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UK Approves World's First CRISPR Gene Therapy for Sickle Cell Disease and β-Thalassemia

Drug ApprovalRare Disease
  • The UK has made a landmark decision to approve the world's first CRISPR-based gene therapy, developed by Vertex Pharmaceuticals, for treating sickle cell disease and β-thalassemia.
  • This one-time treatment offers potential transformation of patient lives by addressing the genetic root cause of these blood disorders, marking a historic milestone in gene editing technology.
  • Despite its therapeutic promise, concerns remain about the high cost of the treatment and questions about accessibility for patients in need, particularly in regions with high disease prevalence.

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880RecruitingPhase 1
Beam Therapeutics Inc.
Posted 8/30/2022

Biocon Biologics Secures UK Approval for Aflibercept Biosimilar YESAFILI, Expanding European Market Access

Drug Approval
  • Biocon Biologics received MHRA approval for YESAFILI, a biosimilar of aflibercept, for treating multiple retinal conditions in the UK market.
  • The approval follows European Commission authorization in September 2023, demonstrating comparable quality, safety, and efficacy to reference product Eylea.
  • YESAFILI targets a significant market opportunity with aflibercept generating $790 million in UK sales as of June 2023.
  • The biosimilar expands Biocon's ophthalmology portfolio and strengthens its global presence beyond oncology and diabetes therapeutics.

FDA Approves Leqvio as First-Line Monotherapy for Cholesterol Management

Drug ApprovalReal World Evidence
  • The FDA has approved Novartis' Leqvio (inclisiran) for use as monotherapy alongside diet and exercise to reduce LDL cholesterol in adults with hypercholesterolemia, removing the previous requirement for concurrent statin therapy.
  • The twice-yearly injectable treatment addresses a critical unmet need, as up to 80% of atherosclerotic cardiovascular disease patients fail to reach guideline-recommended LDL cholesterol targets below 70 mg/dL.
  • Real-world data from the V-INITIATE trial demonstrated that early addition of Leqvio to statin therapy achieved 60% LDL cholesterol reduction compared to 7% with usual care, with 81.8% of patients reaching target levels versus 22.2% in the control group.

Inclisiran for Subjects With ASCVD or ASCVD-Risk Equivalents and Elevated Low-density Lipoprotein Cholesterol

NCT03400800CompletedPhase 3
The Medicines Company
Posted 11/1/2017

A Randomized Study to Evaluate the Effect of an "Inclisiran First" Implementation Strategy Compared to Usual Care in Patients With Atherosclerotic Cardiovascular Disease and Elevated LDL-C Despite Receiving Maximally Tolerated Statin Therapy (VICTORION-INITIATE)

NCT04929249CompletedPhase 3
Novartis Pharmaceuticals
Posted 6/25/2021

Inclisiran for Participants With Atherosclerotic Cardiovascular Disease and Elevated Low-density Lipoprotein Cholesterol

NCT03399370CompletedPhase 3
The Medicines Company
Posted 12/21/2017

Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)

NCT03397121CompletedPhase 3
The Medicines Company
Posted 11/28/2017

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C

NCT03814187CompletedPhase 3
Novartis Pharmaceuticals
Posted 4/16/2019

Cuba Advances Phase III Clinical Trial of Jusvinza for Rheumatoid Arthritis Treatment

Drug Approval
  • Cuban scientists are conducting a Phase III clinical trial of Jusvinza, an immunomodulatory and anti-inflammatory drug, in rheumatoid arthritis patients across the country.
  • The drug received sanitary registration from Cuban regulatory authorities in September after demonstrating quality, safety, and efficacy in treating rheumatoid arthritis.
  • Patients in the trial have shown improved quality of life with no adverse effects reported in the first four doses of treatment.
  • Camagüey province leads enrollment with 212 volunteers, with participants receiving monthly doses for six months after completing the initial four-dose regimen.

FDA Approves Bosutinib for Pediatric Chronic Myelogenous Leukemia Patients

Drug Approval
  • The FDA has approved bosutinib (Bosulif) for pediatric patients aged 1 year or older with Philadelphia chromosome-positive chronic-phase chronic myelogenous leukemia.
  • The approval covers both newly diagnosed patients and those with disease resistant or intolerant to previous therapy.
  • Clinical trial data showed major cytogenetic response rates of 76.2% in newly diagnosed patients and 82.1% in resistant/intolerant patients.
  • The FDA also approved new 50 mg and 100 mg capsule formulations to facilitate pediatric dosing.

Bosutinib in Pediatric Patients With Newly Diagnosed Chronic Phase or Resistant/Intolerant Ph + Chronic Myeloid Leukemia

NCT04258943Active, Not RecruitingPhase 1
Children's Oncology Group
Posted 7/6/2020

Bispecific T-Cell Engagers Show Promise as Later-Line Therapies for Relapsed/Refractory DLBCL

CAR-TDrug ApprovalOncology
  • Two bispecific T-cell engagers, epcoritamab-bysp and glofitamab-gxbm, received FDA accelerated approval in 2023 for relapsed/refractory diffuse large B-cell lymphoma, offering new treatment options for heavily pretreated patients.
  • Both agents demonstrate remarkable activity with overall response rates above 50-60% and complete response rates around 40% in patients with CAR T-cell therapy failure, addressing a significant unmet medical need.
  • Epcoritamab offers convenient subcutaneous administration in outpatient settings with manageable cytokine release syndrome rates below 3%, while glofitamab provides fixed-duration therapy with durable responses lasting over one year after treatment completion.
  • These bispecific antibodies may challenge the paradigm that CAR T-cell therapy is the only curative option for relapsed/refractory patients, with emerging data suggesting potential for long-term disease control.

First-in-Human (FIH) Trial in Patients With Relapsed, Progressive or Refractory B-Cell Lymphoma

NCT03625037Active, Not RecruitingPhase 1
Genmab
Posted 6/26/2018

A Dose Escalation Study of Glofitamab (RO7082859) as a Single Agent and in Combination With Obinutuzumab, Administered After a Fixed, Single Pre-treatment Dose of Obinutuzumab in Participants With Relapsed/Refractory B-cell Non-hodgkin's Lymphoma

NCT03075696RecruitingPhase 1
Hoffmann-La Roche
Posted 2/21/2017

Lupin Receives FDA Approval for Generic Bromfenac Ophthalmic Solution for Post-Cataract Surgery Treatment

Drug Approval
  • Lupin Limited received FDA approval for its abbreviated new drug application (ANDA) for bromfenac ophthalmic solution 0.09%, a generic equivalent of Bausch + Lomb's Bromday ophthalmic solution.
  • The topical NSAID is indicated for treating postoperative inflammation and reducing ocular pain following cataract surgery, with a dosing regimen of one drop daily starting one day before surgery through 14 days post-surgery.
  • The generic product will be manufactured at Lupin's Pithampur facility in India and targets an estimated annual US market worth $11 million according to IQVIA data.
  • The approval enables Lupin to market a lower-cost alternative to the brand-name drug without requiring costly clinical trials, as the generic demonstrated bioequivalence to the original formulation.

European Regulators Recommend Cabometyx for Radioactive Iodine-Refractory Thyroid Cancer Based on 78% Reduction in Disease Progression

Drug Approval
  • The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Cabometyx (cabozantinib) for treating radioactive iodine-refractory differentiated thyroid cancer patients who have progressed after prior systemic therapy.
  • The recommendation is based on the COSMIC-311 Phase III trial, which demonstrated a 78% reduction in risk of disease progression or death versus placebo (HR: 0.22; 96% CI: 0.13-0.36; p<0.0001) with a median progression-free survival of 11.0 versus 1.9 months.
  • This positive opinion addresses a critical unmet medical need, as there are currently no standard-of-care treatment options for patients with radioactive iodine-refractory differentiated thyroid cancer who progress after first-line therapy.
  • The CHMP recommendation follows the FDA's approval of Cabometyx in September 2021 for the same indication in adult and pediatric patients 12 years and older.

First Veteran Receives FDA-Approved Qalsody for SOD1-ALS at Houston VA Medical Center

NeurologyRare DiseaseDrug ApprovalFDA Approval
  • Roy Swearingen, a 65-year-old Marine Corps veteran from Texas, became the first veteran in the United States to receive Qalsody, an FDA-approved medication for SOD1-mutated ALS.
  • Qalsody represents the first approved treatment targeting a genetic cause of ALS, specifically for patients with mutations in the superoxide dismutase 1 (SOD1) gene, affecting 1-2% of ALS patients.
  • The Houston VA ALS Center's rapid implementation of this cutting-edge therapy demonstrates the VA's commitment to providing veterans with timely access to innovative treatments.
  • Veterans face higher ALS risk compared to the general population, making early detection and specialized care crucial for optimal quality of life management.

Vorasidenib Shows Promise as First Targeted Therapy for IDH-Mutant Low-Grade Gliomas

Targeted TherapyDrug ApprovalOncology
  • Vorasidenib, the first targeted therapy developed specifically for brain cancer, more than doubled progression-free survival in patients with recurrent grade 2 glioma carrying IDH1/IDH2 mutations, extending the time without disease progression from 11.1 months to 27.7 months.
  • The international INDIGO trial involving 331 patients demonstrated that vorasidenib delayed the need for chemotherapy and radiation by nearly 17 months compared to placebo, with 85.6% of patients going 18 months before requiring next treatment.
  • The drug showed excellent tolerability with limited adverse effects, offering a new treatment option for younger patients typically in their 30s and 40s who face cognitive deficits from standard radiation and chemotherapy treatments.
  • Results from this phase 3 study, published in the New England Journal of Medicine and presented at ASCO, are expected to establish a new standard of care for IDH-mutant low-grade gliomas pending FDA approval.

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