A group of more than 40 EU lawmakers backed the idea of an EU action plan for rare diseases, in a letter addressed to European Commission President Ursula von der Leyen.
treatment News
EMERYVILLE, Calif. – Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T-cell therapies to treat solid tumors, today announced the initiation of ARYA-2, a Phase I/II open-label, dose escalation clinical trial of ET140203 ARTEMIS® T-cell therapy in pediatric patients with relapsed or refractory hepatoblastoma (HB), hepatocellular neoplasm not otherwise specified (HCN-NOS),...
LONDON, UK – Vertex Pharmaceuticals Inc. (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy. CASGEVY is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD)...
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one...
NEW YORK, NY — Pfizer Inc. (NYSE: PFE) announced today that the European Commission (EC) has granted marketing authorization for VELSIPITY® (etrasimod) in the European Union to treat patients 16 years of age and older with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response,...
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)...
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...
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...