Lenmeldy (atidarsagene autotemcel) · Orchard Therapeutics (Europe) Ltd.

LENMELDY is an autologous gene therapy which includes hematopoietic stem cells (HSCs) that have been genetically modified ex vivo. The nature of LENMELDY is such that conventional pharmacokinetic studies on absorption, distribution, metabolism, and excretion are not applicable.

The safety and efficacy of LENMELDY was assessed in 39 children across two single-arm, open-label clinical trials and a European Union (EU) expanded access program (EAP). Two children with advanced disease were excluded from the efficacy analysis. The clinical trials enrolled 13 children with PSLI, 6 children with PSEJ and 9 children with ESEJ MLD. The EU EAP enrolled 7 children with PSLI, 1 child with PSEJ and 1 child with ESEJ MLD. All children had documented biochemical and molecular diagnosis of MLD based on ARSA activity below the normal range and identification of two disease-causing ARS...

The most common non-laboratory adverse reactions (occurring in ≥10% of all children within Year 1 of treatment) were: febrile neutropenia (85%), stomatitis (77%), respiratory tract infections (54%), rash (33%), device related infections (31%), other viral infections (28%), pyrexia (21%), gastroenteritis (21%), and hepatomegaly (18%). The following clinically significant adverse reactions are described elsewhere in the labeling: Thrombosis and Thrombotic Events [ see Warnings and Precautions (5.1...

None. None ( 4 )

Thrombosis and Thromboembolic Events : Evaluate the risk factors for thrombosis prior to and after LENMEDLY infusion. Consider prophylaxis with anti-thrombotic agents prior to treatment with LENMELDY. ( 5.1 ) Encephalitis : Monitor children for signs or symptoms of encephalitis after treatment with LENMELDY. ( 5.2 ) Serious Infection : Monitor children for serious infection after myeloablative conditioning and LENMELDY infusion. ( 5.3 ) Veno-occlusive Disease : Monitor children for signs and sym...

7 DRUG INTERACTIONS No formal drug interaction studies have been performed. LENMELDY is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug transporters. Anti-retrovirals : Do not take anti-retroviral medications for at least one month prior to initiating medications for stem cell mobilization and for the expected duration of time needed for the elimination of the ...