STAMFORD, Conn. — SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany, announced today that the European Commission (EC) granted conditional marketing authorization for EZMEKLY® (mirdametinib) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in pediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and...
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Approval of Lynozyfic is based on data showing deep and durable responses in relapsed/refractory multiple myeloma Lynozyfic will provide a new option with convenient dosing and administration to patients who face cycles of relapse and remission TARRYTOWN, N.Y. — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the European Commission (EC) has granted conditional...
CHARLESTOWN, Mass. — Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that the European Commission (EC), acting upon the positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products, has granted Orphan...
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)...
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...
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...