Skytrofa denials in California external review

What the reviewers wrote

Where the denial was overturned

Findings: The physician reviewer found that Nature of Statutory Criteria/Case Summary: The patient’s parent has requested authorization and coverage for Skytrofa. The U.S. Food and Drug Administration (FDA) approves the use of lonapegsomatropin (Skytrofa), a depot formulation of growth hormone (GH) administered weekly, to treat pediatric GH deficiency. Researchers report that treatment with weekly lonapegsomatropin is non-inferior to daily somatotropin with respect to safety and efficacy for the treatment of GH deficiency. The authors concluded that children treated with once-weekly lonapegsomatropin showed continued improvement of height standard deviations (SD) scores through the second year of therapy without excess advancement of bone age.

Nature of Statutory Criteria/Case Summary: The enrollee has been diagnosed with pediatric growth hormone deficiency. After an unremarkable term gestation and labor, the patient was delivered vaginally and weight was appropriate for gestational age. The patient’s neonatal course included mild jaundice that did not require phototherapy. The patient’s early childhood was unremarkable with normal acquisition of developmental milestones. By apparent self-reports of parental heights, the treating pediatric endocrinologist estimated the patient’s mid-parental target height. The Health Plan indicates that the requested treatment is not medically necessary for the treatment of the patient’s medical condition. At issue is whether brand name medication Skytrofa for weekly injections is medically necessary for the treatment of the patient’s medical condition. The U.S.

Where the denial was upheld

Nature of Statutory Criteria/Case Summary: The parent of an enrollee has requested authorization and coverage for the medication Skytrofa Cartridge.Since 8/25/21, The U.S. Food and Drug Administration has approved Skytrofa (lonapegsomatropin-tcgd), a depot formulation of growth hormone administered weekly, to treat pediatric growth hormone deficiency. Published phase III randomized controlled trials (RCTs) have shown weekly lonapegsomatropin is non-inferior to daily somatotropin with respect to safety and efficacy for the treatment of growth hormone deficiency. Since lonapegsomatropin is administered weekly, the frequency of self-administration is reduced 86% each year, which improves compliance compared to daily injections.

Nature of Statutory Criteria/Case Summary: The patient’s parent has requested authorization and coverage for Skytrofa® Cartridge injections. This patient’s growth chart displays his height tracked a normal range between 10-25th percentiles until approximately age 13, then a normal range between 5-10th percentiles by approximately age 14 years. The patient’s weight was also normal and ranged from the 30-48th percentiles until age 13, and then above the 17th percentile by age 14 years. The U.S. Food and Drug Administration (FDA) defines idiopathic short stature (ISS) as a height z-score of at least -2.25 standard deviations (SDs) below the mean in the absence of identifiable pathology.

These outcomes describe eligible cases completed through California DMHC’s Independent Medical Review program. They do not estimate the chance that an internal appeal, an external review in another jurisdiction, or your individual case will succeed. Use the record to identify evidence patterns involving Skytrofa, then check the rights and deadlines that apply to your plan.