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Ionis Reports Positive Phase 3 Results for Olezarsen in Hypertriglyceridemia

Drug Approval
  • Olezarsen demonstrated significant triglyceride reductions of 61% and 58% at 6 months with 80mg and 50mg monthly doses respectively in patients with moderate hypertriglyceridemia.
  • The Phase 3 Essence study met all primary and secondary endpoints, with most participants achieving triglyceride levels within normal range despite already being on standard lipid-lowering therapies.
  • Following recent FDA approval of olezarsen (Tryngolza) for familial chylomicronemia syndrome, these results support potential expanded indication for severe hypertriglyceridemia pending upcoming CORE and CORE2 trial data.

A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia

NCT05610280CompletedPhase 3
Ionis Pharmaceuticals, Inc.
Posted 11/21/2022

Ensifentrine Shows Promise as Novel Monotherapy and Add-On Treatment for COPD

Drug Approval
  • New data from the Phase 3 ENHANCE trials demonstrates that ensifentrine, a dual phosphodiesterase 3 and 4 inhibitor, significantly improves lung function and reduces exacerbations in COPD patients.
  • Ensifentrine monotherapy showed meaningful improvements in patient-reported outcomes including dyspnea, COPD symptoms, and health-related quality of life compared to placebo in patients not on long-acting medications.
  • As the first nebulized non-steroidal therapy with a novel dual mechanism of action, ensifentrine represents an important addition to the COPD treatment arsenal, offering benefits both as first-line and add-on therapy.

Dose Ranging Study of RPL554 in Chronic Obstructive Pulmonary Disease (COPD) Patients

NCT03443414CompletedPhase 2
Verona Pharma plc
Posted 6/1/2017

A Phase 3 Trial to Evaluate the Safety and Efficacy of Ensifentrine in Patients With COPD

NCT04542057CompletedPhase 3
Verona Pharma plc
Posted 9/22/2020

A Phase 3 Clinical Trial to Evaluate the Safety and Efficacy of Ensifentrine in Patients With COPD

NCT04535986CompletedPhase 3
Verona Pharma plc
Posted 9/29/2020

FDA Expands Jivi Approval for Hemophilia A Treatment in Children Ages 7-12

Drug Approval
  • The FDA has approved Bayer's Jivi (antihemophilic factor recombinant, PEGylated-aucl) for pediatric patients aged 7 to 12 years with hemophilia A, expanding treatment options for this age group.
  • The approval was based on data from the Alfa-PROTECT and PROTECT Kids studies, which demonstrated the safety and efficacy of Jivi in pediatric patients with severe hemophilia A.
  • Jivi was previously approved in 2018 for adults and adolescents aged 12 and older, and can be used for on-demand treatment, perioperative management, and routine prophylaxis to reduce bleeding episodes.

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

NCT01775618CompletedPhase 3
Bayer
Posted 5/29/2013

A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood

NCT05147662CompletedPhase 3
Bayer
Posted 3/23/2022

AKANTIOR® Receives UK Marketing Authorization as First Approved Treatment for Acanthamoeba Keratitis

Orphan DrugDrug ApprovalRare Disease
  • SIFI's AKANTIOR® (polihexanide 0.08%) has received both Marketing Authorization and Promising Innovative Medicine designation from the UK's MHRA, marking it as the first approved treatment for Acanthamoeba keratitis.
  • The approval confirms AKANTIOR's Orphan Drug Designation and New Active Substance status, recognizing its efficacy against an ultra-rare corneal infection that can lead to blindness if untreated.
  • Following its European approval in August 2024, this UK authorization represents a significant advancement for patients with this devastating eye infection, with SIFI planning to file for NICE reimbursement by June 2025.

FDA Approves 18 New Personalized Medicines in 2024, Marking Significant Shift in Treatment Paradigm

Drug ApprovalOncologyRare DiseaseAIReal World EvidencePrecision Medicine
  • 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.

Taiwan Administers First Alzheimer's Drug Targeting Amyloid Plaques

Monoclonal AntibodyDrug Approval
  • Far Eastern Memorial Hospital in New Taipei City administered Taiwan's first dose of Kisunla (donanemab), a new Alzheimer's drug that targets underlying disease causes rather than just symptoms.
  • The monoclonal antibody treatment, developed by Eli Lilly, was given to an 83-year-old woman and represents a breakthrough approach to reducing amyloid plaques in the brain.
  • Both Kisunla and Leqembi require 12-18 months of treatment to significantly reduce amyloid accumulation, with fewer than 100,000 of Taiwan's 350,000 dementia patients eligible for these therapies.
  • While promising, experts caution these drugs are not cures and require ongoing monitoring, with long-term effectiveness and side effects still under evaluation.

FDA Approves Novavax COVID-19 Vaccine with Restricted Use for High-Risk Groups

Drug Approval
• The FDA has granted full approval for Novavax's protein-based COVID-19 vaccine, but with unusual restrictions limiting its use to adults 65 and older or those 12-64 with health risk factors.
• Novavax's vaccine represents the only traditional protein-based coronavirus vaccine option in the US market, previously available under emergency authorization for anyone 12 and older.
• The restricted approval comes amid shifting vaccine policies under the Trump administration, with the FDA requiring Novavax to conduct additional post-approval studies on potential heart conditions and benefits for certain age groups.

First Biologic Drug for Pulmonary Sarcoidosis in 50 Years Administered at Oxford Hospital

Rare DiseaseDrug Approval
• 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.

Cardioplexol™ Demonstrates Safety and Efficacy in Phase 3 Trial for Cardiac Surgery

Drug Approval
  • A pivotal phase 3 non-inferiority trial has confirmed the safety and efficacy of Cardioplexol™, a new low-volume cardioplegic solution, compared to standard Buckberg blood cardioplegia in cardiac surgeries.
  • Cardioplexol™ showed significant advantages including faster cardiac arrest (11 vs 71 seconds), reduced cross-clamp time, lower defibrillation rates (10% vs 52%), and decreased need for post-operative inotropic support.
  • Following successful clinical validation, Cardioplexol™ received marketing authorization in Switzerland in September 2023 and in 10 European countries in April 2024.

China Approves First Domestically Developed Enzyme Replacement Therapy for Gaucher Disease

Orphan DrugDrug ApprovalRare 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.

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