Author: Willem van Lawick

SAN RAFAEL, Calif., Aug. 19, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for drisapersen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 51 skipping treatment. The FDA has previously granted drisapersen Orphan and Fast Track Status, Breakthrough Therapy Designation, as well as Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. (more…)

Amsterdam, the Netherlands, July 31, 2015 – Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”) (Euronext Amsterdam and Euronext Brussels: KDS), a recently-listed clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, today announces the partial exercise of the Over-Allotment Option by Kempen & Co N.V. acting on behalf of the Underwriters and the end of the stabilisation period in relation to its initial public offering (the “IPO” or the “Offering”). (more…)

The target 23 patients have now been enrolled

Amsterdam, the Netherlands, July 30, 2015, 7.00 CEST – Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”) (Euronext Amsterdam and Euronext Brussels: KDS), a recently listed clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, today announces full enrolment of patients into its ongoing Phase II clinical study with its lead product, ATIR101™.
ATIR101™ is a cell-based product designed to enable stem cell transplantations from partially matched (haploidentical) family donors for blood cancer patients who do not have a matching stem cell donor available. The ongoing Phase II study with ATIR101™ is an international multi-centre study in which a total of 23 leukaemia patients, including patients with acute myeloid leukaemia (AML), acute lymphoblastic leukaemia (ALL) and myelodysplastic syndrome (MDS), will be enrolled and treated with ATIR101™ as part of their transplant regimen. (more…)

FDA Grants Priority Review Status; FDA PDUFA Date is December 27, 2015

SAN RAFAEL, Calif., June 29, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, and the Prescription Drug User Fee Act (PDUFA) goal date for a decision is December 27, 2015. The FDA has granted drisapersen Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. (more…)

SAN RAFAEL, Calif., June 25, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA confirms that the submission is complete and starts the EMA’s standard review process. Day 120 questions will be received on 22 October 2015, leading to a potential CHMP opinion in the first half of 2016 and a European Commission Decision by the third quarter of 2016. (more…)