The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
May 10th 2024
A Veterans Affairs study finds low alpha-1 antitrypsin phenotyping rates, indicating the need for improved healthcare provider education on AATD.
Phase 3 OLYMPUS Trial Interim Analysis for UTUC Shows Efficacy
Yesterday, UroGen Pharma Ltd released positive data from its interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101 (MitoGel) for the non-surgical treatment of low-grade upper tract urothelial cancer (UTUC).
Phase III HAVEN 3 and Phase III HAVEN 4 Study in Hemophilia A Show Efficacy
Results from Roche's phase III HAVEN 3 and phase III HAVEN 4 studies, which assessed the administration of Hemlibra (emicizumab) in people with hemophilia A without factor VIII inhibitors, show efficacy.
Pitolisant Receives FDA Breakthrough Therapy and Fast Track Designations
Harmony Biosciences, LLC, announced that the FDA has granted Breakthrough Therapy and Fast Track designations to its product, pitolisant, for the treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy.
First Patient Enrolled in Phase 3 PFIC Trial of A4250
The first patient has been enrolled in PEDFIC-1, a phase 3 clinical trial of A4250, an ileal bile acid transporter (IBAT) inhibitor being studied for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC).
FDA Accepts IND Application for BIVV003, a Gene Therapy for Sickle Cell Disease
The US Food and Drug Administration (FDA) has accepted the Bioverativ's Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease.
Combinability of X4P-001-IO with Nivolumab Demonstrated in ccRCC Shows Promise
Data is released from a pilot study of X4P-001-IO in combination with nivolumab in patients with clear cell renal cell carcinoma (ccRCC) who are non-responsive to the anti-PD-1 checkpoint inhibitor nivolumab alone.
FDA Approves Epoetin Alfa-Epbx as a Biosimilar to Epogen/Procrit (Epoetin Alfa) for Anemia
FDA approves Retacrit (epoetin alfa-epbx) as a biosimilar to Epogen/Procrit (epoetin alfa) for the treatment of anemia caused by chronic kidney disease, chemotherapy, or the use of zidovudine in patients with HIV infection.
First Hemophilia A Patient in Phase 1/2 Study Dosed with Valoctocogene Roxaparvovec
The first patient has been dosed in aphase 1/2 study (BMN 270-203) evaluating the investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.