Cologne, Germany – Zanubrutinib, a second-generation Bruton tyrosine kinase (BTK) inhibitor, is approved for several therapeutic indications. The German Institute for Quality and Efficiency in Health Care (IQWiG) investigated whether the drug has an added benefit compared with the appropriate comparator therapy for adult patients with chronic lymphocytic leukaemia (CLL)...
treatment News
DUARTE, California – The novel drug, zanzalintinib (XL092), showed promising cancer-fighting abilities in patients with advanced clear-cell renal cell carcinoma (ccRCC), according to initial results from the STELLAR-001 clinical trial. According to trial results, which were presented at the 2023 International Kidney Cancer Symposium, antitumor activity was observed in patients...
Copenhagen, Denmark – The FDA rejected Zealand Pharma’s glucagon receptor agonist in congenital hyperinsulinism (CHI) after identifying problems at a third-party manufacturing site. The company said it remains confident that it can resubmit in the first half of 2024. Zealand revealed last week that the regulator issued a complete response...
CELEBRATION, Fla. — Zevra Therapeutics Inc., a rare disease therapeutics company, today announced it resubmitted its New Drug Application (NDA) for arimoclomol, an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC) to the U.S. Food and Drug Administration (FDA) on December 22, 2023. Based on standard...
ATLANTA, GA — UCB, a global biopharmaceutical company, announced today that ZILBRYSQ® (zilucoplan) is now available in the U.S. for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. ZILBRYSQ was approved by the U.S. Food and Drug Administration (FDA) on October 17,...
EMERYVILLE, Calif. – Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing rare disease therapies today announced the U.S. Food and Drug Administration (FDA) has accepted for filing and granted Priority Review to the company’s supplemental New Drug Application (sNDA) for the use of FINTEPLA® for the treatment of seizures associated...
EMERYVILLE, Calif. – Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing rare disease therapies, today announced new data showing the positive impact of treatment with FINTEPLA® (fenfluramine) oral solution on everyday executive function for children and young adults with Lennox-Gastaut syndrome (LGS). FINTEPLA is being investigated for the treatment of...
EMERYVILLE, Calif. – Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing and commercializing rare disease therapies, announced that its product FINTEPLA® (fenfluramine) oral solution has been recognized by two distinguished clinicians in an Epilepsy & Behavior editorial titled “Raising the Bar: Fenfluramine Sets New Treatment Standards for Dravet Syndrome.” In the editorial, the authors, Joseph...
DEVON, Pa. – Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that the European Patent Office has issued Patent No. 3687513, titled “Treatment of Fragile X Syndrome and Autism with Cannabidiol,” which includes claims directed to methods...
DEVON, Pa. – Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced the Company has received guidance from the U.S. Food and Drug Administration (FDA) on a confirmatory Phase 3 trial of Zygel in patients with Fragile X...