DDInter1840 DDInter1341
Trabectedin, also referred as ET-743 during its development, is a marine-derived antitumor agent discovered in the Carribean tunicate _Ecteinascidia turbinata_ and now produced synthetically. Trabectedin has a unique mechanism of action. It binds to the minor groove of DNA interfering with cell division and genetic transcription processes and DNA repair machinery. It is approved for use in Europe, Russia and South Korea for the treatment of advanced soft tissue sarcoma. It is currently under evaluation for the treatment of breast cancer, prostate cancer, in addition to pediatric sarcomas. Both the European Commission and the U.S. Food and Drug Administration (FDA) have approved trabectedin as an orphan drug in soft tissue sarcomas and ovarian cancer. On October 23, 2015, the FDA approved trabectedin, (as Yondelis), for the treatment of specific soft tissue sarcomas.
Onasemnogene abeparvovec is an adeno-associated virus vector-based gene therapy that has been approved by the FDA in May 2019 for the treatment of infant patients (less than 2 years of age) with spinal muscular atrophy (SMA) and a specific mutation in the survival motor neuron 1 (SMN1) gene. SMA is a rare genetic disease that affects the survival and function of motor neurons, leading to debilitating and often fatal muscle weakness. As there is no cure for SMA, onasemnogene abeparvovec is a disease-modifying agent that decelerates the disease progression, improves motor function, and manages the symptoms. The use and effectiveness of onasemnogene abeparvovec in patients with advanced SMA, such as those with complete paralysis of the limbs and permanent dependence on ventilators, has not been evaluated. Onasemnogene abeparvovec is the first gene therapy that was approved for this indication in the USA. [Nusinersen] is another gene therapy that is currently approved by the FDA for the treatment of SMA in pediatric and adult patients. Developed by AveXis, a Novartis company, onasemnogene abeparvovec is commonly marketed as Zolgensma®, which is available as a single-dose intravenous infusion. Onasemnogene abeparvovec for therapeutic use and marketing is currently being assessed by the EU and an intrathecal formulation of the drug is currently undergoing clinical development in the USA.
Trabectedin {u} (Compound) binds {v} (Gene) SRC
Trabectedin {u} (Compound) is included by {v} (Pharmacologic Class) Alkylating Activity
Trabectedin {u} (Compound) causes {v} (Side Effect) Alanine aminotransferase increased
Trabectedin (Compound) binds SRC (Gene)
Trabectedin (Compound) is included by Alkylating Activity (Pharmacologic Class)
Trabectedin (Compound) causes Alanine aminotransferase increased (Side Effect)