Lenmeldy (atidarsagene autotemcel)

It isessential that the manufacturer's labeling be consulted for more detailed information on dosage and administration of this drug. Dosage summary:

For autologous use only. For one-time single-dose IV use only.

*Children are required to undergo hematopoietic stem cell (HSC) mobilization followed by apheresis to obtain CD34+ cells for atidarsagene autotemcel manufacturing.

*Myeloablative conditioning must be administered before infusion of atidarsagene autotemcel.

*Confirm that the child's identity matches the unique patient identification information on the atidarsagene autotemcel infusion bag(s) prior to infusion.

*Dosing of atidarsagene autotemcel is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight. The minimum recommended dose is based on the MLD disease subtype (see Table 1 below).

*Administer each infusion bag of atidarsagene autotemcel as an IV infusion via a central venous catheter within 30 minutes via gravity or infusion pump. An infusion flow rate should be calculated based on the volume in each infusion bag.

*Do not sample, alter, irradiate, or refreeze atidarsagene autotemcel.

*Do not use a leukodepleting filter.

*See Full Prescribing Information for additional details on preparation and administration.

Table 1: Minimum and Maximum Recommended Dose of Atidarsagene Autotemcel

MLD Subtype Minimum Recommended Dose Maximum Recommended Dose (CD34+ cells/kg) (CD34+ cells/kg) Pre-symptomatic late 4.2 x 106 30 x 106 infantile Pre-symptomatic early 9 x 106 30 x 106 juvenile Early symptomatic early 6.6 x 106 30 x 106 juvenile

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Contraindicated Drug Combination: This drug combination is contraindicated and generally should not be dispensed or administered to the same patient.

Atidarsagene Autotemcel/Anti-Retrovirals

SEVERITY LEVEL: 1-Contraindicated Drug Combination: This drug combination is contraindicated and generally should not be dispensed or administered to the same patient.

MECHANISM OF ACTION: Atidarsagene autotemcel is prepared from apheresed cells that are transduced with a replication defective, self-inactivating lentiviral vector. Antiretrovirals may interfere with the manufacturing of apheresed cells.

CLINICAL EFFECTS: Use of antiretrovirals before mobilization and apheresis may interfere with the production of atidarsagene autotemcel.

PREDISPOSING FACTORS: None determined.

PATIENT MANAGEMENT: Discontinue antiretrovirals for at least one month prior to mobilization (or the expected duration of time needed for elimination of the medication) until all cycles of apheresis are completed. If a patient requires antiretrovirals for HIV prophylaxis, then confirm a negative HIV test before beginning mobilization and apheresis of CD34+ cells.

DISCUSSION: Antiretroviral medications may interfere with the manufacturing of atidarsagene autotemcel therapy.(1)

There are no severe interactions for this drug.

There are no moderate interactions for this drug.

*None

There are no contraindication contraindications for this drug.

• Hepatic veno-occlusive disease
• Infection
• Thromboembolic disorder
• Thrombotic disorder

There are no moderate contraindications for this drug.

*The most common non-laboratory adverse reactions (incidence >= 10%) 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 most common laboratory abnormalities were elevated D-dimer (67%), neutropenia (28%), and elevated liver enzymes (23%).

Less Frequent

• Bacterial sepsis
• Fungal infection of lung
• Pneumonia

More Frequent

• Abnormal hepatic function tests
• Fever
• Gastroenteritis
• Hepatomegaly
• Neutropenic disorder
• Skin rash
• Stomatitis
• Upper respiratory infection
• Viral infection

Less Frequent

• Bronchitis
• Pharyngitis
• Rhinitis
• Tonsillitis

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The safety and efficacy of atidarsagene autotemcel have been established in children with PSLI, PSEJ and ESEJ MLD. The clinical trials of atidarsagene autotemcel treated 20 PSLI, 7 PSEJ, and 10 ESEJ MLD children who received atidarsagene autotemcel between ages 8 - 19 months (median age of 12 months), 11 months - 5.56 years (median age of 2.57 years), and 2.54 - 11.64 years (median age of 5.84 years), respectively. The safety and efficacy of atidarsagene autotemcel have not yet been established in children with the late juvenile form of the disease.

There are no clinical data from the use of atidarsagene autotemcel in pregnant women. No animal reproductive and developmental toxicity studies have been conducted with atidarsagene autotemcel to assess whether it can cause fetal harm when administered to a pregnant woman. Atidarsagene autotemcel must not be administered during pregnancy because of the risk associated with myeloablative conditioning. Pregnancy after atidarsagene autotemcel infusion should be discussed with the treating physician.

In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.

There are no data on the presence of atidarsagene autotemcel in human or animal milk, the effects on the breastfed child, or the effects on milk production. Because of the potential risks associated with myeloablative conditioning, breast-feeding should be discontinued during conditioning. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for atidarsagene autotemcel and any potential adverse effects on the breastfed child from the treatment or from the underlying maternal condition. Breast-feeding after atidarsagene autotemcel infusion should be discussed with the treating physician. Children receiving breast milk may continue to do so throughout their treatment under the advice of their treating physician.

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