Tividenofusp alfa
| Clinical data | |
|---|---|
| Trade names | Avlayah |
| Other names | DNL-310, tividenofusp alfa-eknm |
| AHFS/Drugs.com | |
| License data | |
| Routes of administration | Intravenous |
| ATC code |
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| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C4990H7554N1305O1487S29 |
| Molar mass | 110548.71 g·mol−1 |
Tividenofusp alfa, sold under the brand name Avlayah, is an enzyme replacement therapy used for the treatment of Hunter syndrome.[2] Tividenofusp alfa is a hydrolytic lysosomal glycosaminoglycan-specific enzyme.[1]
The most common side effects include upper respiratory tract infection, ear infection, fever, anemia, cough, vomiting, diarrhea, rash, COVID-19, runny nose, nasal congestion, fall, headache, skin abrasion, and hives.[2]
Tividenofusp alfa was approved for medical use in the United States in March 2026.[2]
Medical uses
Tividenofusp alfa is indicated for the treatment of neurologic manifestations of Hunter syndrome (mucopolysaccharidosis type II) when initiated in presymptomatic or symptomatic children weighing at least 5 kg (11 lb) prior to advanced neurologic impairment.[1][2]
Hunter syndrome is a rare inherited lysosomal disorder in which sugar molecules called glycosaminoglycans build up within the cells' lysosomes.[2] This substrate accumulation affects physical and mental development by causing abnormalities in the skeleton, heart, respiratory system, brain, and other organs.[2]
Adverse effects
The US prescription label includes a boxed warning for allergic reactions including anaphylaxis.[2]
The most common side effects include upper respiratory tract infection, ear infection, fever, anemia, cough, vomiting, diarrhea, rash, COVID-19, runny nose, nasal congestion, fall, headache, skin abrasion, and hives.[2]
Society and culture
Legal status
Tividenofusp alfa was approved for medical use in the United States in March 2026.[2][3] The US Food and Drug Administration (FDA) granted the application for tividenofusp alfa breakthrough therapy, fast track, priority review, and orphan drug designations.[2] The FDA granted accelerated approval for Avlayah to Denali Therapeutics.[2]
Names
Tividenofusp alfa is the international nonproprietary name.[4]
Tividenofusp alfa is sold under the brand name Avlayah.[2]
References
- 1 2 3 "Avlayah- tividenofusp alfa-eknm injection, powder, lyophilized, for solution". DailyMed. 20 March 2026. Retrieved 8 May 2026.
- 1 2 3 4 5 6 7 8 9 10 11 12 "FDA Approves Drug to Treat Neurologic Manifestations of Hunter Syndrome". U.S. Food and Drug Administration. 25 March 2026. Retrieved 6 April 2026.
This article incorporates text from this source, which is in the public domain. - ↑ "Denali Therapeutics Announces U.S. FDA Approval of Avlayah (tividenofusp alfa-eknm) for Treatment of Hunter Syndrome (MPS II)". Denali Therapeutics (Press release). 25 March 2026. Retrieved 6 April 2026.
- ↑ World Health Organization (2023). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 90". WHO Drug Information. 37 (3). hdl:10665/373341.
External links
- Clinical trial number NCT04251026 for "A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome" at ClinicalTrials.gov