News

Eisai’s multiple receptor tyrosine kinase inhibitor lenvatinib and Merck’s anti–PD-1 therapy pembrolizumab as a combination therapy for patients with renal cell carcinoma received the designation based on results from the renal cell carcinoma cohort in Study 111.

BioMarin Chief Executive Officer Jean-Jacques Bienaime spoke about the company’s development of valoctocogene roxaparvovec, a gene therapy in development for the treatment of Hemophilia A.

The FDA has granted the combination of the PD-1 inhibitor pembrolizumab and the VEGF/FGF inhibitor lenvatinib a breakthrough therapy designation for the treatment of patients with advanced and/or metastatic renal cell carcinoma.

Global Blood Therapeutics, Inc. was granted Breakthrough Therapy Designation from the U.S. Food and Drug Administration for voxelotor for the treatment of sickle cell disease.

Last week, Pfizer announced a new partnership with Sangamo. As part of the agreement, the sides will team to develop a potential gene therapy to treat ALS, or Lou Gehrig’s disease.

While chemotherapy with thoracic radiation has been established as the standard of care for the initial treatment of non-metastatic small-cell lung cancer, a large proportion of patients do not receive these treatments and in turn have lower overall survival, according to a study published in JAMA Oncology.

Recent data have found that the same gene editing platform can disable the defective gene responsible for amyotrophic lateral sclerosis in mice. The therapy, which extended the lifespan of the mouse models by 25%, delayed the onset of the muscle wasting which characterizes the disease.

Spark Therapeutics CEO Jeff Marrazzo reassured patients and the medical community he has an obligation to ensure access to the novel gene therapy.

New vision loss gene therapy gets a price tag below the expected $1 million mark; new evidence finds that the prevalence of autism spectrum disorders has plateaued; hospitals are ill-equipped to care for dementia patients, but a new effort could change that.

The FDA has expanded the label for cabozantinib (Cabometyx) to include first-line treatment of advanced renal cell carcinoma regardless of treatment status. Cabozantinib was initially approved in patients who had previously received anti-angiogenic therapy.

Sagus Sampath, MD, discusses ongoing studies exploring checkpoint inhibitors with radiation therapy and the promise of integrating these 2 treatments in the field of non-small cell lung cancer.

The FDA has granted the PD-L1 inhibitor avelumab a breakthrough therapy designation for use in combination with the VEGF inhibitor axitinib in treatment-naïve patients with advanced renal cell carcinoma.

The combination therapy increased the attraction of immune cells to fight non-small cell lung cancer and also boosted the response to immune checkpoint inhibitors.

Universal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.

CAR T cells targeting CD19 can be effective at treating relapsed/refractory diffuse large B-cell lymphoma or follicular lymphoma, with high rates of durable remission.

The FDA approved Spark Therapeutics Inc.’s voretigene neparvovec-rzyl (Luxturna), the first gene therapy for inherited vision loss caused by faulty gene mutations.

Ghassan K. Abou-Alfa, MD, discusses what he sees coming down the pike in epatocellular carcinoma with regard to novel agents, including CAR T-cell therapy, and the burgeoning questions with optimal sequencing of these treatments.

This is the first gene therapy approved in the US targeting mutations in a specific gene.

Every week, The American Journal of Managed Care® recaps the top managed care news of the week, and you can now listen to it on our podcast, Managed Care Cast.

Marcela V. Maus, MD, PhD, evaluates the next generation of chimeric antigen receptor (CAR) T cells in both hematologic malignancies and solid tumors

Chimeric antigen receptor (CAR) T cells have hit prime time, with the 2 recent FDA approvals for this class of cell-based therapy.

Renova Therapeutics is planning a 536-patient phase 3 trial, FLOURISH, to begin in early 2018.

Researchers found that 2 naturally occurring molecules can make the process of cell generation faster and more efficient. This could have major implications for multiple sclerosis therapy research.

Maintenance therapy with elotuzumab (Empliciti) and lenalidomide (Revlimid) after autologous stem cell transplant improves the quality of response achieved with induction therapy in patients with multiple myeloma.

MMG49 has been identified as a monoclonal antibody that can be targeted using CAR T-cell therapy for patients with multiple myeloma.

A proof-of-concept study has demonstrated that resistance to treatment in multiple myeloma and mantle cell lymphoma could be linked to a protein called Nrf1, which appears to respond to proteasome insufficiency or pharmacological inhibition.

A much-anticipated session on the second day of the 59th Annual Meeting and Exposition of the American Society of Hematology provided long-term updates on trials evaluating 2 chimeric antigen receptor-T (CAR T) treatments: tisagenlecleucel or CTL019 (Kymriah) for the treatment of adult relapsed/refractory diffuse large B-cell lymphoma (DLBCL) and axicabtagene ciloleucel (Yescarta) evaluated in patients with refractory aggressive non-Hodgkin lymphoma.

Continuous lenalidomide therapy improves outcomes among patients with newly diagnosed myeloma who are not autologous stem cell transplant candidates, according to findings from a large phase III clinical trial.