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09.12.2017 13:30
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BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at 59th American Society of Hematology (ASH) Annual Meeting Concurrent with NEJM Public
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PR Newswire
SAN RAFAEL, Calif., Dec. 9, 2017
SAN RAFAEL, Calif., Dec. 9, 2017 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The updated results will be presented during an oral presentation at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition by John Pasi, M.B., Ch.B., Ph.D., from Barts and the London School of Medicine and Dentistry and primary investigator for this Phase 1/2 study. On Monday, Dec. 11, 2017, Professor Pasi will present the data, which will include sustained normal or near-normal Factor VIII levels in severe hemophilia A for most patients with a maximum follow up of 19 months. Previously, the company provided updated data on the 4e13 vg/kg dose on Oct. 18, 2017 and on the 6e13 vg/kg dose on July 11, 2017 at a medical meeting.
The data presented at ASH is the most current data (Nov. 16, 2017 cut off) and includes 78 weeks of data for the 6e13 vg/kg dose and 48 weeks of data for the 4e13 vg/kg dose.
"With 1.5 years of data on valoctocogene roxaparvovec, our understanding of this novel gene therapy is considerable, and we are looking forward to drawing on that knowledge in the Phase 3 GENEr8 studies, which we expect will begin enrolling patients this month," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We're pleased that this work is being presented in one of the most highly regarded peer reviewed publications and at a pace that is faster than other in vitro gene therapies. This most current data presented at ASH combined with the one-year data published in NEJM will contribute to an increase in understanding within the scientific community about gene therapy in general and specifically about the exciting new developments in a potential one-time treatment for severe hemophilia A."
Efficacy Data of 4e13 vg/kg Dose as Presented at ASH
As of Nov. 16, 2017, a total of six patients with severe hemophilia A, who had all been treated with prophylactic Factor VIII pre-study, received a single dose of valoctocogene roxaparvovec at 4e13 vg/kg. Since the last data update provided on Oct. 18, 2017, the three patients with the longest follow-up (at week 48) have Factor VIII activity levels that are in or near to the normal range with both median and mean values of 49%. Median annualized bleed and factor VIII use rates for the 4e13 vg/kg cohort were zero after Week 4 when their Factor VIII activity rose above 5%. According to the World Federation of Hemophilia rankings of severity of hemophilia A, the mild hemophilia A range of Factor VIII activity levels is between 5% and 40%, and the normal range of Factor VIII activity levels for people without disease is between 50% and 150%, in each case expressed as a percentage of normal factor activity in blood.
Factor VIII Levels (%) of 4e13 vg/kg Dose Patients* by Visit (N=6) at Nov. 16, 2017 data cut
| Week**
| 4 | 8 | 12 | 16 | 20 | 24 | 28 | 32 | 36 | 40 | 44 | 48 |
| 4e13 vg/kg Dose | | | | | | | | | | | | |
| n | 6 | 6 | 6 | 6 | 6 | 6 | 6 | 6 | 6 | 3 | 3 | 3 |
| Median Factor VIII Level*** (%) | 4 | 15 | 21 | 29 | 34 | 28 | 34 | 42 | 38 | 48 | 56 | 49 |
| Mean Factor VIII Level*** (%) | 5 | 13 | 19 | 26 | 31 | 29 | 31 | 37 | 35 | 50 | 61 | 49 |
| Range ( low, high) | (2,10) | (3,21) | (6,32) | (5,38) | (7,45) | (7,43) | (4,44) | (4,54) | (4,55) | (37,64)
| (45,83) | (38,60) |
*All patients had severe hemophilia A at baseline, defined as less than or equal to 1% of Factor VIII activity levels, expressed as a percentage of normal factor activity in blood.
**Weeks were windowed by +/- 2 weeks
*** Bolded numbers are in the mild to normal range of Factor VIII activity as defined by the World Federation of Hemophilia, http://www.wfh.org/en/page.aspx?pid=643 (link current as of Dec. 6, 2017). Factor VIII levels are determined by one-stage assay.
Valoctocogene Roxaparvovec Reduces Bleeds and Factor VIII Use: Summary of Annualized Bleeding Rate (ABR) and FVIII Use Rate of 4e13 vg/kg Dose for Patients Previously on Prophylaxis (N=6) at Nov. 16, 2017 data cut
| | Before valoctocogene | After valoctocogene roxaparvovec |
| | Median (mean, SD) | Median (mean, SD) |
| Annualized Bleeding Rate* (bleeding episodes per year per subject) | 8.0 (12.2, 15.4) | 0.0 (0.6, 1.4) |
| Annualized FVIII Use Rate* (infusions per year per subject) | 155.5 (146.5, 41.6) | 0.0 (2.0, 4.8) |
*Post-infusion data were based on data after Week 4.
Efficacy Data on 6e13 vg/kg Dose as Presented at ASH
As of the Nov. 16, 2017 data cutoff, all seven patients at the 6e13 vg/kg dose had been followed for at least 78 weeks post infusion. Median and mean Factor VIII levels from week 20 through 78 for the 6e13 vg/kg dose cohort have been consistently within the normal range, expressed as a percentage of normal factor activity in blood. At 78 weeks post infusion, the median and mean Factor VIII levels of the 6e13 vg/kg cohort are 90 and 89% respectively. Median annualized bleed and factor VIII use rates for the 6e13 vg/kg were zero after Week 4. After 52 weeks of follow-up, the phase 1/2 study protocol specified that planned patient visits were reduced in frequency from once every one to two weeks to once every three months. Consequently, as of May 31, 2017 when all patients in the 6e13 vg/kg dose had reached 52 weeks of follow up, all patients in the 6e13 vg/kg dose converted to a quarterly follow-up visit schedule.
Factor VIII Levels (%) of 6e13 vg/kg Dose Patients* by Visit (N=7) at Nov. 16, 2017 data cut
| Week**
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