SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that both the United States Food and Drug Administration (FDA) and European Commission (EC) have granted Orphan Drug Designation (ODD) for PTC923 for the treatment of patients with hyperphenylalaninemia. Phenylketonuria (PKU) accounts for 98% of all hyperphenylalaninemia cases and is a metabolic condition that...
treatment News
WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA. “The validation of the MAA is an important step towards making sepiapterin available to children and adults affected by PKU in Europe,” said Matthew B....
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...
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...
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), 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), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for LYT-200 in combination with anti-PD1 therapy for the treatment of...
REHOVOT, Israel — Purple Biotech Ltd. (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that harness the power of the tumor microenvironment to overcome tumor immune evasion and drug resistance, today announced it has determined 100mg/kg is the recommended Phase 2 dose (RP2D) for NT219 in combination with cetuximab in...
PARSIPPANY, N.J. – Pyros Pharmaceuticals, Inc., a leader in the development of enhanced specialty pharmaceuticals for rare diseases, announced today that VIGPODER™ (vigabatrin) for oral solution, USP, a treatment for appropriate patients with infantile spasms (IS), is now available. As a therapeutically equivalent product, VIGPODER™ is anticipated to offer the...