SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial chylomicronemia syndrome (FCS) in Brazil. FCS is a rare genetic disease which results in significant disease burden to patients including potentially fatal pancreatitis...
treatment News
WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. These data provide further evidence of the potential meaningful benefits of sepiapterin treatment for the...
SOUTH PLAINFIELD, N.J. — PTC Therapeutics, Inc. (PTC) today announced the receipt of two government grants to support clinical development programs in rare disorders. The U.S. Department of Defense Neurofibromatosis Research Program has awarded an $822,345 grant to support a new open-label Phase 2 clinical trial of PTC’s product candidate...
– 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...
WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call today, April 28 at 4:30 p.m. ET to share results from the 24-month interim analysis of the PIVOT-HD long-term extension study of votoplam. PTC’s partner Novartis stated on its quarterly earnings call today that long-term extension data are supportive of the now-initiated...
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 — 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...