SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) has granted its request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A. Accelerated assessment reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP)...
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PHILADELPHIA – Amicus Therapeutics (Nasdaq: FOLD) today announced that the European Medicines Agency (EMA) validated the Marketing Authorization Applications (MAA) for AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. Validation of the application confirms the submission is accepted, and the EMA’s centralized procedure with Committee for...
SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. With today’s validation the MAA review can now commence. A CHMP opinion is anticipated...
Ten leading scientists in Europe – members of the nonprofit European Society for ME (http://esme-eu.com) – have formed a Think Tank for ME and will hold their first meeting with a press conference Saturday, June 13 in Stavanger, Norway. (See Scientific Panel Member Listing, below.) They want to initiate an...
Paris — A European strategy that calls upon Member States to implement national plans for rare diseases, before the end of 2013, was adopted by the Council of Health Ministers of the EU today. The Council Recommendation is important because it calls for concerted action at EU and national level...
HEMEL HEMPSTEAD, England & BEIJING, China & CAMBRIDGE, EUSA Pharma (UK) Limited and BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160) today announced that the Biologics License Application (BLA) for SYLVANT® (siltuximab for injection) was accepted by the China National Medical Products Administration (NMPA) and granted priority review. Siltuximab is a monoclonal...
Larchmont, NY – A new study evaluates an artificial intelligence (AI)-based algorithm for autocontouring prior to radiotherapy in head and neck cancer. Manual contouring to pinpoint the area of treatment requires significant time, and an AI algorithm to enable autocontouring has been introduced. The study is published in the peer-reviewed journal...
The Journal of Blood Medicine has recently published data evaluating treatment options for haemophilia A. The publication reported in an indirect comparison that individualised prophylaxis with Elocta® (rFVIIIFc-efmoroctog alfa) is more efficacious than Hemlibra™ (emicizumab) administered every four weeks, and at least as effective as more frequent emicizumab regimens for...
Treatment with anagrelide was associated with prolonged responses in patients with essential thrombocythemia (ET), according to results of a long-term observational, retrospective study reported in the British Journal of Haematology.[1] Anagrelide is an oral imidazo-quinazoline compound that was approved in 1997 by the US Food and Drug Administration (FDA) to reduce platelet...
BUFFALO, NY- February 7, 2024 – A new research perspective was published in Oncotarget’s Volume 15 entitled, “Preclinical and clinical evaluation of the Janus Kinase inhibitor ruxolitinib in multiple myeloma.” In this new paper, researchers Ashley Del Dosso, Elizabeth Tadevosyan, and James R. Berenson from ONCOtherapeutics, Berenson Cancer Center, and...