Paris, France – The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of Sarclisa (isatuximab) subcutaneous (SC) in combination with approved standard-of-care regimens for the treatment of patients with multiple myeloma (MM) across all currently approved indications for Sarclisa intravenous...
treatment News
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...
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...
COPENHAGEN, Denmark — Evaxion Biotech A/S (NASDAQ: EVAX), a clinical-stage TechBio company specializing in developing AI-Immunology™ powered vaccines, announces that the first patient in its EVX-01 Phase 2 trial in metastatic melanoma received the last vaccine dose in combination with KEYTRUDA® (NCT05309421). The Company initiated its Phase 2 clinical study...
SHANGHAI, China and SOUTH SAN FRANCISCO, Calif. — Everest Medicines (HKEX 1952.HK) and Kezar Life Sciences Inc. (Nasdaq: KZR) announced today that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) approved Kezar’s investigational new drug (IND) application for initiation of the Phase 2b PALIZADE trial in...
SHANGHAI, China — Everest Medicines, a biopharmaceutical company focused on the development, manufacturing and commercialization of innovative medicines and vaccines, announced today that its New Drug Application (NDA) for Nefecon® has been accepted for review by South Korean’s Ministry of Food and Drug Safety (MFDS) for the treatment of primary...