HOUSTON, TX – Moleculin Biotech, Inc., (Nasdaq: MBRX), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to Annamycin for the treatment of AML. Annamycin is the Company’s next-generation anthracycline...
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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...
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Washinton, DC – The FDA on Monday published a revised draft guidance document, aimed at helping sponsors and pharmaceutical companies to develop therapies for Alzheimer’s disease, particularly in its early stages before the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...
Many people with Lambert-Eaton myasthenic syndrome (LEMS) wait an extremely long time for a diagnosis. Our family waited nine months, a relatively short time compared with others in the LEMS community. As we waited for a diagnosis to be confirmed for our 16-year-old daughter Grace, a fog of uncertainty hung over our...
NORTH BETHESDA, Md. — The Foundation for the National Institutes of Health (FNIH) announces the launch of a public-private partnership to build the largest data source for amyotrophic lateral sclerosis (ALS) and expedite the identification of biomarkers and clinical outcome assessments that would allow earlier diagnosis and accelerated drug development...
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...