FDA Puts Partial Hold On Aeglea's Rare Disorder Trial In Pediatric Patients

Aeglea BioTherapeutics Inc AGLE received an FDA letter responding to a recently submitted protocol amendment for the Phase 1/2 trial of pegtarviliase for Classical Homocystinuria.
Classical Homocystinuria is a rare disorder of methionine metabolism, leading to an abnormal accumulation of homocysteine and its metabolites in blood and urine.
The protocol amendment requested the inclusion of adolescent patients at clinical trial sites in the U.S.
The FDA stated that the protocol did not provide adequate justification and evidence to support the prospect of direct clinical benefit for pediatric patients.
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The agency placed the trial on partial clinical hold for enrolling patients under 18 years of age under this Investigational New Drug (IND).
The company believes the letter will not impact the planned enrollment and dosing of patients aged 18 & above.
Enrollment in the trial remains on track, and Aeglea continues to expect to announce data in the fourth quarter of 2022, including data from cohort 3.
Price Action: AGLE shares are down 3.64% at $0.53 on the last check Tuesday.