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FDA Approves Breakthrough Twice-Yearly HIV Prevention Drug Lenacapavir with Nearly 100% Efficacy

Drug Approval
  • The FDA has approved lenacapavir, a breakthrough preventative treatment for HIV that offers nearly 100 percent protection from the virus and could change the course of the AIDS epidemic.
  • The new drug represents a significant advancement in HIV prevention with its twice-yearly dosing schedule, potentially improving patient adherence compared to daily prevention medications.
  • Despite the promising therapeutic breakthrough, deep cuts to health initiatives could hinder the rollout of this potentially game-changing HIV prevention treatment.

FDA Approves Zanubrutinib for Waldenström's Macroglobulinemia Treatment

Drug ApprovalRare Disease
  • The FDA has approved zanubrutinib (Brukinsa) for treating adult patients with Waldenström's macroglobulinemia, marking the second therapy specifically approved for this rare lymphoma.
  • The approval was based on the phase 3 ASPEN trial comparing zanubrutinib to ibrutinib, showing a higher very good partial response rate of 28% versus 19% respectively.
  • Zanubrutinib demonstrated improved tolerability compared to the first-generation BTK inhibitor ibrutinib across several clinically important side effects.
  • The drug is administered orally at either 160 mg twice daily or 320 mg once daily, offering patients a convenient single-agent treatment option.

A Study Comparing BGB-3111 and Ibrutinib in Participants With Waldenström's Macroglobulinemia (WM)

NCT03053440CompletedPhase 3
BeiGene
Posted 1/25/2017

UK Approves First COVID-19 Monoclonal Antibody Treatment as AstraZeneca Reports 77% Efficacy in Prevention Trial

Monoclonal AntibodyDrug Approval
  • The UK's MHRA approved Ronapreve, the first monoclonal antibody therapy specifically designed to treat and prevent COVID-19, developed by Regeneron and Roche.
  • The treatment combines two monoclonal antibodies administered by injection or infusion, working by binding to the virus and preventing respiratory system entry.
  • AstraZeneca simultaneously announced that its AZD7442 antibody combination reduced COVID-19 symptom risk by 77% in a trial of 5,172 participants, with over three-quarters having comorbidities affecting vaccine response.
  • Both treatments represent significant advances for vulnerable populations who may not respond adequately to COVID-19 vaccines alone.

FDA Approves Aduhelm: First Disease-Modifying Alzheimer's Treatment in Nearly Two Decades

Drug ApprovalMonoclonal AntibodyNeurology
  • The FDA granted accelerated approval to Biogen's aducanumab (Aduhelm) as the first disease-modifying Alzheimer's treatment in nearly two decades, despite divided expert opinions on its clinical efficacy.
  • Aduhelm, priced at $56,000 annually, works by reducing amyloid plaques in the brain and will require monthly intravenous infusions with comprehensive clinical and MRI monitoring for potential adverse effects.
  • As part of the accelerated approval, Biogen must conduct a Phase 4 confirmatory trial, while experts view this approval as a potential catalyst for developing more effective Alzheimer's treatments targeting multiple pathways.

221AD302 Phase 3 Study of Aducanumab (BIIB037) in Early Alzheimer's Disease

NCT02484547TerminatedPhase 3
Biogen
Posted 9/15/2015

221AD301 Phase 3 Study of Aducanumab (BIIB037) in Early Alzheimer's Disease

NCT02477800TerminatedPhase 3
Biogen
Posted 8/13/2015

A Study to Evaluate Safety and Tolerability of Aducanumab in Participants With Alzheimer's Disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205

NCT04241068TerminatedPhase 3
Biogen
Posted 3/2/2020

Multiple Dose Study of Aducanumab (BIIB037) (Recombinant, Fully Human Anti-Aβ IgG1 mAb) in Participants With Prodromal or Mild Alzheimer's Disease

NCT01677572TerminatedPhase 1
Biogen
Posted 10/5/2012

A Study of Aducanumab in Participants With Mild Cognitive Impairment Due to Alzheimer's Disease or With Mild Alzheimer's Disease Dementia to Evaluate the Safety of Continued Dosing in Participants With Asymptomatic Amyloid-Related Imaging Abnormalities

NCT03639987TerminatedPhase 2
Biogen
Posted 12/20/2018

FDA Approves Monjuvi in Combination with Lenalidomide for Relapsed/Refractory DLBCL

Drug ApprovalMonoclonal AntibodyOncology
  • The FDA has approved Monjuvi (tafasitamab-cxix) in combination with lenalidomide as the first second-line treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma who are not eligible for autologous stem cell transplant.
  • The approval was based on the Phase 2 L-MIND study, which demonstrated a 55% overall response rate with a 37% complete response rate and median duration of response of 21.7 months.
  • Approximately 10,000 patients are diagnosed annually in the United States with relapsed or refractory DLBCL who are not eligible for stem cell transplant, representing a significant unmet medical need.
  • Monjuvi is a humanized CD19-targeting monoclonal antibody that received accelerated approval and will be co-commercialized by MorphoSys and Incyte in the United States.

Open Label Study to Evaluate the Safety and Efficacy of Lenalidomide With MOR00208 in Patients With R-R DLBCL

NCT02399085CompletedPhase 2
MorphoSys AG
Posted 3/29/2016

Pertuzumab Regimen Shows Modest Survival Benefit in HER2-Positive Early Breast Cancer

Monoclonal AntibodyOncologyDrug ApprovalTargeted Therapy
• The APHINITY trial's 6-year analysis reveals that adjuvant pertuzumab with trastuzumab plus chemotherapy improved overall survival by 0.9% in HER2-positive early breast cancer patients, though not reaching statistical significance.
• Patients with node-positive disease showed the most significant benefit from the pertuzumab regimen, with a 4.5% absolute improvement in invasive disease-free survival at 6 years compared to placebo.
• The pertuzumab combination maintained a favorable cardiac safety profile with severe cardiac events occurring in less than 1% of patients, supporting its continued use in high-risk HER2-positive early breast cancer.

China Grants Conditional Approval to Seaweed-Derived Alzheimer's Drug Oligomannate

Drug Approval
  • Chinese regulators have granted conditional approval to Oligomannate, a seaweed-derived drug for mild to moderate Alzheimer's disease, marking the first new Alzheimer's therapy approval in nearly two decades.
  • The drug, developed by Shanghai Green Valley Pharmaceuticals, showed cognitive improvements in a Phase 3 trial of 818 patients with benefits observed as early as four weeks.
  • Unlike traditional approaches targeting brain protein buildup, Oligomannate works by modulating the gut microbiome to reduce neuroinflammation associated with Alzheimer's disease.
  • The company plans to launch a global Phase 3 trial in 2020 with sites in the United States, Europe, and Asia to seek international regulatory approval.

FDA Approves First Treatment for Primary Hemophagocytic Lymphohistiocytosis, Marking 24-Year Breakthrough

Monoclonal AntibodyDrug ApprovalRare DiseaseOrphan Drug
  • The FDA has approved emapalumab-lzsg (Gamifant) as the first treatment specifically indicated for primary hemophagocytic lymphohistiocytosis (HLH), an ultra-rare and life-threatening immune disorder.
  • In a pivotal phase 2/3 study of 27 patients with refractory disease, 63% demonstrated overall response and 70% proceeded to hematopoietic stem cell transplant.
  • The monoclonal antibody targets interferon gamma, representing the first significant improvement in primary HLH induction therapy in 24 years.
  • Primary HLH typically affects children within the first year of life and has a median survival of less than two months without treatment.

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

NCT01818492CompletedPhase 2
Swedish Orphan Biovitrum
Posted 7/1/2013

Novartis's Kymriah: Breakthrough CAR-T Therapy Priced at $475,000 Sparks Value Debate

CAR-TDrug ApprovalOncology
  • 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.

FDA Approves Two New Ustekinumab Biosimilars, Bringing Total US Biosimilar Count to 65

Monoclonal AntibodyDrug Approval
  • The FDA has approved Biocon's Yesintek (ustekinumab-kfce) and Celltrion's Steqeyma (ustekinumab-stba) in November and December 2024, expanding treatment options for autoimmune conditions including Crohn's disease and psoriasis.
  • Of the 65 FDA-approved biosimilars, 14 have received interchangeable designation, allowing pharmacists to substitute them for reference products without prescriber intervention, enhancing patient access to more affordable treatments.
  • The biosimilar landscape continues to evolve with products spanning five major classes: insulin, granulocyte colony-stimulating factors, monoclonal antibodies, TNF-alpha inhibitors, and VEGF inhibitors.

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