over 10 companies developing innovative therapies across various clinical stages, according to DelveInsight's 2025 report.
- Recent clinical advances include promising results for rituximab as a potential first-line therapy and Pfizer's positive Phase 3 AFFINE trial results for giroctocogene fitelparvovec gene therapy.
- Key pharmaceutical players including Novo Nordisk, Sanofi, Pfizer, and Belief Biomed are advancing treatments ranging from gene therapies to monoclonal antibodies targeting this rare autoimmune bleeding disorder.
Highlighted Clinical Trials:
Pfizer
Posted 8/18/2020
Acadia Pharmaceuticals Secures Patent Victory for Parkinson's Drug Nuplazid Until 2038
- The U.S. District Court for the District of Delaware ruled in favor of Acadia Pharmaceuticals, upholding their '721 formulation patent for Nuplazid against Aurobindo Pharma's generic challenge.
- Judge Gregory B. Williams determined that Aurobindo's proposed generic pimavanserin capsules would infringe Acadia's patent, which provides protection for the 34 mg capsule formulation until August 2038.
- The ruling represents a significant win for Acadia, securing long-term market exclusivity for their FDA-approved treatment for hallucinations and delusions associated with Parkinson's disease psychosis.
Neurogene Unveils Protocol to Reverse HLH Syndrome Associated with High-Dose AAV Gene Therapy
- Neurogene has developed a monitoring and treatment algorithm designed to reverse hemophagocytic lymphohistiocytosis (HLH), a rare hyperinflammatory syndrome linked to high-dose AAV gene therapy.
- The protocol focuses on daily monitoring of the "three Fs" - elevated ferritin levels, fever, and falling blood counts - with high-dose corticosteroids as first-line treatment and anakinra as second-line therapy.
- Despite using a lower dose (E13 vg/kg range) in its Phase 1/2 trial of NGN-401 for Rett syndrome, Neurogene has incorporated this protocol as a precautionary measure, as HLH has not been reported at this dose level.
Highlighted Clinical Trials:
Neurogene Inc.
Posted 6/13/2023
Pfizer
Posted 8/8/2022
Acuitas Therapeutics and CHOP Pioneer Personalized CRISPR Therapy for Infant with Urea Cycle Disorder
- Acuitas Therapeutics, Children's Hospital of Philadelphia, and University of Pennsylvania successfully delivered the world's first personalized LNP-delivered CRISPR gene-editing therapy to an infant with urea cycle disorder, with no adverse events reported.
- The groundbreaking therapy was developed, manufactured, and delivered in just six months, establishing a new model for rapid development of personalized gene therapies through cross-functional partnerships.
- Acuitas also presented advances in targeted LNP delivery, including DARPin-conjugated formulations achieving up to 98% binding and 90% expression in human CD8+ T cells, expanding therapeutic applications beyond the liver.