The FDA is in a forgiving mood: After clearing uniQure’s gene therapy to restart trials today, it’s handing the same reprieve to Voyager Therapeutics.
The biotech was slapped with a clinical hold on an IND for the program, VY-HTT01, because of issues with chemistry, manufacturing and controls (CMC). This marked a miserable time for the program, coming one year after Sanofi’s Genzyme unit pulled out of a collab for it.
But now the FDA has lifted its hold on the gene therapy candidate in Huntington’s disease (HD), and “confirmed that the company may proceed with its planned phase 1/2 clinical trial,” according to a statement from the company, which plots to start this year in early HD patients.
This comes after Voyager cleared up its CMC issues: “The decision was made following a comprehensive review of the Chemistry, Manufacturing and Controls information previously submitted to the FDA,” the biotech added, though the FDA does not give its version of events in these sorts of decisions.
“The decision by the FDA regarding our IND application for VY-HTT01 for Huntington’s disease represents an important milestone for Voyager and is the result of years of commitment to developing an impactful new therapy to address this devastating disease,” said Andre Turenne, president and CEO of Voyager.
This comes amid a dearth of successful R&D in Huntington’s, a progressive brain disorder caused by a defective gene that is usually fatal after around two decades. It typically starts in young to middle-aged adults.
Just last month, a once-promising program from Wave Life Sciences for HD, using a different approach, namely antisense oligonucleotides, was thrown out after both drugs were deemed unable to help patients.
Just days before, Roche and Ionis also stopped work on tominersen, also known as IONIS-HTTRx and RG6042, which is designed to reduce the production of HTT, with low efficacy scuppering its development.
Voyager’s shares were up 14% on the news mid-morning Monday.