TEL AVIV, Israel — BioLineRx Ltd., a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced that the first patient has been dosed in the l evaluating motixafortide as monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies...
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TEL AVIV, Israel – BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, today announced positive top-line results from the Company’s GENESIS Phase 3 trial evaluating its lead clinical candidate, Motixafortide, in combination with granulocyte colony stimulating factor (G-CSF, the standard of care in this...
SARASOTA, Fla. – Biom Therapeutics, a clinical stage biotech company, announced today it has received orphan drug designation status for BIO017—which is designed to treat Angelman syndrome—from the U.S. FDA’s Office of Orphan Product Development. Biom anticipates commencing clinical trials for BIO017 in Q4 of 2021, following submission of an...
RMAT Designation Granted by FDA During Bleeding Disorders Awareness Month SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A....
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ® (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone. Safety results were consistent with the...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data characterizing the efficacy and safety of PALYNZIQ® (pegvaliase-pqpz) for the treatment of adolescents with phenylketonuria (PKU), which were presented at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan, Sept. 2-6, 2025. The Phase 3 PEGASUS study...
SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene...
SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company has completed full enrollment in a global Phase 2 randomized, placebo-controlled study of vosoritide, an investigational, once-daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature...
New data in children under 5 years with achondroplasia showed treatment with VOXZOGO was associated with improvement in tibial bowing, a common cause of pain and impaired function Data from the largest retrospective, multi-year observational study of more than 600 children and adults with hypochondroplasia found significantly higher rates of...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ® (pegvaliase-pqpz) in adults with phenylketonuria (PKU). The latest findings further validate the long-term benefit and established...