Clinical Trial News
FDA Approves Mylan's Generic Versions of Copaxone for Multiple Sclerosis Treatment
- The FDA has approved Mylan's glatiramer acetate injections in two dosage forms (40 mg/mL three-times-weekly and 20 mg/mL once-daily) as therapeutically equivalent alternatives to Teva's Copaxone for relapsing forms of multiple sclerosis.
- Mylan's generic versions demonstrated the same active ingredient, dosage form, route of administration, and strength as Copaxone, allowing them to be substituted with comparable safety and efficacy profiles.
- Alongside the medication launch, Mylan introduced the comprehensive MS Advocate support program offering injection training, 24/7 patient support, co-pay assistance, and ongoing nurse support to help patients adhere to their treatment plans.
Advancing Traumatic Brain Injury Treatment Through the TED Initiative
The Traumatic Brain Injury Endpoints Development (TED) Initiative, funded by the Department of Defense, aims to improve clinical trials for TBI by developing precise diagnostic tools and effective treatments. Over its first 18 months, TED has made significant progress, including engaging with the FDA, creating a comprehensive metadataset, and advancing biomarker and clinical outcome assessment validation.
SpringWorks Therapeutics Launches with $103M to Advance Pfizer's Rare Disease Portfolio
- SpringWorks Therapeutics emerges as a Pfizer spin-off with $103 million in funding from multiple investors, including Pfizer, LifeArc, and Bain Capital, to develop four promising rare disease drug candidates.
- The company's pipeline includes nirogacestat for desmoid tumors and a MEK 1/2 inhibitor for neurofibromatosis, both advancing to phase 3 trials, along with treatments for hereditary xerocytosis and PTSD.
- Led by former Pfizer executive Lara Sullivan, SpringWorks represents a novel model for accelerating drug development by revitalizing lower-priority assets through focused development and strategic partnerships.
Ibrutinib Shows Promise in Treating Chronic Graft-Versus-Host Disease
A study reveals that Ibrutinib, a Bruton tyrosine kinase inhibitor, has shown significant efficacy in treating chronic graft-versus-host disease (cGVHD) in patients who have failed corticosteroid therapy, with a high rate of sustained responses and acceptable safety profile.
Highlighted Clinical Trials:
Pharmacyclics LLC.
Posted 5/11/2017
Pharmacyclics LLC.
Posted 7/14/2014
Novartis's Kymriah: Breakthrough CAR-T Therapy Priced at $475,000 Sparks Value Debate
- Novartis's Kymriah, the first FDA-approved CAR-T cell therapy for pediatric acute lymphoblastic leukemia, demonstrates an impressive 83% remission rate in patients who failed traditional treatments.
- The therapy's $475,000 price tag has ignited debate among healthcare experts, with some defending it as comparable to bone marrow transplants while others argue it's excessive despite its breakthrough status.
- Novartis has implemented innovative value-based pricing models, including outcome-based contracts where payment is contingent upon patient response and indication-specific pricing for future approvals.
Catalyst Biosciences Announces Positive Factor IX Clinical Data
Catalyst Biosciences, Inc. has announced positive clinical data from the first Cohort of its ongoing Phase 1/2 proof-of-concept clinical trial for CB 2679d, a next-generation coagulation Factor IX variant, in individuals with severe hemophilia B. The data shows CB 2679d is approximately 22 times more potent than current hemophilia B therapy, with a longer circulation time in the body.
Phase 2 Trial Shows High Efficacy of BOVen Regimen in Treating Chronic Lymphocytic Leukaemia
A phase 2 trial investigating the BOVen regimen (zanubrutinib, obinutuzumab, and venetoclax) for treating chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL) has shown promising results, with 89% of patients achieving undetectable minimal residual disease (MRD) in both peripheral blood and bone marrow. The study highlights the regimen's safety and efficacy, suggesting a potential new standard for first-line therapy in CLL/SLL patients.
Highlighted Clinical Trials:
Memorial Sloan Kettering Cancer Center
Posted 2/22/2019
Atezolizumab, Bevacizumab, and GEMOX Combination Shows Promise in Advanced Biliary Tract Cancer
- Atezolizumab, bevacizumab, and GEMOX (gemcitabine plus oxaliplatin) demonstrated efficacy and safety in treating advanced biliary tract cancer (BTC).
- The combination therapy achieved a 76.7% overall response rate (ORR) and a 90.0% disease control rate (DCR) in a study of 30 patients.
- Median progression-free survival (PFS) was 12.0 months, and the median overall survival (OS) was not reached during the study period.
- Alterations in the ALS2CL gene may serve as a potential predictive biomarker for the efficacy of this triple combination therapy.
Strategic Approaches to Securing Market Access for Orphan Drugs: Beyond HTA Approval
- Clinician engagement and education are crucial for orphan drug adoption, requiring targeted materials that effectively communicate disease burden and clinical benefits.
- Real-world evidence collection through independent registries plays a vital role in demonstrating orphan drug effectiveness, despite manufacturer funding challenges.
- Patient support networks, including home nurse visits and online platforms, can enhance treatment outcomes while helping justify high orphan drug costs to payers.
Medical Affairs Evolution: Eisai's Kirk Shepard Leads Transformation Towards Patient-Centric Pharma
- Medical Affairs has evolved from a bridge between R&D and commercial teams to become central in driving patient-centric, data-driven pharmaceutical development and decision-making.
- Kirk Shepard, Eisai's SVP of Global Medical Affairs Oncology, spearheads the Medical Affairs Professional Society (MAPS) to establish global standards and elevate the profession's impact.
- Medical Science Liaisons serve as crucial field representatives, gathering real-world insights from healthcare providers and facilitating evidence-based discussions with stakeholders.