– Label includes full spectrum of PKU patients – European launch preparations underway WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for...
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WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington’s disease (HD) patients. The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p<0.0001) at Week 12 and favorable safety...
Washington, DC – The FDA will host Rare Disease Day, a virtual public meeting, on March 1, 2024 in global observance of Rare Disease Week. This year’s Rare Disease Day is dedicated to patients and health care professionals. Panels will discuss: The legal framework for approving studies and medical products...
LACHEN, Switzerland — Octapharma announced today the publication of the results from the clinical study WIL-31, which investigated the efficacy and safety of prophylaxis with wilate®, a von Willebrand factor/factor VIII (VWF/FVIII) concentrate, in patients with all types of von Willebrand disease (VWD). The results have been published in the...
NEW YORK, NY & MIAMI, Florida – New research has found that genetic variants associated with Parkinson’s disease (PD) are more common than researchers previously believed. Investigators in the Parkinson’s Foundation-backed PD GENEration study – which reached its goal of 15,000 participants ahead of schedule this spring – found that...
WASHINGTON — Think your job’s tedious? Try beheading 100 mosquitoes an hour. Gently, no smushing allowed. Malaria parasites lurk in these mosquitoes’ salivary glands, and a small company on the outskirts of the nation’s capital needs them unharmed for a dramatic test — attempting the first live vaccine to fight...
SAN FRANCISCO – PulmoSIM Therapeutics (PulmoSIM), the pharmaceutical subsidiary of VeriSIM Life, Inc., is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for asset PT001 for the treatment of Pulmonary Arterial Hypertension (PAH). PT001 targets multiple responsible pathways in PAH, to provide a...
BOSTON, Mass. — PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, demonstrated its growing leadership in idiopathic pulmonary fibrosis (IPF) at the 2025 IPF Summit this week in Boston. The Company highlighted the strength...
BOSTON, Mass. – PureTech Health plc (Nasdaq: PRTC, LSE: PRTC), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, today announced that enrollment has been completed in the ELEVATE IPF Phase 2b clinical trial evaluating LYT-100 (deupirfenidone) in patients with idiopathic pulmonary fibrosis (IPF). LYT-100 is...
BOSTON, Mass. – PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value, today announced the successful completion of the End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) regarding the development of deupirfenidone (LYT-100) for...