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ph-91249

Sohonos (palovarotene) Prior Authorization with Quantity Limit Program Summary

Policy Number: PH-91249

This program applies to Blue Partner, Commercial, GenPlus, NetResults A series, SourceRx, SourceRx-Performance, and Health Insurance Marketplace formularies.

POLICY REVIEW CYCLE

Effective Date	Date of Origin
01-01-2026	01-01-2026

FDA LABELED INDICATIONS AND DOSAGE

Agent(s)	FDA Indication(s)	Notes	Ref#
Sohonos® (palovarotene) Capsule	Reduction in volume of new heterotopic ossification in adults and pediatric patients aged 8 years and older for females and 10 years and older for males with fibrodysplasia ossificans progressiva (FOP)		1

Agent(s)

FDA Indication(s)

Notes

Ref#

Sohonos®

(palovarotene)

Capsule

Reduction in volume of new heterotopic ossification in adults and pediatric patients aged 8 years and older for females and 10 years and older for males with fibrodysplasia ossificans progressiva (FOP)

See package insert for FDA prescribing information: https://dailymed.nlm.nih.gov/dailymed/index.cfm

CLINICAL RATIONALE

Fibrodysplasia Ossificans Progressiva

Fibrodysplasia ossificans progressiva (FOP) is an extremely rare genetic connective tissue disorder characterized by the abnormal development of bone in areas of the body where bone is not normally present (heterotopic ossification [HO]), such as the ligaments, tendons and skeletal muscles. Specifically, this disorder causes the body’s skeletal muscles and soft connective tissues to undergo a metamorphosis, essentially a transformation into bone, progressively locking joints in place and making movement difficult or impossible. Patients with FOP have malformed big toes that are present at birth (congenital). Other skeletal malformations may occur. The abnormal episodic development of bone at multiple soft tissue sites leads to stiffness in affected areas, limited movement and eventual fusion (ankylosis) of affected joints (neck, back, shoulders, elbows, hips knees, wrists, ankles, jaw – often in that order).(2,3)

Episodic flare-ups (inflammatory soft tissue swellings) of FOP usually begin during early childhood and progress throughout life. Most cases of FOP occur as the result of a sporadic new variant in the activin receptor IA (ACVR1) gene in the bone morphogenetic protein (BMP) signaling pathway, which is important during the formation of the skeleton in the embryo and the repair of the skeleton following birth.(2,3)

Definitive genetic testing of FOP by DNA sequence analysis of the ACVR1 locus can confirm a diagnosis of FOP prior to the appearance of HO. Clinical suspicion of FOP early in life on the basis of malformed great toes can lead to early clinical diagnosis, confirmatory diagnostic genetic testing, and the avoidance of harmful diagnostic and treatment procedures.(2,3) Clinicians should be aware of the early diagnostic signs of FOP - congenital malformation of the great toes and episodic soft tissue swelling even before the appearance of HO.(2)

Sohonos (palovarotene), a retinoic acid receptor γ (RARγ) agonist, was approved by the U.S. Food and Drug Administration (FDA) as the first treatment for FOP to reduce extra-skeletal bone formation in adults and children aged 8 years and older for females, and 10 years and older for males. Other management is mainly supportive. High-dose glucocorticoids have limited use but are most effective in the management of the early inflammatory flare-ups affecting major joints of the appendicular skeleton and jaw, especially when used immediately after the onset of a flare-up. Oral and topical non-steroidal anti-inflammatory medications, cyclo-oxygenase-2 inhibitors, mast cell stabilizers, leukotriene inhibitors and occasional intravenous aminobisphosphonates are reported by patients to manage chronic pain, arthritic symptoms or ongoing disease progression.(2,3)

Efficacy

Study PVO-1A-301 (NCT03312634, Study 301) was a single arm study in 97 subjects with FOP with R206H mutation aged 4 years and older utilizing the Natural History Study (NHS, PVO-1A-001) as an external control (n=101). The primary efficacy endpoint was annualized volume of new heterotopic ossification (HO) as assessed by low-dose, whole body CT (WBCT) imaging (excluding head). All WBCT images from treated subjects in the 301 study and untreated subjects in the NHS were read in a manner blinded to study origination.(1)

Study 301 subjects received Sohonos 5 mg daily with increased dosing at the time of a flare-up defined as at least one symptom (e.g. pain, swelling, redness) consistent with a previous flare-up or a substantial high-risk traumatic event likely to lead to a flare-up, to 20 mg once daily for 4 weeks followed by 10 mg once daily for 8 weeks (denoted as the chronic/flare-up regimen), with flare-up treatment extension in 4-week increments for persistent symptoms. Anytime during flare-up treatment, the 12-week treatment restarted if the subject had another flare-up or substantial high-risk traumatic event. The dosing was adjusted according to body weight in skeletally immature children (children who had not reached at least 90% skeletal maturity defined as a bone age of greater than or equal to 12 years 0 months for girls and greater than or equal to 14 years 0 months for boys).(1)

The mean annualized new HO was 9.4 cm^3/year in subjects receiving the chronic/flare-up Sohonos treatment and 20.3 cm^3/year in untreated subjects in the NHS based on a linear mixed effect model. The treatment effect was about 10.9 cm^3/year with 95% confidence interval (-21.2 cm^3/year, -0.6 cm^3/year).(1,4)

Secondary endpoints did not show a difference between treatment arms for the prevention of new HO formation. Therefore, it is believed that palovarotene's benefit lies in reducing the volume of new HO rather than preventing new HO altogether.(5)

Safety

Sohonos is contraindicated in pregnancy, and any patient with hypersensitivity to retinoids or any component of Sohonos.(1)

Sohonos has a boxed warning for embryo-fetal toxicity and premature epiphyseal closure in growing pediatric patients.(1)

REFERENCES

Number	Reference
1	Sohonos prescribing information. Ipsen Biopharmaceuticals, Inc. March 2025.
2	Kaplan FS, Al Mukaddam M, Baujat G, et al. The medical management of fibrodysplasia ossificans progressiva: Current treatment considerations (3rd edition). International Clinical Council on FOP. 2024 Jul:1-159.
3	National Organization for Rare Disorders (NORD). Fibrodysplasia ossificans progressiva. Last updated June 2024. Available at: https://rarediseases.org/rare-diseases/fibrodysplasia-ossificans-progressiva/
4	Pignolo RJ, Hsiao EC, Al Mukaddam M, et al. Reduction of new heterotopic ossification (HO) in the open-label, phase 3 MOVE trial of palovarotene for fibrodysplasia ossificans progressiva (FOP). J Bone Miner Res. 2023 Mar;38(3):381-394. doi: 10.1002/jbmr.4762
5	Food and Drug Administration (FDA) Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC). Palovarotene. June 28, 2023. Available at: https://www.fda.gov/media/174518/download

POLICY AGENT SUMMARY PRIOR AUTHORIZATION

Target Brand Agent(s)	Target Generic Agent(s)	Strength	Targeted MSC	Available MSC	Final Age Limit	Preferred Status

Sohonos	palovarotene cap	1 MG ; 1.5 MG ; 10 MG ; 2.5 MG ; 5 MG	M ; N ; O ; Y	N

POLICY AGENT SUMMARY QUANTITY LIMIT

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	QL Amount	Dose Form	Day Supply	Duration	Addtl QL Info	Allowed Exceptions	Targeted NDCs When Exclusions Exist

Sohonos	palovarotene cap	1 MG	112	Capsules	28	DAYS
Sohonos	palovarotene cap	1.5 MG	112	Capsules	28	DAYS
Sohonos	palovarotene cap	2.5 MG	140	Capsules	28	DAYS
Sohonos	palovarotene cap	5 MG	84	Capsules	28	DAYS
Sohonos	palovarotene cap	10 MG	56	Capsules	28	DAYS

CLIENT SUMMARY – PRIOR AUTHORIZATION

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	Client Formulary
Sohonos	palovarotene cap	1 MG ; 1.5 MG ; 10 MG ; 2.5 MG ; 5 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx ; SourceRx-Performance

CLIENT SUMMARY – QUANTITY LIMITS

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	Client Formulary
Sohonos	palovarotene cap	1.5 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx ; SourceRx-Performance
Sohonos	palovarotene cap	2.5 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx ; SourceRx-Performance
Sohonos	palovarotene cap	10 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx ; SourceRx-Performance
Sohonos	palovarotene cap	5 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx ; SourceRx-Performance
Sohonos	palovarotene cap	1 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx ; SourceRx-Performance

PRIOR AUTHORIZATION CLINICAL CRITERIA FOR APPROVAL

Module

Clinical Criteria for Approval

Initial Evaluation

Target Agent(s) will be approved when ALL of the following are met:

ONE of the following:
1. The requested agent is eligible for continuation of therapy AND ONE of the following:

Agents Eligible for Continuation of Therapy

Sohonos (palovarotene)

1. 1. The patient has been treated with the requested agent (starting on samples is not approvable) within the past 90 days OR
  2. The prescriber states the patient has been treated with the requested agent (starting on samples is not approvable) within the past 90 days AND is at risk if therapy is changed OR
2. BOTH of the following:
  1. ONE of the following:
    1. The patient has a diagnosis of fibrodysplasia ossificans progressiva (FOP) AND ALL of the following:
      1. Genetic analysis confirms mutation in the activin receptor IA (ACVR1) gene AND
      2. The patient has signs of heterotopic ossification (HO) AND
      3. The requested agent will be used to reduce the volume of new HO OR
    2. The patient has another FDA labeled indication for the requested agent and route of administration AND
  2. If the patient has an FDA labeled indication, then ONE of the following:
    1. The patient’s age is within FDA labeling for the requested indication for the requested agent OR
    2. There is support for using the requested agent for the patient’s age for the requested indication AND
The prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist, rheumatologist), or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
The patient does NOT have any FDA labeled contraindications to the requested agent

Length of Approval: 12 months

NOTE: If Quantity Limit applies, please refer to Quantity Limit Criteria.

Renewal Evaluation

Target Agent(s) will be approved when ALL of the following are met:

The patient has been previously approved for the requested agent through the plan’s Prior Authorization process (Note: patients not previously approved for the requested agent will require initial evaluation review) AND
The patient has had clinical benefit with the requested agent AND
The prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist, rheumatologist), or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
The patient does NOT have any FDA labeled contraindications to the requested agent

Length of Approval: 12 months

NOTE: If Quantity Limit applies, please refer to Quantity Limit Criteria.

QUANTITY LIMIT CLINICAL CRITERIA FOR APPROVAL

Module	Clinical Criteria for Approval
Universal QL	Quantity Limit for the Target Agent(s) will be approved when ONE of the following is met: The requested quantity (dose) does NOT exceed the program quantity limit OR The requested quantity (dose) exceeds the program quantity limit AND ONE of the following: BOTH of the following: The requested agent does NOT have a maximum FDA labeled dose for the requested indication AND There is support for therapy with a higher dose for the requested indication OR BOTH of the following: The requested quantity (dose) does NOT exceed the maximum FDA labeled dose for the requested indication AND There is support for why the requested quantity (dose) cannot be achieved with a lower quantity of a higher strength that does NOT exceed the program quantity limit OR BOTH of the following: The requested quantity (dose) exceeds the maximum FDA labeled dose for the requested indication AND There is support for therapy with a higher dose for the requested indication Length of Approval: up to 12 months

Module

Clinical Criteria for Approval

Universal QL

Quantity Limit for the Target Agent(s) will be approved when ONE of the following is met:

The requested quantity (dose) does NOT exceed the program quantity limit OR
The requested quantity (dose) exceeds the program quantity limit AND ONE of the following:
1. BOTH of the following:
  1. The requested agent does NOT have a maximum FDA labeled dose for the requested indication AND
  2. There is support for therapy with a higher dose for the requested indication OR
2. BOTH of the following:
  1. The requested quantity (dose) does NOT exceed the maximum FDA labeled dose for the requested indication AND
  2. There is support for why the requested quantity (dose) cannot be achieved with a lower quantity of a higher strength that does NOT exceed the program quantity limit OR
3. BOTH of the following:
  1. The requested quantity (dose) exceeds the maximum FDA labeled dose for the requested indication AND
  2. There is support for therapy with a higher dose for the requested indication

Length of Approval: up to 12 months

This pharmacy policy is not an authorization, certification, explanation of benefits or a contract. Eligibility and benefits are determined on a case-by-case basis according to the terms of the member’s plan in effect as of the date services are rendered. All pharmacy policies are based on (i) information in FDA approved package inserts (and black box warning, alerts, or other information disseminated by the FDA as applicable); (ii) research of current medical and pharmacy literature; and/or (iii) review of common medical practices in the treatment and diagnosis of disease as of the date hereof. Physicians and other providers are solely responsible for all aspects of medical care and treatment, including the type, quality, and levels of care and treatment.

The purpose of Blue Cross and Blue Shield of Alabama’s pharmacy policies are to provide a guide to coverage. Pharmacy policies are not intended to dictate to physicians how to practice medicine. Physicians should exercise their medical judgment in providing the care they feel is most appropriate for their patients.

Neither this policy, nor the successful adjudication of a pharmacy claim, is guarantee of payment.

Commercial _ PS _ Sohonos_PAQL _ProgSum_ 01-01-2026