Drugs / efzimfotase alfa
efzimfotase alfa
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | hypophosphatasia | 2026-09-18 | “Biologics License Application (BLA) for efzimfotase alfa has been accepted and granted Priority Review by the US Food and Drug Administration (FDA)” astrazeneca.com ↗ |
| Filed | Japan (PMDA) | — | hypophosphatasia | — | “Regulatory submissions for efzimfotase alfa based on MULBERRY, CHESTNUT and HICKORY are also under review in Japan and other markets” astrazeneca.com ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT04980248 | Sep 2021 → Aug 2022 | hypophosphatasia | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 33 trials · 1 met primary · 1 missed | ||||||
| Phase 3 | NCT06079359 | May 2024 → Jan 2026 overdue | hypophosphatasia | Alexion Pharmaceuticals, Inc. | Active not recruiting | Met primary |
| Phase 3 | NCT06079372 | Apr 2024 → Jul 2025 overdue | hypophosphatasia | Alexion Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT06079281 | Jan 2024 → Jul 2025 overdue | hypophosphatasia | Alexion Pharmaceuticals, Inc. | Active not recruiting | Missed primary |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2026-06-28 | AstraZeneca | Results Efzimfotase alfa demonstrated improvements in bone health in treatment-naïve paediatric patients with hypophosphatasia achieving median difference of 1.67 vs placebo in RGI-C Score at week 25 in MULBERRY Phase III trial astrazeneca.com ↗
Positive results from the MULBERRY Phase III trial showed that efzimfotase alfa (ALXN1850), an investigational enzyme replacement therapy, demonstrated a statistically significant and clinically meaningful improvement in bone health in children (2 to <12 years of age) with hypophosphatasia (HPP) who have not been previously treated with Strensiq (asfotase alfa) as measured by Radiographic Global Impression of Change (RGI-C) Score at week 25 compared to placebo. |
| 2026-03-31 | AstraZeneca | Results Efzimfotase alfa demonstrated positive results from global Phase III clinical programme in hypophosphatasia astrazeneca.com ↗
The efzimfotase alfa (ALXN1850) Phase III clinical programme, designed to study a broad hypophosphatasia (HPP) patient population, demonstrated positive results. |
All press releases naming this drug 3 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 5 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | efzimfotase alfa | “Efzimfotase alfa (ALXN1850) is a second-generation TNSALP enzyme replacement therapy in development for HPP.” PMID 39135540 ↗ Sep 2024 |
| Known as | ALXN1850 | “Efzimfotase alfa (ALXN1850) is a second-generation TNSALP enzyme replacement therapy in development for HPP.” PMID 39135540 ↗ Sep 2024 |
| Known as | Efzimfotasa alfa | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314736 ↗ |
| Known as | TPP-2027 | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314736 ↗ |
| Known as | YRO201 | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314736 ↗ |
| Modality | Protein / enzyme biologic | “Efzimfotase alfa (ALXN1850) is a second-generation TNSALP enzyme replacement therapy in development for HPP.” PMID 39135540 ↗ Sep 2024 |
| Route | Intravenous | “received efzimfotase alfa in doses of 15 mg (cohort 1), 45 mg (cohort 2), or 90 mg (cohort 3) as one intravenous (i.v.) dose followed by 3 weekly subcutaneous (s.c.) doses.” PMID 39135540 ↗ Sep 2024 |
| Route | Subcutaneous | “received efzimfotase alfa in doses of 15 mg (cohort 1), 45 mg (cohort 2), or 90 mg (cohort 3) as one intravenous (i.v.) dose followed by 3 weekly subcutaneous (s.c.) doses.” PMID 39135540 ↗ Sep 2024 |