News

Europe’s CHMP Supports Carvykti T-cell Therapy Approval in EU

Carvykti (ciltacabtagene autoleucel; cilta-cel) has been recommended for approval in Europe by the Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), for eligible adults with relapsed and refractory multiple myeloma. The CAR T-cell therapy will be available for patients who have…

Longer Survival for Black vs White Myeloma Patients With Same Care

Black people with multiple myeloma live longer than white patients with similar disease symptoms when both receive the same new and updated medical treatments, according to an analysis of the Surveillance, Epidemiology, and End Results (SEER) database. However, Black myeloma patients were less likely to receive such novel treatments…

RaDaR Serves as Starting Point for Creating Rare Disease Registry

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…

Sarclisa Extends Survival in RRMM, Long-term Phase 3 Trial Data Shows

The therapy Sarclisa (isatuximab) extended survival by nearly seven months in relapsed/refractory multiple myeloma patients also given Pomalyst (pomalidomide) and dexamethasone as standard treatment, according to updated three-year data from the ICARIA-MM Phase 3 trial. The data, “Isatuximab plus pomalidomide and low-dose dexamethasone versus pomalidomide…

#RAREis Representation Program Promotes Equity, Diversity

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…

FDA Places HPN217, Immunotherapy for Advanced Disease, on Fast Track

The U.S. Food and Drug Administration (FDA) has given fast track designation to Harpoon Therapeutics’ HPN217 as a potential immunotherapy for people with relapsed, refractory multiple myeloma (RRMM) who have tried at least four prior therapies. Fast track status is given to therapies that show potential in addressing serious conditions for…

Rare Disease Day Panel Opens Window to Patient Experience

BioNews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…

FDA Approves Cilta-cel as Carvykti for Heavily Treated Myeloma

The U.S. Food and Drug Administration (FDA) has approved the CAR T-cell therapy Carvykti (ciltacabtagene autoleucel) — formerly known as cilta-cel — for certain adults with relapsed or refractory multiple myeloma. In particular, the approval makes Carvykti available for people who have received at least four prior lines…