Author: Willem van Lawick

Marketing Application in Europe Remains Under Review

SAN RAFAEL, Calif., Jan. 14, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) issued a Complete Response letter to the Company’s New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.
The FDA issues Complete Response letters to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. FDA has concluded that the standard of substantial evidence of effectiveness has not been met. BioMarin is reviewing the Complete Response Letter and will work with the FDA to determine the appropriate next steps regarding this application. (more…)

~ ATIR201™ on track to enter Phase I/II clinical development in Q1 2016 ~

Amsterdam, The Netherlands, December 21, 2015 – Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”) (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, today announces a collaboration with the Thalassaemia International Federation (TIF), an internationally renowned organisation that seeks to address the needs of patients, carers, healthcare professionals and the general public in the area of thalassaemia, an inherited blood disorder which results in improper oxygen transport and destruction of red blood cells in a patient. (more…)

SAN RAFAEL, Calif., Dec. 18, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) has notified the Company that they had not yet completed their review process and would be unable to take an action by the Prescription Drug User Fee Act (PDUFA) action date for KyndrisaTM (drisapersen) of December 27, 2015, and anticipate taking action in early January 2016.

– Statistically significant increase in Overall Survival and reduction in Transplant Related Mortality observed in comparison to a historical control group
– Zero patients developed grade III-IV acute Graft-versus-Host-Disease upon infusion of ATIR101™
– Analysis of full read out of primary endpoint data anticipated to be reported in April 2016

Amsterdam, The Netherlands, December 7, 2015 – Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”) (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, today presents data from its ongoing Phase II clinical trial in leukaemia patients at the American Society of Hematology (ASH) 57th Annual Meeting in Orlando, Florida.
The Company presents an update on its fully enrolled ongoing open label Phase II clinical trial (NCT01794299/EudraCT 2012-004461-41) with its lead product ATIR101™ added as a donor lymphocyte infusion to a haploidentical T-cell depleted stem cell transplantation.
Data presented in Session 732 (Clinical Allogeneic Transplantation) have a cut off date of November 23, 2015. As per that date a statistically significant reduction in Transplant Related Mortality (TRM) and a statistically significant increase in Overall Survival (OS) is observed in comparison to a historical control group based on Kaplan-Meier estimates (p=0.002 and p=0.0006 respectively). (more…)

Amsterdam, The Netherlands, November 27, 2015 – Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”) (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, today announces its Interim Management Statement for the nine months ended 30 September 2015.

Operational review
Kiadis Pharma continued to make good progress in developing its lead product ATIR101TM for blood cancer:
Full enrolment of the ongoing Phase II clinical study (CR-AIR-007) with ATIR101TM has been accomplished. The primary endpoint for the last patient in this trial will be reached at the end of Q1, 2016 and top-line results will follow at the beginning of Q2, 2016.
Another study (CR-AIR-008), testing repeat dose administration of ATIR101TM in parallel with the ongoing Phase II trial, has been initiated and has enrolled its first patient.
Establishing a closed manufacturing process that allows for automation which is advancing according to plan. (more…)