European Medicine Agency (EMA)
This week, the European Medicines Agency (EMA) and the European Commission have approved new drugs for specific indications:
ELREXFIO® (elranatamab) by Pfizer: This drug has been approved for the treatment of relapsed and refractory multiple myeloma. ELREXFIO is a bispecific antibody immunotherapy delivered subcutaneously. It targets BCMA on myeloma cells and CD3 on T-cells, thus activating T-cells to kill myeloma cells. The approval is based on the results from the MagnetisMM-3 study, which also established a dosing schedule for the drug. Pfizer Press Release
ZILBRYSQ® (zilucoplan) by UCB: Zilucoplan has been approved for the treatment of adults with generalized Myasthenia Gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive. It is the first once-daily subcutaneous C5 inhibitor approved for self-administration. The approval is supported by safety and efficacy data from the RAISE study. UCB’s Press Release
Imfinzi (durvalumab) by AstraZeneca: The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a new indication for Imfinzi. It is now indicated for the first line treatment of adults with advanced or unresectable hepatocellular carcinoma (HCC). Imfinzi is already indicated for several other types of cancer, including non-small cell lung cancer, small cell lung cancer, and biliary tract cancer.CMHP Positive Opinion
US Food and Drug Administration (FDA)
The FDA approved the following new drugs this week:
Casgevy (exagamglogene autotemcel [exa-cel]): Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, this drug is a CRISPR/Cas9 genome-edited cell therapy approved for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). This treatment involves editing a patient’s own stem cells to produce more fetal hemoglobin and transplanting them back, which helps in preventing red blood cells from assuming a sickle shape, thus reducing the complications associated with SCD. FDA ’s Press Release
Lyfgenia (lovotibeglogene autotemcel, also known as lovo-cel): Developed by bluebird bio, this cell-based gene therapy is approved for the treatment of sickle cell disease in patients aged 12 and older who have a history of vaso-occlusive events (VOEs). Similar to Casgevy, it involves modifying stem cells to prevent red blood cells from becoming sickle-shaped. However, there is a black box warning for the risk of blood cancer among patients who receive this therapy. Bluebirdbio’s Press Release
CRESEMBA® (isavuconazonium sulfate): Astellas Pharma US, Inc. announced the FDA’s approval of CRESEMBA for the treatment of invasive aspergillosis (IA) and invasive mucormycosis (IM) in pediatric patients. This drug is an azole antifungal medication. Astella’s Press Release
Safety Surveillance PRAC and FDA
In a less glamorous, the PRAC (Pharmacovigilance Risk Assessment Committee) added the following drugs to the under-surveillance list:
- Ebglyss
- Herwenda
- Jaypirca
- Vanflyta
- Yorvipath
Others got cleared, for more details, see the last PRAC press release: PRAC 29 Nov 2023.
Recently, the FDA issued the following alert: potential serious reactions linked to an antiseizure medication: levetiracetam. The agency warned of a rare but serious reaction known as Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS), which can be life-threatening if not treated promptly. The reaction typically starts as a rash but can quickly progress and cause damage to organs. FDA warning levetiracetam 11 Nov 2023