Mucopolysaccharidosis Type 2 (MPS II, Hunter Syndrome) Treatments
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Brand Name
Avlayah
Generic Name
Tividenofusp Alfa-Eknm
View Brand Information FDA approval date: March 20, 2026
Form: Injection
What is Avlayah (Tividenofusp Alfa-Eknm)?
AVLAYAH is indicated for the treatment of neurologic manifestations of Hunter syndrome when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment. This indication is approved under accelerated approval based on the reduction of cerebrospinal fluid heparan sulfate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Limitations of Use AVLAYAH is not recommended for use in combination with other enzyme replacement therapies for the treatment of Hunter syndrome. AVLAYAH is a hydrolytic lysosomal glycosaminoglycan -specific enzyme indicated for the treatment of neurologic manifestations of Hunter syndrome when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment. This indication is approved under accelerated approval based on reduction of cerebrospinal fluid heparan sulfate observed in patients treated with AVLAYAH. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial. Limitations of Use AVLAYAH is not recommended for use in combination with other enzyme replacement therapies.
Approved To Treat
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Brand Information
AVLAYAH (tividenofusp alfa-eknm)
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies, including AVLAYAH, have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
Initiate AVLAYAH in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue AVLAYAH and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur
1INDICATIONS AND USAGE
AVLAYAH is indicated for the treatment of neurologic manifestations of Hunter syndrome (Mucopolysaccharidosis type II, MPS II) when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment.
This indication is approved under accelerated approval based on the reduction of cerebrospinal fluid heparan sulfate
Limitations of Use
AVLAYAH is not recommended for use in combination with other enzyme replacement therapies for the treatment of Hunter syndrome.
2DOSAGE FORMS AND STRENGTHS
For injection: 150 mg of tividenofusp alfa-eknm as a white to off-white lyophilized powder with a cake-like appearance in a single-dose vial for reconstitution and further dilution.
3CONTRAINDICATIONS
None.
4ADVERSE REACTIONS
The following clinically significant adverse reactions are described elsewhere in the labeling:
- Hypersensitivity Reactions Including Anaphylaxis
- Infusion-Associated Reactions
- Anemia
- Membranous Nephropathy
4.1Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The safety of AVLAYAH was evaluated in male pediatric patients with Hunter syndrome in Trial 1
In Trial 1, the most common adverse reactions (≥20%) reported in AVLAYAH-treated patients were infusion-associated reaction (IAR), upper respiratory tract infection, ear infection, pyrexia, anemia, cough, vomiting, diarrhea, rash, COVID-19, rhinorrhea, nasal congestion, fall, headache, skin abrasion, and urticaria.
Dose interruptions of AVLAYAH due to an adverse reaction occurred in 91% of patients. The most frequently reported adverse reaction leading to dose interruption was IAR (31 [66%] patients). Other frequently reported adverse reactions leading to dose interruption were COVID-19 (18 [38%] patients), pyrexia (16 [34%]), upper respiratory tract infection (16 [34%]), nasal congestion (6 [13%]), and vomiting (6 [13%]). Dose interruption included skipped infusions due to an adverse reaction as well as temporary infusion pauses with subsequent completion during the same visit.
Dose reductions of AVLAYAH due to adverse reactions occurred in 57% of patients; the majority of these reactions were IARs.
In Trial 1, one (2%) AVLAYAH-treated patient experienced anaphylaxis, which occurred in the first month of treatment.
Table 4 summarizes adverse reactions that occurred in >15% of AVLAYAH-treated pediatric patients with Hunter syndrome.
Description of Selected Adverse Reactions
Infusion-Associated Reaction
Three (6%) AVLAYAH-treated patients experienced severe IARs. One patient permanently discontinued treatment due to an IAR.
Anemia
Two (4%) AVLAYAH-treated patients experienced severe anemia (defined as hemoglobin <8 g/dL) prior to Week 24. One (2%) AVLAYAH-treated patient, aged 0.5 years, experienced moderate anemia (hemoglobin 9.2 g/dL), which was considered serious due to the patient's age.
Membranous Nephropathy
A case of biopsy-confirmed, steroid-refractory membranous nephropathy with immune complex deposits in the kidney was reported in an AVLAYAH-treated patient.
5DESCRIPTION
Tividenofusp alfa-eknm is a fusion protein consisting of the hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme iduronate-2-sulfatase (IDS) fused to the N-terminus of an immunoglobulin G1 (IgG1) fragment, crystallizable (Fc). It is produced by recombinant DNA technology in Chinese Hamster Ovary (CHO) cells. The approximate molecular weight of tividenofusp alfa-eknm is 110 kDa.
AVLAYAH (tividenofusp alfa-eknm) for injection is a sterile, preservative-free, white to off-white lyophilized powder with a cake-like appearance for intravenous infusion after reconstitution and dilution. Each single-dose vial contains 150 mg tividenofusp alfa-eknm, and the inactive ingredients dibasic sodium phosphate (5 mg), methionine (7.5 mg), monobasic sodium phosphate (7.7 mg), polysorbate 20 (3 mg), sodium chloride (14.6 mg), and sucrose (300 mg). The pH is 6.5 after reconstitution.
6CLINICAL STUDIES
Trial 1 (NCT04251026) was a Phase 1/2 multi-center, international, multi-cohort, single-arm, open-label trial of AVLAYAH in 47 pediatric patients with Hunter syndrome including 44 patients with neuronopathic Hunter syndrome and 3 patients with non-neuronopathic Hunter syndrome. Of the 47 patients enrolled, 46 patients completed part 1 of the study (up to Week 24) and 1 patient discontinued the study due to an adverse reaction prior to Week 24. In part 1 of Trial 1, patients received a starting dosage of AVLAYAH ranging from 3 mg/kg to 15 mg/kg weekly and a maximum dosage ranging from 3 mg/kg to 30 mg/kg weekly (0.2 to 2 times the approved recommended maintenance dosage)
All 47 patients in Trial 1 were male, with a baseline median age of 5 years (range: 3 months to 13 years of age). The patient population consisted of 27 White (57%), 4 Black or African American (9%), 4 Asian (9%), 3 who were more than one race (6%), 1 of another race (2%), and 8 of an unknown race (17%). Ethnicity consisted of 7 patients who were Hispanic or Latino (15%), 38 who were Not Hispanic or Latino (81%), and 2 of an unknown ethnicity (4%). Of the 47 patients, 15 patients were enzyme replacement therapy (ERT)-naïve, and 32 patients were ERT-experienced, 2 of whom had a history of prior treatment for Hunter syndrome with hematopoietic stem cell transplantation (HSCT), and 2 of whom had a history of prior treatment for Hunter syndrome with gene therapy. The median duration of previous ERT treatment was 26 months (range: 1 to 134 months).
In Trial 1, treatment with AVLAYAH resulted in a significant reduction of CSF HS. For the 44 patients who had measurements at Week 24, the CSF HS mean percent reduction from baseline was 91% (95% CI: 89%, 92%); the minimum and maximum percent change in CSF HS from baseline were 72% and 98%, respectively. At baseline, 0% (0 of 47) of patients had CSF HS levels below the upper limit of normal (ULN). At Week 24, 93% (41 of 44) of AVLAYAH-treated patients had CSF HS levels below the ULN.
7HOW SUPPLIED/STORAGE AND HANDLING
How Supplied
AVLAYAH (tividenofusp alfa-eknm) for injection is supplied as a sterile, preservative-free, white to off-white lyophilized powder with a cake-like appearance in a single-dose vial. Each vial contains 150 mg of tividenofusp alfa-eknm. AVLAYAH is available as one carton containing a 150 mg single-dose vial (NDC 84976-001-01).
8PATIENT COUNSELING INFORMATION
Hypersensitivity Reactions Including Anaphylaxis, and Infusion-Associated Reactions
Advise the patient and/or caregiver that life-threatening hypersensitivity reactions, including anaphylaxis, and infusion-associated reactions (IARs) may occur with AVLAYAH treatment.
Advise the patient and/or caregiver that anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
Inform the patient and/or caregiver of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis, and IARs and to seek immediate medical care should symptoms occur
Anemia
Inform the patient and caregiver that anemia may occur during AVLAYAH treatment. Advise the patient and/or caregiver of the symptoms of anemia (e.g., fatigue, pallor). Instruct the patient and/or caregiver to contact their healthcare provider if those symptoms occur
9PRINCIPAL DISPLAY PANEL - 150 mg Vial Label
Rx only
AVLAYAH™
150 mg/vial
For Intravenous Infusion after
10PRINCIPAL DISPLAY PANEL - 150 mg Vial Carton
NDC 84976-001-01
AVLAYAH™
150 mg/vial
For Intravenous Infusion after
One 150 mg Single-Dose Vial
DEnALI
