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Chemotherapy-Free Regimens and CAR-T Therapies Transform B-Cell Malignancy Treatment Paradigms

CAR-TTargeted TherapyDrug Approval
  • Chemotherapy-free regimens are shifting treatment paradigms in mantle cell lymphoma, with BTK inhibitor-based therapies increasingly used for high-risk patients with TP53 mutations who respond poorly to traditional chemotherapy.
  • The FDA approval of lisocabtagene maraleucel for double-refractory chronic lymphocytic leukemia provides a new treatment option for patients who have failed both BTK inhibitors and BCL2 inhibitors like venetoclax.
  • Selective BTK inhibitors zanubrutinib and acalabrutinib are replacing ibrutinib in clinical practice, with combination regimens including venetoclax showing promising results in frontline CLL therapy.
  • Novel combination strategies including acalabrutinib plus venetoclax and triplet regimens with obinutuzumab are expanding treatment options for CLL patients, though regulatory approval is still pending.

Venetoclax, Ibrutinib, Prednisone, Obinutuzumab, and Revlimid (ViPOR) in Relapsed/Refractory B-cell Lymphoma

NCT03223610RecruitingPhase 1
National Cancer Institute (NCI)
Posted 2/9/2018

Tisagenlecleucel in Adult Patients With Aggressive B-cell Non-Hodgkin Lymphoma

NCT03570892Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 5/7/2019

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

Study Evaluating Safety and Efficacy of JCAR017 in Subjects With Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL)

NCT03331198RecruitingPhase 1
Juno Therapeutics, a Subsidiary of Celgene
Posted 11/27/2017

Comparison of the Treatments of Obinutuzumab + Venetoclax Versus Obinutuzumab + Chlorambucil in Patients With Chronic Lymphocytic Leukemia

NCT02242942Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 12/31/2014

Study of Acalabrutinib (ACP-196) in Combination With Venetoclax (ABT-199), With and Without Obinutuzumab (GA101) Versus Chemoimmunotherapy for Previously Untreated CLL

NCT03836261Active, Not RecruitingPhase 3
Acerta Pharma BV
Posted 2/25/2019

A Study to Compare the Efficacy and Safety of JCAR017 to Standard of Care in Adult Subjects With High-risk, Transplant-eligible Relapsed or Refractory Aggressive B-cell Non-Hodgkin Lymphomas

NCT03575351CompletedPhase 3
Celgene
Posted 10/23/2018

ASCT After a Rituximab/Ibrutinib/Ara-c Containing iNduction in Generalized Mantle Cell Lymphoma

NCT02858258RecruitingPhase 3
Prof. Dr. M. Dreyling (co-chairman)
Posted 7/1/2016

Study of Effectiveness of Axicabtagene Ciloleucel Compared to Standard of Care Therapy in Patients With Relapsed/Refractory Diffuse Large B Cell Lymphoma

NCT03391466CompletedPhase 3
Kite, A Gilead Company
Posted 1/25/2018

Tocilizumab Biosimilar CT-P47 Demonstrates Long-Term Efficacy and Safety in Rheumatoid Arthritis Treatment

Monoclonal AntibodyDrug Approval
  • CT-P47, a tocilizumab biosimilar, showed comparable efficacy and safety to reference Actemra over 52 weeks in a phase 3 trial involving 444 rheumatoid arthritis patients.
  • Patients who switched from Actemra to CT-P47 maintained similar disease control with no new safety concerns, supporting biosimilar interchangeability.
  • The biosimilar demonstrated successful self-administration via autoinjector with high patient satisfaction scores and minimal injection site reactions.
  • Multiple tocilizumab biosimilars have gained regulatory approval, offering potential cost savings of 16-26% compared to the originator biologic.

A Study to Compare Efficacy and Safety of CT-P47 and RoActemra in Patients With Rheumatoid Arthritis

NCT05489224CompletedPhase 3
Celltrion
Posted 9/14/2022

A Study to Evaluate Usability of Subcutaneous Auto-injector of CT-P47 in Patients With Active Rheumatoid Arthritis

NCT05725434Not Yet RecruitingPhase 3
Celltrion
Posted 2/6/2023

FDA Approves First Treatment for Alkaptonuria: Nitisinone Shows Clinical Benefits in Rare Metabolic Disorder

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA approved nitisinone (Harliku) as the first and only treatment for alkaptonuria, a rare genetic metabolic disorder affecting 1 in 250,000 to 1 million Americans.
  • A 3-year randomized clinical trial of 40 patients demonstrated that nitisinone improved pain, energy levels, and physical functioning compared to untreated controls.
  • Alkaptonuria causes buildup of homogentisic acid leading to joint problems, ochronosis, and complications in kidneys and heart, with the medication expected to launch in July 2025.
  • The approval represents a significant breakthrough for patients who previously had no approved therapeutic options for this debilitating condition.

Long-Term Study of Nitisinone to Treat Alkaptonuria

NCT00107783CompletedPhase 2
National Human Genome Research Institute (NHGRI)
Posted 1/1/2005

FDA Approves Tafasitamab Triple Combination for Relapsed/Refractory Follicular Lymphoma

Drug ApprovalMonoclonal Antibody
  • The FDA has approved tafasitamab-cxix (Monjuvi) in combination with lenalidomide and rituximab for adult patients with relapsed or refractory follicular lymphoma.
  • The phase 3 inMIND trial demonstrated a median progression-free survival of 22.4 months with the tafasitamab combination versus 13.9 months with placebo plus lenalidomide and rituximab.
  • This approval provides the first CD19- and CD20-targeted immunotherapy combination for this patient population, offering a chemotherapy-free treatment option.
  • Serious adverse effects were reported in 33% of patients receiving tafasitamab, with serious infections occurring in 24% of patients.

Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome

NCT04401748Active, Not RecruitingPhase 3
AbbVie
Posted 9/10/2020

A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

NCT02942290Active, Not RecruitingPhase 1
AbbVie
Posted 1/12/2017

Phase I/II Study of AD-PluReceptor Plus Tafasitamab-cxix and Lymphodepleting Chemotherapy in Patients With Autoimmune Disorders

NCT06434363RecruitingPhase 1
M.D. Anderson Cancer Center
Posted 7/31/2024

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

MInimal Residual Disease Adapted Strategy

NCT04934475Active, Not RecruitingPhase 3
Intergroupe Francophone du Myelome
Posted 12/8/2021

Tafasitamab and Zanubrutinib for the Treatment of Patients With Newly Diagnosed Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma, TaZA CLL Study

NCT05718869RecruitingPhase 2
City of Hope Medical Center
Posted 5/18/2023

A Phase 3 Study to Assess Efficacy and Safety of Tafasitamab Plus Lenalidomide and Rituximab Compared to Placebo Plus Lenalidomide and Rituximab in Patients With Relapsed/Refractory (R/R) Follicular Lymphoma or Marginal Zone Lymphoma.

NCT04680052Active, Not RecruitingPhase 3
Incyte Corporation
Posted 4/15/2021

Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

NCT02598661Active, Not RecruitingPhase 2
Geron Corporation
Posted 1/12/2016

Talazoparib + Enzalutamide vs. Enzalutamide Monotherapy in mCRPC

NCT03395197Active, Not RecruitingPhase 3
Pfizer
Posted 12/18/2017

BrightSpring's Onco360 Pharmacy Selected as National Partner for Three New Cancer and Rare Disease Therapies

OncologyRare DiseaseDrug ApprovalTargeted Therapy
  • BrightSpring Health Services announced that its specialty pharmacy Onco360 has been selected as the national pharmacy partner for three newly approved therapies targeting advanced cancers and rare genetic disorders.
  • The partnership includes GOMEKLI for neurofibromatosis type 1 patients with plexiform neurofibromas, AVMAPKI FAKZYNJA CO-PACK for KRAS-mutated recurrent low-grade serous ovarian cancer, and IBTROZI for ROS1-positive non-small cell lung cancer.
  • Onco360 will provide comprehensive patient support including access, education, data management, and expert clinical guidance for these innovative treatments across all 50 states.

New Zealand Approves Over-the-Counter Melatonin and Prescription Psilocybin for Treatment-Resistant Depression

Drug Approval
  • New Zealand's MedSafe has approved melatonin for over-the-counter sale in pharmacies, making it available in doses up to 5mg for treating insomnia and jet lag.
  • Psilocybin will become available as an unapproved medicine for patients with treatment-resistant depression under the supervision of one experienced psychiatrist.
  • The psilocybin approval marks the first time the psychedelic compound will be legally available in New Zealand outside of clinical trials.
  • Both regulatory changes represent significant shifts in New Zealand's approach to alternative therapeutic options for sleep disorders and mental health conditions.

Bavarian Nordic Sells Priority Review Voucher for $160 Million Following Chikungunya Vaccine Approval

Drug ApprovalFDA Approval
  • Bavarian Nordic announced the sale of its Priority Review Voucher for $160 million, awarded following FDA approval of its chikungunya vaccine VIMKUNYA in February 2025.
  • The transaction is expected to close in Q3 2025 and will positively impact the company's EBITDA, with the National Institutes of Health receiving 20% of proceeds under a licensing agreement.
  • The sale reflects an uptick in PRV values in 2025, with recent transactions ranging from $150-160 million compared to the historical average of around $100 million.
  • Priority Review Vouchers reduce FDA drug review times from ten months to six months and are awarded for treatments addressing neglected tropical diseases, rare pediatric conditions, or material threat medical countermeasures.

Health Canada Approves Expanded Dosing and Pediatric Indication for FIRDAPSE in Lambert-Eaton Myasthenic Syndrome

Drug ApprovalRare Disease
  • Health Canada has approved Kye Pharmaceuticals' supplemental new drug submission for FIRDAPSE (amifampridine), allowing some patients with Lambert-Eaton myasthenic syndrome to benefit from an increased maximum daily dose of up to 100mg.
  • The expanded approval includes pediatric dosing guidance for patients 6 years of age and older, providing pediatric neurologists with a proven treatment option for children diagnosed with LEMS.
  • FIRDAPSE is a potassium channel blocker that works by increasing acetylcholine release at the neuromuscular junction, helping improve muscle function in patients with this rare autoimmune disorder.
  • The label update offers healthcare providers and patients greater flexibility in treatment regimens for managing LEMS, a condition characterized by muscle weakness and fatigue.

Sanofi's Tzield Debuts in China as First FDA-Approved Therapy to Delay Type 1 Diabetes Onset

Drug Approval
  • Sanofi's Tzield, the world's first FDA-approved therapy for postponing type 1 diabetes onset, has been prescribed in Boao, Hainan province, marking its debut among the first batch in Asia.
  • The innovative treatment delays progression from stage 2 to stage 3 type 1 diabetes in patients aged 8 or older, with 14-day continuous usage capable of postponing onset by nearly three years.
  • This breakthrough represents a shift from passive treatment to active intervention for type 1 diabetes in China, protecting pancreatic islet function and allowing patients symptom-free time without insulin therapy.
  • China has approximately 600,000 type 1 diabetes patients, ranking third globally, with high incidence among children and adolescents aged 10-14.

Canada Outpaces OECD Countries in New Medicine Launches Despite Rising Treatment Costs

Drug Approval
  • Canada launched more new medicines than the median for OECD countries in 2022-2023, according to the PMPRB's 9th annual Meds Entry Watch report.
  • Most new medicines entering the Canadian market carry high treatment costs exceeding $10,000 per year or $5,000 per 28-day cycle for oncology treatments.
  • Specialty medicines including biologics, orphan drugs, and cancer treatments represent a growing share of the new drug landscape in Canada.
  • The report analyzed 48 medicines receiving first-time market approval in 2022 and provided preliminary analysis of 63 medicines approved in 2023.

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