In the company’s financial results for 2021 released on May 11, Takeda announced the decision to discontinue the development of TAK-609, a therapeutic for Hunter Syndrome. Additionally, Takeda is collaborating with JCR Pharmaceuticals in another therapeutic attempt for the condition.
The U.S. Food and Drug Administration approved Takeda Pharmaceutical Company Limited’s Vonvendi [von Willebrand factor (Recombinant)] for routine prophylaxis to reduce the frequency of bleeding episodes in patients with severe Type 3 von Willebrand disease (VWD) receiving on-demand therapy.
In a flurry of activity, multiple biopharma companies made their first appearance on the Nasdaq Stock Exchange on June 25, raising hundreds of millions of dollars to advance the development of next-generation therapeutics and scale their businesses.
Shares of Avrobio were up in trading after the company posted positive clinical data from gene therapy trials in three different rare lysosomal diseases: Fabry, Gaucher type 1 and cystinosis.
While the world has largely been focused on the development of vaccines and therapeutics for Covid-19, the U.S. Food and Drug Administration has remained busy lining up potential approvals of medications for other diseases and illnesses.
In a controlled laboratory test, Eagle Pharmaceuticals’ malignant hyperthermia treatment Ryanodex (dantrolene sodium) inhibited the growth of SARS-CoV-2, the virus causing the COVID-19 pandemic. The company hopes to launch a clinical trial testing the efficacy of the drug in patients.
Zogenix, with headquarters in Emeryville, California, announced the acquisition of Oakland, California-based Modis. Both companies focus on rare diseases.
Orchard Therapeutics secured an exclusive license for an ex vivo autologous hematopoietic stem cell gene therapy program for the treatment of MPS-I developed by SR-Tiget.
Orchard Therapeutics announced that the European Medicines Agency granted Priority Medicines (PRIME) designation to OTL-300, an investigational autologous ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta-thalassemia.
