News and Trends 2 May 2023 Schizophrenia injection gets FDA approval Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd., and MedinCell have announced the U.S. Food and Drug Administration (FDA) has approved UZEDY (risperidone) extended-release injectable suspension for the treatment of schizophrenia in adults. UZEDY is the first subcutaneous, long-acting formulation of risperidone that utilizes SteadyTeq, a copolymer technology proprietary to MedinCell that controls […] May 2, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 27 Apr 2023 Biogen ALS drug gets FDA ok The U.S. Food and Drug Administration (FDA) has approved Biogen’s QALSODY (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients […] April 27, 2023 - 5 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 20 Apr 2023 FDA approves Genentech’s lymphoma drug Genentech, a member of the Roche Group, has announced the U.S. Food and Drug Administration (FDA) has approved Polivy (polatuzumab vedotin-piiq) in combination with Rituxan (rituximab), cyclophosphamide, doxorubicin and prednisone (R-CHP). The approval covers the treatment of adult patients who have previously untreated diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS) or high-grade B-cell […] April 20, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 11 Apr 2023 Sumitomo Pharma Oncology receives orphan drug designation for Ewing sarcoma treatment Sumitomo Pharma Oncology, Inc., a clinical-stage company focused on novel cancer therapeutics, said the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for TP-1287, an investigational oral CDK9 inhibitor, for the treatment of Ewing sarcoma. “We are delighted to have received this designation for TP-1287 which underscores the need for additional treatment […] April 11, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 29 Mar 2023 Pharming immunodeficiency drug given FDA approval Pharming Group N.V. has announced that the US Food and Drug Administration (FDA) has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Pharming’s Joenja, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the […] March 29, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 3 Mar 2023 Orphan drug designation for Laevoroc metabolic immune checkpoint inhibitor Laevoroc Immunology, a privately-owned, Swiss oncology development company, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for immune checkpoint inhibitor LR 09. LR 09 is a novel metabolic immune checkpoint inhibitor, for the treatment of patients with hematological malignancies who are diagnosed with a relapse after allogeneic stem […] March 3, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 28 Feb 2023 European Commission approves first gene therapy for hemophilia B The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […] February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
Opinion 28 Feb 2023 Life changing treatments for rare disease – a beacon of hope By Sander Slootweg, managing partner, Forbion More than 300 million people around the globe are affected by a rare disease. This number does not even take into account the impact on these individuals’ support units that includes family, friends and caregivers. A rare disease is defined as a disease that affects no more than 1 […] February 28, 2023 - 6 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
News and Trends 24 Feb 2023 Sanofi hemophilia drug gets FDA approval The U.S. Food and Drug Administration (FDA) has approved Sanofi’s ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia […] February 24, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 21 Feb 2023 FDA approves alpha-mannosidosis drug Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), says the U.S. Food and Drug Administration (FDA) has approved Lamzede (velmanase alfa-tycv) for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. AM is an ultra-rare, progressive lysosomal storage disorder […] February 21, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 10 Feb 2023 FDA approves GSK drug for recurrent or advanced mismatch repair-deficient endometrial cancer GSK plc says the US Food and Drug Administration (FDA) has given full approval for Jemperli (dostarlimab-gxly) for the treatment of adult patients with mismatch repair-deficient (dMMR) recurrent or advanced endometrial cancer, as determined by a US FDA-approved test, that has progressed on or following a prior platinum-containing regimen in any setting and are not […] February 10, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 10 Feb 2023 Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment Rocket Pharmaceuticals, Inc says the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to RP-A501. RP-A501 is Rocket Pharmaceuticals’ investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. RMAT designation was […] February 10, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email