re QURE's HemoB trial (news from 6/22) | SGMO Message Board Posts


Sangamo Therapeutics, Inc.

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Msg  158509 of 159355  at  6/24/2021 9:13:11 AM  by

retired@49


re QURE's HemoB trial (news from 6/22)

QURE was planning to file BLA based on 52 week (starting on date of dosing) data, but now will have to wait until 52 weeks of "steady state" factor levels.  Goalposts moved?  
 
This could effect SGMO's HemoA timeline.... 
 
"The Company and its partner, CSL Behring, have had recent communications with U.S. Food and Drug Administration (FDA), including a pre-biologics licensing application (BLA) submission meeting held on June 4, 2021. The FDA confirmed that the primary evidence of durability of effect to inform regulatory decision-making will come from patients followed for at least a 52-week period beginning when etranacogene dezaparvovec-derived FIX levels have achieved steady state, rather than when etranacogene dezaparvovec is administered. This feedback was based upon review of statistical analysis plans, as no clinical data was provided or discussed. All patients in the HOPE-B pivotal study achieved steady-state FIX activity levels by 26-weeks after administration of etranacogene dezaparvovec. As a result, uniQure will now conduct as the sole primary endpoint a non-inferiority analysis of annualized bleeding rates (ABR) at 78 weeks after the administration (approximately 52-weeks after steady-state is achieved). The Company expects all patients to complete their 78-week follow-up visits by the end of the third quarter of 2021, and the Company and CSL Behring expect to submit the BLA in first quarter of 2022." 


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Replies
Msg # Subject Author Recs Date Posted
158510 Re: re QURE's HemoB trial (news from 6/22) TECH32 1 6/24/2021 9:25:06 AM
158512 Re: re QURE's HemoB trial (news from 6/22) Watsonncrick 16 6/24/2021 9:56:25 AM
158514 Re: re QURE's HemoB trial (news from 6/22) Bioimmuno 0 6/24/2021 10:00:21 AM


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