Polaris Group (The Company, TWSE:6550), today announced that the Company has initiated the rolling submission of its Biologic License Application (BLA) for ADI-PEG 20 to the U.S. Food and Drug Administration (FDA) for the systemic treatment of patients with malignant pleural mesothelioma with non-epithelioid histology, in combination with a platinum agent and pemetrexed. This submission is based on the results of the successful phase 3 trial of ADI-PEG 20 in combination with a platinum agent and
Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that the European Medical Agency (EMA) has awarded its Priority Medicines (PRIME) designation for elsunersen (PRAX-222) for the treatment of SCN2A Gain of Function (GoF) developmental and epileptic encephalopathy (DEE). The EMA
QTORIN™ rapamycin has potential to be first approved therapy and standard of care in the U.S. for Microcystic Lymphatic Malformations
Microcystic Lymphatic Malformations is a chronically debilitating and lifelong genetic disease affecting an estimated more than 30,000 patients in the U.S.
Palvella Therapeutics, Inc., a clinical-stage biopharmaceutical company developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no