FDA Granted Approvals for Myeloma and Alzheimer's Drugs in July, Yet Dismissed Two Cellular Gene Therapies
Regeneron's Lynozyfic Approved for Multiple Myeloma: A New Standard of Care?
In a significant development for the pharmaceutical industry, Regeneron's Lynozyfic (linvoseltamab) has received accelerated approval from the FDA for treating adults with relapsed or refractory multiple myeloma (RRMM) who have failed at least four prior treatments. This approval marks an important new option for hard-to-treat multiple myeloma patients [1][3][5].
Regeneron's CEO, George Yancopoulos, has expressed optimism about Lynozyfic, stating that it could potentially become a new standard of care for multiple myeloma. The drug is dosed every two weeks, with the possibility of extending to every four weeks if a patient achieves very good partial response or better [1].
Meanwhile, Eli Lilly's Alzheimer's disease therapy, Kisunla, has received a label expansion from the FDA. The new dosing regimen aims to improve safety in Alzheimer's treatment, based on the TRAILBLAZER-ALZ 6 study, which showed a lower risk of amyloid-related imaging abnormalities indicative of fluid accumulation (ARIA-E) [5].
Moderna's COVID-19 vaccine, Spikevax, has also received full FDA approval for children aged 6 months through 11 years at high risk of infection. Additionally, its next-generation COVID-19 vaccine, mNEXSPIKE, was approved for older adults (65+) and at-risk individuals 12-64, and the RSV vaccine, mResvia, received broader approval for at-risk adults 18-59 [5].
Bayer's Kerendia, initially approved in July 2021, has received a new indication from the FDA. The drug is now indicated to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent visits for heart failure in adult heart failure patients with left ventricular ejection fraction of at least 40% [6].
In the pharmaceutical world, regulatory approvals and label expansions are a testament to the progress being made in treating various diseases. These recent milestones, including Regeneron's Lynozyfic, Eli Lilly's Kisunla, and Moderna's expanded COVID-19 vaccine approvals, stand out as key developments in their respective therapeutic areas [1][3][5].
Other notable approvals this month include the first-ever on-demand oral therapy for hereditary angioedema, KalVista's sebetralstat (Ekterly), and Bayer's Kerendia, which demonstrated a significant 16% reduction in the primary endpoint of the Phase III FINEARTS-HF study [6][7].
However, not all applications were successful. The FDA turned away a cell therapy for Duchenne muscular dystrophy and a gene therapy for the rare disease Sanfilippo syndrome [8].
References:
- Regeneron's Lynozyfic Approved for Multiple Myeloma
- GSK Enters Licensing Agreement for Blood-Brain Barrier Shuttle Platform
- Regeneron's Lynozyfic: A Potential New Standard of Care for Multiple Myeloma
- FDA Approves Moderna's Spikevax for Children at High Risk of Infection
- Moderna's COVID-19 Vaccine Approval Expansions
- Bayer's Kerendia Granted New Indication for Heart Failure Patients
- Kerendia Shows Significant Reduction in Heart Failure Events and Cardiovascular Death
- FDA Rejects Cell Therapy for Duchenne Muscular Dystrophy and Gene Therapy for Sanfilippo Syndrome
In light of Regeneron's Lynozyfic receiving accelerated approval for treating multiple myeloma, the role of science in addressing chronic diseases like this is becoming increasingly prominent. Moreover, the potential of Lynozyfic to establish a new standard of care for multiple myeloma patients underscores the significance of medical-condition specific therapies and treatments in health-and-wellness management.
