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ALXN2030
ALXN2030 is a Enzyme replacement therapy Small molecule drug developed by Alexion Pharmaceuticals, Inc.. It is currently in Phase 2 development for Mucopolysaccharidosis type I (MPS I).
ALXN2030 is a recombinant human alpha-L-iduronidase enzyme replacement therapy.
ALXN2030 is a recombinant human alpha-L-iduronidase enzyme replacement therapy. Used for Mucopolysaccharidosis type I (MPS I).
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Baseline phase 2 → approval rate
+15.3pp
Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Rare-disease pathway favourability
+5.0pp
Rare-disease drugs benefit from FDA Orphan Drug Act, smaller pivotal trials, and more flexible endpoints. Approval rates run ~5pp above baseline.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2031–2034 | — |
| EMA | EU | 2032–2035 | +0.7 yr |
| MHRA | GB | 2032–2035 | +0.7 yr |
| Health Canada | CA | 2032–2036 | +0.9 yr |
| TGA | AU | 2032–2036 | +1.2 yr |
| PMDA | JP | 2032–2036 | +1.5 yr |
| NMPA | CN | 2033–2037 | +2.3 yr |
| MFDS | KR | 2032–2036 | +1.4 yr |
| CDSCO | IN | 2032–2037 | +1.8 yr |
| ANVISA | BR | 2033–2037 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | ALXN2030 |
|---|---|
| Sponsor | Alexion Pharmaceuticals, Inc. |
| Drug class | Enzyme replacement therapy |
| Target | alpha-L-iduronidase |
| Modality | Small molecule |
| Therapeutic area | Genetics and Rare Diseases |
| Phase | Phase 2 |
Mechanism of action
It works by replacing the deficient enzyme in patients with mucopolysaccharidosis type I (MPS I), thereby reducing the accumulation of glycosaminoglycans in various tissues and organs.
Approved indications
- Mucopolysaccharidosis type I (MPS I)
Common side effects
- Infections
- Allergic reactions
- Pain or swelling at the injection site
Key clinical trials
- Phase 2 Study of ALXN2030 in Patients With Antibody-Mediated Rejection After Kidney Transplantation (PHASE2)
- Safety, PK/PD, and Immunogenicity Study of SC ALXN2030 in Healthy Participants (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- ALXN2030 CI brief — competitive landscape report
- ALXN2030 updates RSS · CI watch RSS
- Alexion Pharmaceuticals, Inc. portfolio CI
Frequently asked questions about ALXN2030
What is ALXN2030?
How does ALXN2030 work?
What is ALXN2030 used for?
Who makes ALXN2030?
What drug class is ALXN2030 in?
What development phase is ALXN2030 in?
What are the side effects of ALXN2030?
What does ALXN2030 target?
Related
- Drug class: All Enzyme replacement therapy drugs
- Target: All drugs targeting alpha-L-iduronidase
- Manufacturer: Alexion Pharmaceuticals, Inc. — full pipeline
- Therapeutic area: All drugs in Genetics and Rare Diseases
- Indication: Drugs for Mucopolysaccharidosis type I (MPS I)
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing