4:37 PM
 | 
Nov 07, 2018
 |  BC Extra  |  Company News

Phase II trial alone may not support BLA for Voyager's Parkinson's gene therapy

Voyager Therapeutics Inc. (NASDAQ:VYGR) said late Wednesday that FDA now considers a randomized Phase II trial of VY-AADC to be an "early phase exploratory study," suggesting that the Phase II trial by itself may no longer be sufficient to support a BLA for the Parkinson's disease gene therapy. The company plans to meet with FDA this year to clarify the agency's responses.

Voyager, which announced the news in its 3Q18 earnings after market close, added $0.36 to $13.99 on Wednesday. The company lost $1.69 (12%) to $12.30 in after-hours trading.

In July, FDA told Voyager in a written response that a single Phase II trial in 42 patients could be considered sufficient for the agency to accept a BLA, if the gene therapy were to meet the trial’s primary endpoint of improving diary on-time without dyskinesia vs. placebo and show no major safety concerns. The company said Wednesday that it received an addendum to the written response in late October. In the addendum, FDA informed the company that the agency currently considers the Phase II trial an early phase exploratory study even though the Phase II data could support the safety and efficacy of VY-AADC and could be considered in the BLA review.

Voyager said patient screening is ongoing for the Phase II trial, and the company will announce when the first patient has been dosed with VY-AADC.

VY-AADC is an adeno-associated virus (AAV) serotype 2 capsid and a cytomegalovirus promoter designed to deliver the dopa decarboxylase (DDC; AADC) gene directly into the putamen.

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