New Medicines

Haemophilia A


New molecular entity

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Nov 17Dimension Therapeutics acquired by Ultragenyx Pharmaceutical [3].
Jun 14Dimension Therapeutics enters into a collaboration with Bayer HealthCare to develop and commercialise gene therapies for the treatment of haemophilia A [3].


Gene therapy product designed using the NAV® gene delivery technology and a recombinant adeno-associated viral (rAAV) vector
It affects 1:4,000 to 1:5,000 live male births worldwide. It is five times as common as haemophilia B [1].
Haemophilia A

Trial or other data

Jul 21PI/II study (NCT03588299) now expected to finish collecting primary outcome data in May 27 [6].
Nov 20PI/II study (NCT03588299) is recruiting and expects to complete collection of primary outcome data in Oct 22 [5].
Jul 20Ultragenyx releases efficacy and safety data from cohort 3 of the PI/II trial (NCT03588299). Three cohorts with two patients each have been dosed with AAVhu37 (DTX201 / BAY 2599023). Initial data from the third dose cohort (2.0 x 10^13 GC/kg) and longer-term data from the lower dose cohorts were presented at ISTH. The data demonstrated a dose response with FVIII expression of 72.1% and 12.9% at weeks 29 and 26 in the two Cohort 3 patients. No spontaneous bleeds were reported after Cohort 3 patients reached peak expression despite prophylaxis discontinuation. One traumatic bleed occurred with no need for Factor VIII treatment. No adverse events related to DTX201 were observed, and ALT elevations in both patients were successfully treated with corticosteroids. Longer-term data from the two earlier cohorts showed sustained FVIII expression up to 16 months with no loss of expression [4].
Mar 20PI/II study (NCT03588299) is recruiting [2].
Nov 18Nov 18: PI/II trial to determine the safety and tolerability of DTX201 in adults with severe haemophilia A starts (NCT03588299; 19429). The open-label trial is enrolling approximately 18 volunteers in the US, Bulgaria, France, Germany, the Netherlands, Spain and the UK (sites are Manchester Royal Infirmary and St Thomas Hospital in London). The study is due to complete Jul 25, with primary outcome data collection completing in Jan 22 [2].