Paris, France – The European Medicines Agency has granted orphan designation to rilzabrutinib, a reversible covalent Bruton’s tyrosine kinase (BTK) inhibitor, for IgG4-related disease (IgG4-RD). EMA grants orphan designation to investigational therapies addressing rare, life-threatening or debilitating medical diseases or conditions that affect no more than 5 in 10,000 persons...
treatment News
TOKYO, Japan — Astellas Pharma Inc. President and CEO Naoki Okamura contributed to the development of a pediatric formulation to treat schistosomiasis as a member of the Pediatric Praziquantel Consortium. The Consortium announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted...
RAHWAY, N.J. – Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the European Commission (EC) has approved KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with platinum-containing chemotherapy as neoadjuvant treatment, then continued as monotherapy as adjuvant treatment, for resectable non-small cell lung cancer...
NEW YORK, NY — Mesoblast Limited, a leader in allogeneic cellular medicines for inflammatory diseases, today announced that the United States Food and Drug Administration (FDA) has granted its allogeneic cell therapy Revascor® (rexlemestrocel-L) a Rare Pediatric Disease (RPD) Designation following submission of results from the randomized controlled trial in...
Washinton, DC – The FDA on Monday published a revised draft guidance document, aimed at helping sponsors and pharmaceutical companies to develop therapies for Alzheimer’s disease, particularly in its early stages before the onset of overt dementia. Monday’s draft document updates the FDA’s previous guidance, released in February 2018. The...
EAST LANSING, Michigan – Barth syndrome (BTHS) is an ultra-rare but debilitating genetic disease affecting cell mitochondria. High infant mortality due to cardiac complications and a weakened immune system significantly decrease life expectancy. Current medications treat symptoms but not the cause, leaving an unmet clinical need to develop effective treatments...
Tokyo, Japan – Leukemia is a common term used to refer to a form of blood cancer. However, there are different types of leukemia depending on the cell type involved. One unique form is myeloid/natural killer (NK) cell precursor acute leukemia (MNKPL). Because of its rarity, there is no consensus...
Bellingham, Washington – In the realm of biomedical sciences, the quest for accurate and efficient diagnostic tools is ever-evolving. One such promising innovation making waves is the photoacoustic (PA) technique. In the past decade, PA imaging has emerged as a viable imaging modality demonstrated in many clinical applications with promising...
SEOUL, South Korea and HYDERABAD, India — The International Vaccine Institute (IVI), an international organization with a mission to discover, develop, and deliver safe, effective, and affordable vaccines for global health, today announced that it has commenced a technology transfer of simplified Oral Cholera Vaccine (OCV-S) to Biological E. Limited...
PHILADELPHIA – Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, announced today that the data from the Phase 3 PROPEL pivotal trial, assessing the efficacy, safety and tolerability of AT-GAA in adults with late-onset Pompe disease compared to the standard of...