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 received at least three prior lines of…
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 than white individuals — even when…
Even if they’re fully vaccinated, multiple myeloma patients still have an increased risk of COVID-19 infection compared with people without cancer, according to a study looking at data from more than 500,000 individuals in the U.S. Called breakthrough infections, these post-vaccination illnesses from SARS-CoV-2 — the virus that causes COVID-19 — also were associated with…
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 many registries exist in the U.S.…
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 and low-dose dexamethasone in patients with relapsed and refractory multiple myeloma…
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 several factors, including the social determinants of…
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 which available treatments fall short. It is…
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 Window Into RARE,” was hosted by Liza Bernstein,…
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 of therapy, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD),…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that burden, estimated at nearly $1 trillion,…
