Tividenofusp alfa-eknm

What Tividenofusp alfa-eknm is used for

Quoted from the “Indications and Usage” section of the FDA-approved label.

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 […]. …

— FDA label: Avlayah, Denali Therapeutics Inc., effective March 20, 2026. Read the full label on DailyMed →

Label quoted: the one for the most common form (injection, powder, lyophilized, for solution, intravenous), Avlayah, Denali Therapeutics Inc.; it covers 1 product in the FDA NDC Directory. The side effects, interactions, dosage and pregnancy quotes below come from this same label.

This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.

Boxed warning

A boxed warning (“black box”) is the FDA’s strongest warning on a drug label, for serious or life-threatening risks.

This label has a boxed warning. Its heading: WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS. Read it on the full label →

Tividenofusp alfa-eknm boxed warning: what the label says

Quoted from the boxed warning of the FDA-approved label.

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.

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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 [see Warnings and Precautions (5.1)]. WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. (5.1) Initiate AVLAYAH in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. (5.1) If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue AVLAYAH and immediately initiate appropriate medical treatment, including use of epinephrine. (5.1)

— FDA label: Avlayah, Denali Therapeutics Inc., effective March 20, 2026. Read the full label on DailyMed →

Tividenofusp alfa-eknm side effects

Quoted from the “Adverse Reactions” section of the FDA-approved label. Tell your doctor about any side effect that worries you.

The following clinically significant adverse reactions are described elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1)] Infusion-Associated Reactions [see Warnings and Precautions (5.2)] Anemia [see Warnings and Precautions (5.3)] Membranous Nephropathy [see Warnings and Precautions (5.4)] Most common adverse reactions (incidence ≥20%) were IAR, upper respiratory tract infection, ear infection, pyrexia, anemia, cough, vomiting, diarrhea, rash, COVID-19, rhinorrhea, nasal congestion, fall, headache, skin abrasion, and urticaria.

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(6.1) To report SUSPECTED ADVERSE REACTIONS, contact Denali Therapeutics toll-free at 1-833-ONE-DNLI (1-833-663-3654) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical 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 [see Clinical Studies (14)]. A total of 47 male patients (age range: 3 months to 13 years) received intravenous AVLAYAH at 3 mg/kg to 30 mg/kg (0.2 to 2 times the approved recommended maintenance dose) weekly, and the majority of patients received 15 mg/kg intravenously weekly after Week 24. The median (minimum, maximum) duration of exposure was 117 (19, 219) weeks. 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. Table 4: Adverse Reactions That Occurred in >15% in AVLAYAH-treated Pediatric Patients With Hunter Syndrome (Trial 1) Adverse Reaction Any Severity N (%) (N = 47) a Infusion-associated reaction includes infusion-related reaction. b Ear infection includes ear infection, otitis media, otitis media acute, otitis externa. c Anemia includes anemia, iron deficiency anemia, and decreased hemoglobin.

— FDA label: Avlayah, Denali Therapeutics Inc., effective March 20, 2026. This is part of the section. Read the full label on DailyMed →

Tividenofusp alfa-eknm dosage

Quoted from the “Dosage and Administration” section of the FDA-approved label. Your own dose is the one your doctor prescribes: do not change it on your own.

Administration of AVLAYAH should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. (2.1) Obtain a baseline hemoglobin value in all patients. (2.1) Recommended AVLAYAH maintenance dosage for pediatric patients who weigh at least 5 kg is 15 mg/kg administered once weekly as an intravenous infusion over approximately 4 hours. (2.2, 2.6) Initiate AVLAYAH treatment with a dose escalation regimen.

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(2.2) See the full prescribing information for dosage and administration modifications and monitoring. (2.3) See the full prescribing information for preparation and administration instructions. (2.4, 2.6) 2.1 Important Recommendations Prior to AVLAYAH Treatment Initiation Administer AVLAYAH under the supervision of a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1)]. Initiate AVLAYAH in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1)]. Consider pretreatment with antihistamines, antipyretics, and/or corticosteroids [see Warnings and Precautions (5.1, 5.2)]. Obtain a baseline hemoglobin value in all patients [see Warnings and Precautions (5.3)]. 2.2 Recommended Dosage The recommended starting dosage of AVLAYAH for pediatric patients weighing at least 5 kg is 3 mg/kg administered once weekly via intravenous infusion. To reduce the risk of infusion-associated reactions (IARs), follow the dose escalation regimen in Table 1 [see Warnings and Precautions (5.2)]. Administer each dosage level for at least 4 weeks before escalating to the next dosage level. The recommended maintenance dosage of AVLAYAH for pediatric patients who weigh at least 5 kg is 15 mg/kg administered once weekly via intravenous infusion. Table 1: Recommended AVLAYAH Dosage for Pediatric Patients Weighing ≥5 kg a Dosing Week Dosage Level a Do not escalate the dosage level if the current dosage level is not tolerated [see Dosage and Administration (2.3)]. Week 1 to Week 4 3 mg/kg once weekly Week 5 to Week 8 7.5 mg/kg once weekly Week 9 and beyond 15 mg/kg once weekly (maintenance dosage) 2.3 Dosage and Administration Modifications and Monitoring In the event of a severe hypersensitivity reaction (e.g., anaphylaxis) or a severe IAR, discontinue AVLAYAH and immediately initiate appropriate medical treatment. Consider the risks and benefits of re-administering AVLAYAH following a severe reaction. If the decision is made to re-administer AVLAYAH, re-evaluate pre-treatment medications, slow the infusion rate, and/or reduce the AVLAYAH dose. Monitor patients closely upon re-administration of AVLAYAH [see Warnings and Precautions (5.1, 5.2)]. In the event of a mild to moderate hypersensitivity reaction or a mild to moderate IAR, temporarily hold the infusion and/or reduce the infusion rate by at least 50% from the current rate, then titrate up to the recommended infusion rate as tolerated (see Table 3) [see Warnings and Precautions (5.1, 5.2)].

— FDA label: Avlayah, Denali Therapeutics Inc., effective March 20, 2026. This is part of the section. Read the full label on DailyMed →

Tividenofusp alfa-eknm in pregnancy

Quoted from the pregnancy part of the FDA-approved label. Ask your doctor before you take any medicine while pregnant or planning a pregnancy.

Risk Summary There are no available data on the use of AVLAYAH during pregnancy to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Animal studies to evaluate the potential for embryofetal developmental toxicity and pre- and postnatal developmental toxicity of tividenofusp alfa-eknm have not been conducted. The background risk of major birth defects and miscarriage for the indicated population is unknown.

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All pregnancies have a background risk of birth defects, loss, and other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

— FDA label: Avlayah, Denali Therapeutics Inc., effective March 20, 2026. Read the full label on DailyMed →

Forms and how it is given

Companies that list this medicine

Labelers in the FDA NDC Directory: the company named on the package (maker or distributor), with the number of prescription products.

• Denali Therapeutics Inc.1 product

FDA approval history

• BLA: Biologics License Application: how a biologic medicine (made from living cells) is approved.

Shortage status

The FDA drug shortage list does not show these products.

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Recalls

No recall linked to these products (by their NDC code) appears in the FDA enforcement reports.

Official identifiers

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Sources

1. FDA-approved label on DailyMed (National Library of Medicine) — Avlayah, Denali Therapeutics Inc., effective March 20, 2026; via openFDA, FDA data exported October 30, 2026, retrieved October 31, 2026
2. NDC Directory (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
3. Drugs@FDA (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
4. Drug recall enforcement reports (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 27, 2026, retrieved October 31, 2026
5. Drug shortages (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026

openFDA data is provided by the FDA as is; it may be incomplete. Always rely on the label that comes with your medicine. FDA Enforcement Report search →

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