Efficacy
Pre-symptomatic late infantile (PSLI) MLD
Pre-symptomatic early juvenile (PSEJ) MLD
Early symptomatic early juvenile (ESEJ) MLD
with early onset* MLD in the US1
LENMELDY IS PROVEN SAFE AND EFFECTIVE FOR EXTENDING SEVERE MOTOR IMPAIRMENT-FREE SURVIVAL IN PSLI MLD PATIENTS2
At the age of 5 years, 100% of LENMELDY-treated PSLI patients remained event-free compared with 0% of untreated NHx LI patients.
TREATMENT WITH LENMELDY IN PSLI MLD PATIENTS SIGNIFICANTLY EXTENDS OVERALL SURVIVAL2
OVERALL SURVIVAL FOR PSLI PATIENTS
LENMELDY significantly extends overall survival in patients with PSLI MLD compared with untreated NHx LI patients. All of the treated PSLI patients who took part in the study up to 6 years from birth were still alive at last follow-up. In contrast, 42% (10/24) of the untreated NHx LI patients had died.
COGNITIVE FUNCTION FOR PSLI PATIENTS
19 of 20 treated PSLI patients had performance standard scores and language standard scores above the threshold of severe impairment (performance/language standard score >55) through the last follow-up.
17 of 20 patients with PSLI MLD had performance standard scores and language standard scores at or above the threshold for moderate impairment as compared to untreated NHx patients who demonstrate severe cognitive impairment early in their disease course.
MANY PSEJ MLD PATIENTS TREATED WITH LENMELDY RETAINED MOTOR AND COGNITIVE FUNCTION AT LAST FOLLOW-UP2
Of the 7 treated PSEJ patients, 3 patients were too young at last follow-up to evaluate efficacy of LENMELDY, as symptom onset may not begin until 7 years of age in PSEJ MLD; 1 patient died from a cerebral infarction.
2 patients had evaluable motor and cognitive outcomes.
1 patient had evaluable motor outcomes, but while showing stable normal cognitive function, was neither old enough nor had sibling data for cognitive events to be evaluable.
Motor Function for PSEJ patients
All evaluable (3/3) LENMELDY-treated PSEJ patients maintained the ability to walk independently (GMFC-MLD Level 0 or 1) at time of last assessment.
COGNITIVE FUNCTION FOR PSEJ PATIENTS
Both of the evaluable LENMELDY-treated PSEJ patients (n=2) had normal cognitive function at ages 11.9 and 11.4 years.
MANY LENMELDY-TREATED ESEJ PATIENTS HAD LESS SEVERE DECLINE IN COGNITIVE FUNCTION3
Of the 10 ESEJ LENMELDY-treated patients, 1 patient was not assessed after 7.4 years due to disease progression. 2 patients did not have language or performance assessments due to death.2
COGNITIVE FUNCTION FOR ESEJ PATIENTS
6 of the 7 ESEJ LENMELDY-treated patients who remained event-free at last assessment had normal cognitive function, with performance standard scores above 85.2
4 of the LENMELDY-treated patients who remained event-free had favorable cognitive outcomes in the setting of motor decline.2
EJ, early juvenile; GMFC, Gross Motor Function Classification; HSC, hematopoeitic stem cell; LI, late infantile; MLD, metachromatic leukodystrophy.
References: 1. Lamichhane A, Cabrero RF. Metachromatic leukodystrophy. In: StatPearls [internet]. StatPearls Publishing; January 2024. Accessed March 7, 2024. https://www.ncbi.nlm.nih.gov/books/NBK560744/ 2. LENMELDY (atidarsagene autotemcel) Prescribing Information. Orchard Therapeutics. 3. Lin G, Suh K, Fahim SM, et al. Atidarsagene autotemcel for metachromatic leukodystrophy. Institute for Clinical and Economic Review; October 30, 2023. Accessed March 21, 2024. https://icer.org/assessment/metachromatic-leukodystrophy-2023/#timeline
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TREATMENT WITH LENMELDY IN PSLI MLD PATIENTS SIGNIFICANTLY EXTENDS OVERALL SURVIVAL2
OVERALL SURVIVAL FOR PSLI PATIENTS
LENMELDY significantly extends overall survival in patients with PSLI MLD compared with untreated NHx LI patients. All of the treated PSLI patients who took part in the study up to 6 years from birth were still alive at last follow-up. In contrast, 42% (10/24) of the untreated NHx LI patients had died.
COGNITIVE FUNCTION FOR PSLI PATIENTS
19 of 20 treated PSLI patients had performance standard scores and language standard scores above the threshold of severe impairment (performance/language standard score >55) through the last follow-up.
17 of 20 patients with PSLI MLD had performance standard scores and language standard scores at or above the threshold for moderate impairment as compared to untreated NHx patients who demonstrate severe cognitive impairment early in their disease course.
MANY PSEJ MLD PATIENTS TREATED WITH LENMELDY RETAINED MOTOR AND COGNITIVE FUNCTION AT LAST FOLLOW-UP2
Of the 7 treated PSEJ patients, 3 patients were too young at last follow-up to evaluate efficacy of LENMELDY, as symptom onset may not begin until 7 years of age in PSEJ MLD; 1 patient died from a cerebral infarction.
2 patients had evaluable motor and cognitive outcomes.
1 patient had evaluable motor outcomes, but while showing stable normal cognitive function, was neither old enough nor had sibling data for cognitive events to be evaluable.
Motor Function for PSEJ patients
All evaluable (3/3) LENMELDY-treated PSEJ patients maintained the ability to walk independently (GMFC-MLD Level 0 or 1) at time of last assessment.
COGNITIVE FUNCTION FOR PSEJ PATIENTS
Both of the evaluable LENMELDY-treated PSEJ patients (n=2) had normal cognitive function at ages 11.9 and 11.4 years.
MANY LENMELDY-TREATED ESEJ PATIENTS HAD LESS SEVERE DECLINE IN COGNITIVE FUNCTION3
Of the 10 ESEJ LENMELDY-treated patients, 1 patient was not assessed after 7.4 years due to disease progression. 2 patients did not have language or performance assessments due to death.2
COGNITIVE FUNCTION FOR ESEJ PATIENTS
6 of the 7 ESEJ LENMELDY-treated patients who remained event-free at last assessment had normal cognitive function, with performance standard scores above 85.2
4 of the LENMELDY-treated patients who remained event-free had favorable cognitive outcomes in the setting of motor decline.2
EJ, early juvenile; GMFC, Gross Motor Function Classification; HSC, hematopoeitic stem cell; LI, late infantile; MLD, metachromatic leukodystrophy.
References: 1. Lamichhane A, Cabrero RF. Metachromatic leukodystrophy. In: StatPearls [internet]. StatPearls Publishing; January 2024. Accessed March 7, 2024. https://www.ncbi.nlm.nih.gov/books/NBK560744/ 2. LENMELDY (atidarsagene autotemcel) Prescribing Information. Orchard Therapeutics. 3. Lin G, Suh K, Fahim SM, et al. Atidarsagene autotemcel for metachromatic leukodystrophy. Institute for Clinical and Economic Review; October 30, 2023. Accessed March 21, 2024. https://icer.org/assessment/metachromatic-leukodystrophy-2023/#timeline
