Expanded Access Metreleptin Study

Inclusion Criteria

• •1. Signed Written Informed Consent
• •a) Before any program procedures are performed, the details of the program will be described to the patient and the patient will be given a written informed consent document to read. If the patient agrees to participate in the program, consent will be indicated by signing and dating of the informed consent document in the presence of program personnel.
• •2. Target Population
• •1. Ability to comply with visits and procedures required by program
• •2. Previously enrolled in study FHA101/MB002-002
• •3. Has physician-confirmed partial lipodystrophy and had evidence of benefit with metreleptin treatment based on the following metabolic criteria demonstrated within the last year of metreleptin treatment (if on treatment over 1 year) from baseline values:
• •* TG reduction ≥ 30% OR
• •* HbA1c reduction ≥ 1% OR
• •* Decrease in insulin requirements ≥ 40% OR
• •* Decrease in episodes of pancreatitis OR
• •* Improvement in steatohepatitis OR
• •* Withdrawal of metreleptin led to marked worsening of metabolic parameters
• •3. Age and Reproductive Status
• •1. Male or female, over the age of 6 months
• •2. Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of HCG) within 24 hours prior to the restart of study drug.
• •3. Women must not be breastfeeding
• •4. WOCBP must agree to follow instructions for method(s) of contraception for the duration of treatment with metreleptin plus 5 half-lives of metreleptin plus 30 days (duration of ovulatory cycle) for a total of 6 months post-treatment completion.
• •5. Men who are sexually active with WOCBP must agree to follow instructions for method(s) of contraception for the duration of treatment with metreleptin plus 5 half-lives of the metreleptin plus 90 days (duration of sperm turnover) for a total of 3 months post-treatment completion.