Appeal type: Experimental/Investigational
Decision: Upheld

Diagnosis: Migraine Headaches. 
Treatment: Emgality, Pre-service. 

The insurer denied the Emgality, Pre-service. 
The denial is overturned. 

The patient is a male teen with medical history significant for chronic intractable migraine headache without aura. According to the letter of medical necessity, he has been experiencing 30 headache days per month for the past ten months, most lasting more than four hours. Despite treatment with Aimovig and Botox, he continues to have daily headaches. He was unable to tolerate the following due to side effects: Trokendi XR (mental status changes, feeling hot) and propranolol (dizziness, nausea). He has also tried and failed treatment with cyproheptadine, amitriptyline, topiramate, triptans, and magnesium. One of his current medications, Aimovig, is Food and Drug Administration (FDA) approved for patients 18 years and older but has been covered for this patient. He uses naproxen as acute rescue therapy with some benefit. He was able to use galcanezumab (Emgality) successfully for three months but had to discontinue due to insurance concerns. He is followed very closely by Pediatric Neurology. 

Yes, the requested therapy is likely to be more beneficial for the enrollee than any available standard therapy. 

Galcanezumab is an injectable humanized anti-calcitonin gene-relaged peptide (CGRP) monoclonal antibody developed for the treatment of episodic cluster headache and in the prevention of migraine headaches. It is currently Food and Drug Administration (FDA) approved for persons 18 years and older for these indications. In this population, it has been shown to be effective, raising no significant major safety issues. It has not been formally studied in the pediatric and adolescent population, but there is literature guiding its use. Clinical recommendations include the following: against use in children or adolescents with compromised blood-brain barrier as in meningitis or other central/peripheral nervous system injury; against use in females that are pregnant, breastfeeding, or anticipating getting pregnant within six months; use with caution in those with known bone diseases or osteopenia, with optimized vitamin D level and close attention to growth until post-pubertal; close monitoring of weight and body mass index as marker of pituitary function; against use in those with known immunodeficiency or taking immunosuppressants; and against use in those with certain cardiopulmonary issues such as structural heart defects, cardiomyopathy, pulmonary hypertension, coronary artery disease, and history of or risk factors for stroke. The authors concluded, "until studies in children and adolescents are completed, anti-CGRP mAbs should be considered primarily for post-pubertal adolescents experiencing relatively frequent migraine (i.e. > 8 headache days per month) with moderate or severe migraine-related disability (as measured by PedMIDAS or other validated instrument)  should be done with close follow-up and attention to patient characteristics such as age, pubertal state, and medical comorbidities."  (1) To summarize, suggested indications include post-pubertal adolescent or pre-pubertal child in selected cases, greater than (>) eight headache days per month, PedMIDAS score greater than 30, and failure of greater than two preventive therapies (pharmacologic; nutraceutical such as riboflavin, coenzyme Q10, magnesium, melatonin; and/or non-pharmacologic such a cognitive behavioral therapy). 

In the case of this patient, he has tried and failed all "on-label" options available as well as non-pharmacologic therapies. He was successfully treated with galcanezumab for three months and has since not been able to achieve that same level of treatment efficacy; he remains clinically symptomatic up to 30 headache days per month. He meets the indications described above and has demonstrated efficacy without side effects. 

This patient has tried and failed all available standard therapies as well as off-label treatment with Aimovig. He was able to use galcanezumab for three months with excellent results and no safety concerns.  There are recommendations for use of galcanezumab in appropriately selected post-pubertal pediatric patients such as this patient. This patient meets the recommended indications, and further has demonstrated efficacy. 

Galcanezumab (Emgality) is likely to be more beneficial for this patient than any available standard therapy.