U.S. FDA remains highly supportive of gene therapies - Mizuho
02/09/2021 11:32
** Brokerage Mizuho says it believed the U.S. FDA remains
highly supportive of the gene therapy space, and is focused in a
pragmatic way on mitigating safety risks from known toxicities
** U.S. FDA staff on Wednesday released briefing documents
for a panel of independent experts meeting on Sept. 2-3 to
discuss safety risks of gene therapies that use the
adeno-associated virus (AAV) vector
** AAV is a popular vector used by U.S. approved gene
therapies such as Roche's ROG.S Luxturna and Novartis'
NOVN.S Zolgensma, and drug developers such as Uniqure UQ1.F
and Sarepta Therapeutics SRPT.O
** Mizuho says the safety issues discussed by FDA staff are
not surprising and revolve around a number of known side effects
seen in trials of such gene therapies
** FDA notes that side effects seen in trials include liver
toxicity, findings from brain MRIs and death of patients from
some of the adverse events
** The panel will discuss and provide recommendations on
gene therapies; while FDA is not bound to follow the advice of
its experts, it usually does
(Reporting by Manas Mishra in Bengaluru)
((Manas.Mishra@thomsonreuters.com;
www.twitter.com/Manasmishra24))