Sohonos (palovarotene)

Fibrodysplasia Ossificans Progressiva (FOP) Agents Oral Capsule

Advertisement

Last reviewed · Source: First Databank (FDB) drug data

Overview

Palovarotene is an orally bioavailable retinoid that acts as a retinoic acid receptor (RAR) agonist with selectivity for the gamma subtype of RAR.

In patients with fibrodysplasia ossificans progressiva (FOP), abnormal bone formation, including heterotrophic ossification, is driven by a gain-of-function mutation in the bone morphogenetic protein (BMP) type I receptor activin receptor-like kinase-2 (ALK2). Palovarotene is an orally bioavailable retinoic acid receptor (RAR) agonist with selectivity at the gamma subtype of RAR (RARgamma). Through binding to RARgamma, palovarotene decreases the BMP/ALK2 downstream signaling pathway by inhibiting the phosphorylation of SMAD1/5/8, which reduces ALK2/SMAD-dependent chondrogenesis and osteocyte differentiation resulting in reduced endochondral bone formation.

Palovarotene exposure (AUC) increases proportionally from 0.02 to 50 mg (0.001 to 2.5 times the maximum recommended dosage). Following once-daily oral administration, steady state is achieved by day 3. The median time to achieve peak concentrations (Tmax) of palovarotene is 3--4 hours when palovarotene is administered without food; a delay in Tmax of approximately 2 hours is observed when the drug is administered with a high-fat, high-calorie meal (800-1000 calories, 50-60% fat).

Palovarotene mean AUC and maximum serum concentration (Cmax) are increased by approximately 40 and 16%, respectively, when administered with food. There are no clinically significant differences in the AUC and Cmax of palovarotene capsules when administered whole compared to when the contents are sprinkled onto 5 mL of applesauce following a high-fat, high-calorie breakfast. In vitro, palovarotene is 97.9-99.6% bound to plasma proteins.

The mean elimination half-life of palovarotene is 8.7 hours following once-daily administration of 20 mg for 14 days with a standard breakfast. Palovarotene is extensively metabolized by cytochrome P-450 (CYP) 3A4 and to a minor extent by CYP2C8 and 2C19. Of the 4 known major metabolites (M2, M3, M4a, and M4b), M3 and M4B retain 1.7 and 4.2% of the activity of the parent drug, respectively.

Palovarotene is recovered in the feces (97.1%) and urine (3.2%).

There are no clinically significant differences in the pharmacokinetics of palovarotene based on age (range: 2-85 years), sex, race, smoking status, mild to moderate (creatinine clearance 30 to 89 mL/minute) renal impairment, or mild (Child-Pugh class A) hepatic impairment. The effect of severe renal impairment (creatinine clearance 15 to 29 mL/minute) or moderate to severe (Child-Pugh class B or C) hepatic impairment on the pharmacokinetics of palovarotene is unknown. Body weight (range: 13-130 kg) was found to have a significant effect on the pharmacokinetics of palovarotene resulting in increasing exposure with decreasing weight at the same dose.

The estimated steady-state AUC at the end of the dosing period and Cmax following weight-based dosing for 5, 10, and 20 mg (or dose equivalent) in pediatric patients <14 years old are comparable for the equivalent doses across the different weight groups.

USES: Palovarotene is used to treat a certain rare genetic problem (fibrodysplasia ossificans progressiva-FOP). It works by reducing bone formation in muscles and tendons that can be caused by FOP. Palovarotene belongs to a class of drugs known as retinoids, which are related to vitamin A.

Generic name: palovarotene
Pronunciation: (PAL-oh-VAR-oh-teen)
Drug class: Fibrodysplasia Ossificans Progressiva (FOP) Agents
Therapeutic class: Locomotor System

Reference product & biosimilars

No reference, biosimilar or interchangeable associated with this drug.

Today’s intelligence on Sohonos

Intelligence content is not available yet.

Indications

The following indications are listed for Sohonos:

  • Fibrodysplasia Ossificans Progressiva

Also known as: Munchmeyer Disease, Progressive Myositis Ossificans.

Dosing

Avoid concomitant use of palovarotene with moderate cytochrome P-450 (CYP) 3A inhibitors, if possible. If concomitant use with a moderate CYP3A inhibitor will occur, reduce the dosage of palovarotene by 50% (see Table 4).

Table 4. Dosage Reduction of Palovarotene during Concurrent Use of Moderate CYP3A Inhibitors

Weight Daily Dosage Week 1-4 Flare-up Week 5-12 Dosage Flare-up Dosage 10-19.9 kg 1 mg 5 mg 2.5 mg 20-39.9 kg 1.5 mg 6 mg 3 mg 40-59.9 kg 2 mg 7.5 mg 4 mg >=60 kg (also 2.5 mg 10 mg 5 mg includes all pediatric patients >=14 years of age and adults)

Patients that experience adverse reactions that require a dosage reduction during either chronic daily dosing or flare-up dosing should have their daily dosage reduced to the next lower dosage at the discretion of the clinician (see Table 3). Palovarotene dosage should be further reduced if the adverse reaction does not improve. If the patient is already receiving the lowest possible tolerated dosage, consider temporary interruption or permanent discontinuation of therapy. Subsequent flare-up dosing should be initiated at the same reduced dosage that was previously tolerated.

Table 3. Palovarotene Dosage Reduction for Toxicity

Dosage Prescribed Reduced Dosage 20 mg 15 mg 15 mg 12.5 mg 12.5 mg 10 mg 10 mg 7.5 mg 7.5 mg 5 mg 6 mg 4 mg 5 mg 2.5 mg 4 mg 2 mg 3 mg 1.5 mg 2.5 mg 1 mg

Drug interactions

Palovarotene is extensively metabolized by cytochrome P-450 (CYP) 3A4 and to a minor extent by CYP2C8 and CYP2C19. In vitro, palovarotene is an inducer of CYP3A4 and CYP2B6 but not CYP1A2, CYP2C8, CYP2C9, and CYP2C19. Palovarotene is not an inhibitor of CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, and CYP3A4.

In vitro, palovarotene is not an inhibitor or substrate of uridine diphosphate-glucuronosyltransferase (UGT) enzymes 1A1, UGT1A3, UGT1A4, UGT1A6, UGT1A9, or UGT2B7. Palovarotene is not an inhibitor of breast cancer resistance protein (BCRP), bile salt export pump (BSEP), organic anion transporter (OAT) 1 or OAT3, organic anion transporter polypeptide (OATP) 1B1 or OATP1B3, organic cation transporter (OCT) 1 or OCT2, multidrug and toxin extrusion (MATE) 1, MATE2 K, or P-glycoprotein (P-gp). Palovarotene is not a substrate of BCRP, OATP1B1, OATP1B3, OCT1, or P-gp.

Contraindications

*Pregnancy.

*History of allergy or hypersensitivity to retinoids or to any component of the palovarotene formulation. Anaphylaxis and other allergic reactions have occurred with other retinoids.

Adverse reactions

The most common adverse reactions of palovarotene (>=10% of patients) reported in clinical studies include dry skin, dry lips, arthralgia, pruritus, extremity pain, rash, alopecia, erythema, headache, back pain, skin exfoliation, nausea, musculoskeletal pain, myalgia, dry eyes, hypersensitivity, peripheral edema, and fatigue.

Precautions

No enhanced messaging available.

ICD codes

CodeTypeDescription
Fibrodysplasia ossificans progressiva
M61.1ICD10CMMyositis ossificans progressiva
M61.10ICD10CMMyositis ossificans progressiva, unspecified site
M61.111ICD10CMMyositis ossificans progressiva, right shoulder
M61.112ICD10CMMyositis ossificans progressiva, left shoulder
M61.119ICD10CMMyositis ossificans progressiva, unspecified shoulder
M61.121ICD10CMMyositis ossificans progressiva, right upper arm
M61.122ICD10CMMyositis ossificans progressiva, left upper arm
M61.129ICD10CMMyositis ossificans progressiva, unspecified upper arm
M61.131ICD10CMMyositis ossificans progressiva, right forearm
M61.132ICD10CMMyositis ossificans progressiva, left forearm
M61.139ICD10CMMyositis ossificans progressiva, unspecified forearm
M61.141ICD10CMMyositis ossificans progressiva, right hand
M61.142ICD10CMMyositis ossificans progressiva, left hand
M61.143ICD10CMMyositis ossificans progressiva, unspecified hand
M61.144ICD10CMMyositis ossificans progressiva, right finger(s)
M61.145ICD10CMMyositis ossificans progressiva, left finger(s)
M61.146ICD10CMMyositis ossificans progressiva, unspecified finger(s)
M61.151ICD10CMMyositis ossificans progressiva, right thigh
M61.152ICD10CMMyositis ossificans progressiva, left thigh
M61.159ICD10CMMyositis ossificans progressiva, unspecified thigh
M61.161ICD10CMMyositis ossificans progressiva, right lower leg
M61.162ICD10CMMyositis ossificans progressiva, left lower leg
M61.169ICD10CMMyositis ossificans progressiva, unspecified lower leg
M61.171ICD10CMMyositis ossificans progressiva, right ankle
M61.172ICD10CMMyositis ossificans progressiva, left ankle
M61.173ICD10CMMyositis ossificans progressiva, unspecified ankle
M61.174ICD10CMMyositis ossificans progressiva, right foot
M61.175ICD10CMMyositis ossificans progressiva, left foot
M61.176ICD10CMMyositis ossificans progressiva, unspecified foot
M61.177ICD10CMMyositis ossificans progressiva, right toe(s)
M61.178ICD10CMMyositis ossificans progressiva, left toe(s)
M61.179ICD10CMMyositis ossificans progressiva, unspecified toe(s)
M61.18ICD10CMMyositis ossificans progressiva, other site
M61.19ICD10CMMyositis ossificans progressiva, multiple sites

ICD code information from First Databank (FDB).

Formulary status

Search health-plan coverage for this drug by ZIP code.

Enter a ZIP code to view available health plans.

Last reviewed · Source: First Databank (FDB) drug data