Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Hereditary Angioedema (HAE)

Treatment: Takhzyro 300 mg/2ml syringe (procedure code J0583)

The insurer denied coverage for Takhzyro 300 mg/2ml syringe (procedure code J0583)

The denial is overturned

 
This case involves an adult female diagnosed with hereditary angioedema (HAE) who was denied coverage for Takhzyro 300 mg/2ml syringe (procedure code J0583).  The rationale stated that the medication can be approved when the patient meets certain criteria.  There should be confirmation that the patient has C1 inhibitor (C1-INH)( a gene) antigenic level below normal or that the patient has a problem with the gene that controls that (HAE-causing C1 INH mutation).  The physician responded stating that the patient has hereditary angioedema (HAE) type III.  She was recommended to initiate Takhzyro for prophylaxis treatment of attacks.  The patient tried and failed Xolair, which caused hives and a rash.  She was managing breakthrough attacks with Firazyr since 2017 with resolution.  Prior lab reports were normal including C4 level, C1 inhibitor level, and function.  She endorsed recurrent abdominal pain and angioedema symptoms.  When having an attack, the patient reported 80-90% involved in her abdomen and gastrointestinal tract with the development of significant abdominal distention, nausea, vomiting, and diarrhea lasting for 2 to 4 days in the absence of treatment.  The remainder of her attacks involve either her face or her episodes of significant throat swelling on 2 occasions, which led to respiratory concerns.  She reported that when used early in the course of an attack, icatibant (Firazyr) will relieve symptoms in about 30 to 60 minutes.  The patient was having more frequent attacks and required prophylaxis therapy over the last several months.  Firazyr was helping the attacks when taken right at the start and required approximately 3 doses to resolve symptoms.  The physician stated the patient would not be using both Firazyr and Takhzyro simultaneously and was instructed to use Firazyr for only urgent breakthrough attacks and Takhzyro for prophylaxis to reduce the number of attacks over the year.  The physician again noted that the patient has genetically and clinically diagnosed HAE and reported cases of patients with HAE with normal C4 levels and stated that normal C4 does not exclude the diagnosis of HAE.  This review pertains to the denial of coverage for Takhzyro 300 mg/2ml syringe (procedure code J0583).

The Food and Drug Administration states that Takhzyro is a plasma kallikrein inhibitor (monoclonal antibody) indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 2 years and older.  The patient was previously denied coverage for Takhzyro because she was not found to have C1 inhibitor (C1-INH) antigenic level below normal or that the patient has a problem with the gene that controls that (HAE-causing C1 INH mutation).  However, the proposed medication is not case specific and is intended to treat patients with hereditary angioedema in general.  The dose of the medication is appropriate and within the standard of care set forth by the Food and Drug Administration.  The intended use of the medication is also within the FDA recommendations for prophylaxis to prevent additional attacks.  Given the provided documentation, it was determined that the requested services service is appropriate.  

Based on the above, the insurer's denial must be overturned. The health care plan did not act reasonably and with sound medical judgment and in the best interest of the patient. 

The medical necessity for Takhzyro 300 mg/2ml syringe (procedure code J0583) services is substantiated.