By Counterforce Health Team in Fabrazyme (agalsidase beta) —

If Fabrazyme Isn't Approved by Blue Cross Blue Shield of Michigan: Formulary Alternatives & Exception Paths

Answer Box: Your Options When Fabrazyme Is Denied

If Blue Cross Blue Shield of Michigan (BCBSM) denies Fabrazyme (agalsidase beta) for Fabry disease, you have two main paths: try their preferred alternative (typically Elfabrio or Galafold) or file a formulary exception request. BCBSM covers both Fabrazyme and Elfabrio under their Enzyme Replacement Therapy for Fabry Disease policy, but may designate one as preferred. Start by calling BCBSM member services at 1-800-482-4045 to confirm which Fabry therapy is currently preferred, then work with your specialist to either try the preferred option or document why Fabrazyme specifically is medically necessary for your case.

Table of Contents

  1. When Alternatives Make Sense
  2. BCBSM's Fabry Disease Treatment Options
  3. Comparing Your Options: Pros and Cons
  4. Exception Strategy: Fighting for Fabrazyme
  5. Switching Logistics and Coordination
  6. Re-trying for Fabrazyme Later
  7. Appeals Process in Michigan
  8. FAQ: Common Questions About Fabry Alternatives

When Alternatives Make Sense

Not every Fabrazyme denial means you should immediately fight for that specific medication. Sometimes, trying BCBSM's preferred alternative first can be both medically appropriate and strategically smart.

Consider alternatives when:

  • Your Fabry disease is newly diagnosed and you haven't tried any enzyme replacement therapy
  • You have a GLA mutation that's amenable to migalastat (Galafold) and your kidney function is stable
  • Your current symptoms are mild to moderate without rapid organ progression
  • You want to avoid bi-weekly IV infusions if an oral option might work

Fight for Fabrazyme specifically when:

  • You've been stable on Fabrazyme and switching could disrupt disease control
  • You've tried and failed the preferred alternative (inadequate response or intolerance)
  • Your GLA mutation is non-amenable to migalastat
  • You have severe kidney disease (eGFR <30) where oral therapy may be less effective
From our advocates: We've seen patients successfully transition between Fabry therapies when the medical team documents the rationale clearly. One patient initially denied Fabrazyme was approved after a three-month trial of the preferred alternative showed inadequate symptom control, with detailed before-and-after lab comparisons strengthening the exception request.

BCBSM's Fabry Disease Treatment Options

BCBSM covers three main treatments for Fabry disease under different policies and criteria:

Enzyme Replacement Therapies (Medical Benefit)

Fabrazyme (agalsidase beta)

  • Coverage: BCBSM Fabry ERT policy
  • Administration: IV infusion every 2 weeks (1 mg/kg)
  • Age requirement: 2 years and older
  • Billing codes: J0180

Elfabrio (pegunigalsidase alfa)

  • Coverage: Same BCBSM Fabry ERT policy as Fabrazyme
  • Administration: IV infusion every 2 weeks (1 mg/kg)
  • Age requirement: 18 years and older
  • Billing codes: J2508

Oral Pharmacological Chaperone (Pharmacy Benefit)

Galafold (migalastat)

  • Coverage: Separate BCBSM prior authorization for oral specialty drugs
  • Administration: Oral capsule every other day
  • Key restriction: Only works with amenable GLA mutations
  • Age requirement: 16 years and older (varies by plan)

Comparing Your Options: Pros and Cons

Treatment Pros Cons Best For
Fabrazyme Most clinical experience, proven kidney protection, works regardless of mutation type Bi-weekly infusions, potential for antibody formation Established patients, non-amenable mutations, severe disease
Elfabrio Potentially lower immunogenicity, similar efficacy to Fabrazyme Newer drug with less long-term data, still requires infusions Patients with antibodies to other ERTs, new ERT starts
Galafold Oral therapy, no infusions needed, good for mild-moderate disease Only works with amenable mutations (~35% of patients), less effective in advanced kidney disease Amenable mutations, stable kidney function, preference for oral therapy

Testing Requirements

Before starting any Fabry therapy, BCBSM typically requires:

  • Genetic testing: Confirmed pathogenic GLA mutation
  • Enzyme activity: Alpha-galactosidase A deficiency (especially in males)
  • Specialist evaluation: Genetics, nephrology, or cardiology consultation
  • Baseline assessments: Kidney function, heart imaging, symptom documentation

For Galafold specifically, you'll need amenability testing to confirm your mutation responds to the oral chaperone therapy.

Exception Strategy: Fighting for Fabrazyme

If BCBSM prefers a different Fabry therapy but you need Fabrazyme specifically, here's how to build a strong exception request:

Step-by-Step Exception Process

  1. Get the denial in writing - Request specific reasons why Fabrazyme was denied
  2. Gather medical records - Collect genetic testing, specialist notes, lab trends, imaging
  3. Document medical necessity - Have your Fabry specialist write a detailed letter
  4. Submit exception request - Use BCBSM's formulary exception process
  5. Request peer-to-peer - Ask for your doctor to speak directly with BCBSM's medical director
  6. Appeal if denied - File internal appeal within 180 days

Medical Necessity Letter Checklist

Your specialist's letter should include:

  • Confirmed Fabry diagnosis with genetic and enzyme testing results
  • Clinical severity including organ involvement (kidney, heart, neurologic)
  • Prior therapy history and why alternatives aren't appropriate
  • Specific rationale for Fabrazyme (established efficacy, patient stability, contraindications to alternatives)
  • Treatment goals and monitoring plan
  • Supporting citations from FDA labeling and clinical guidelines
Tip: Reference BCBSM's own policy language. Their Fabry ERT form states both Fabrazyme and Elfabrio are covered when criteria are met, so emphasize patient-specific factors favoring one over the other.

Switching Logistics and Coordination

If you decide to try BCBSM's preferred alternative first, coordinate the transition carefully:

From Fabrazyme to Elfabrio

  • No washout period needed - Can switch directly between ERTs
  • Same dosing schedule - Both are 1 mg/kg every 2 weeks
  • Monitor for reactions - Different protein structures may cause different side effects
  • Track effectiveness - Compare kidney function, symptoms, and biomarkers

From ERT to Galafold

  • Confirm amenability - Must have genetic testing showing amenable mutation
  • Check kidney function - Usually not started if eGFR <30 mL/min/1.73 m²
  • Plan transition timing - No gap needed; start Galafold within 1-2 weeks of last infusion
  • Intensive monitoring - Check labs every 3-6 months in first year

Infusion Center Coordination

  • Schedule final ERT infusion and clearly document the switch plan
  • Cancel future appointments once new therapy is confirmed
  • Arrange monitoring visits for lab draws and assessments
  • Keep pathway open for returning to ERT if oral therapy fails

Counterforce Health helps patients navigate these transitions by ensuring all documentation requirements are met and coordinating between specialists, infusion centers, and insurance companies to minimize gaps in therapy.

Re-trying for Fabrazyme Later

If you try an alternative first but it doesn't work well, you can request Fabrazyme again with stronger documentation:

Document Everything During Alternative Trial

  • Baseline measurements before starting alternative therapy
  • Regular monitoring of kidney function, heart status, symptoms, biomarkers
  • Side effects or limitations with detailed dates and descriptions
  • Objective markers of inadequate response (worsening labs, progression of symptoms)
  • Quality of life impacts including work, daily activities, hospitalizations

Timeline for Reassessment

  • Minimum 6 months on alternative therapy to demonstrate adequate trial
  • Sooner if serious adverse effects or rapid disease progression
  • Annual reviews to reassess optimal therapy choice

Building Your Case

A strong request for Fabrazyme after trying alternatives should show:

  • Inadequate efficacy despite appropriate dosing and duration
  • Tolerability issues that interfere with quality of life or adherence
  • Disease progression despite treatment with the alternative
  • Specialist recommendation for switching based on clinical evidence

Appeals Process in Michigan

If your exception request is denied, Michigan has robust appeal rights under the Patient's Right to Independent Review Act.

Internal Appeals (BCBSM)

  • Timeline: 180 days from denial to file
  • Decision: 30 days for standard, 72 hours for expedited
  • Requirements: Written appeal with medical records and physician support

External Review (Michigan DIFS)

  • Timeline: 127 days after final internal denial
  • Decision: 60 days standard, 72 hours expedited
  • Cost: Free to patients
  • Contact: Michigan DIFS at 877-999-6442

When to Request Expedited Review

  • Declining kidney function requiring urgent treatment
  • Recent cardiac events or progression
  • Neurologic complications from untreated Fabry disease

FAQ: Common Questions About Fabry Alternatives

How long does BCBSM prior authorization take for Fabry therapies? Standard prior authorizations typically take 5-15 business days. Expedited requests (when medically urgent) are decided within 72 hours.

What if my GLA mutation isn't amenable to Galafold? Non-amenable mutations require enzyme replacement therapy. Document this in your medical records to bypass any step therapy requirements for oral therapy.

Can I switch between Fabrazyme and Elfabrio multiple times? Yes, but frequent switching should be medically justified. Each change requires new prior authorization and careful monitoring for effectiveness and side effects.

Does BCBSM require step therapy for all Fabry patients? Step therapy requirements vary by specific plan and can change annually. Check your current formulary or call member services to confirm current policies.

What if I live far from an infusion center? Geographic barriers can support medical necessity arguments for oral therapy (if amenable) or home infusion services. Document travel distance and time in your exception request.

How often do Fabry therapy appeals succeed in Michigan? External reviews in Michigan overturn insurance denials in approximately 50% of cases when strong clinical evidence is provided, according to appeals data.

Medical Disclaimer: This information is for educational purposes only and does not constitute medical advice. Always consult with your healthcare provider and insurance company for guidance specific to your situation.

Need Help? Counterforce Health specializes in turning insurance denials into successful appeals by providing evidence-backed documentation that meets each plan's specific requirements.

Sources & Further Reading

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