sebelipase alfa

Trade name: kanuma

Recombinant proteinapprovedOrphan Drug ^FDAFast Track ^FDABreakthrough Therapy ^FDA

Approved

Dec 8, 2015

Sebelipase alfa, sold under the brand name Kanuma, is a recombinant form of the enzyme lysosomal acid lipase (LAL) that is used as a medication for the treatment of lysosomal acid lipase deficiency (LAL-D). It is administered via intraveneous infusion. It was approved for medical use in the European Union and in the United States in 2015. — Wikipedia

Clinical trial activity

8 trials · 1 clinical orgs · 1 marketing orgs

Phase 1

Phase 2

Phase 3

Phase 4

Earliest trial started May 1, 2011 (NCT01307098)

Timeline

2010s

Jul 1, 2010
Orphan Drug Designation
Jan 1, 2011
Alexion — Earliest Phase 1 Sponsor(trial)
Jan 1, 2011
Alexion — Earliest Phase 2 Sponsor(trial)
Jan 1, 2013
Alexion — Earliest Phase 3 Sponsor(trial)
Dec 8, 2015
Alexion — Earliest FDA Approval
Dec 8, 2015
Alexion — Marketing Organization
Dec 8, 2015
Alexion — NDA Organization

Indications

Approved for

Cholesterol Ester Storage Disease

Studied for

Mechanism of action

TriglycerideHYDROLYTIC ENZYME
Triglyceride hydrolytic enzyme
Cholesterol esterHYDROLYTIC ENZYME
Cholesterol ester hydrolytic enzyme
Lysosomal acid lipase/cholesteryl ester hydrolaseSupplementation
LIPA supplementation
- Lysosomal acid lipase/cholesteryl ester hydrolase (LIPA) ↗

Approval history

approvedFast trackBreakthroughDec 8, 2015

Chemistry & pharmacology

Chirality: Single Stereoisomer
Inorganic: No
Polymer: No
Delivery: Parenteral
Availability: Prescription Only
Multi-specific: No

Oral

Parenteral

Yes

Topical

Black box warningFirst in class

Sources

Also known as

kanuma
kanuma
kanuma
kanuma
lipa
sbc-102
sbc-102
sbc-102
sebelipase alfa
sebelipase alfa
sebelipase alfa
sebelipase alfa
sebelipase alfa
sebelipase alfa
sebelipase alfa
sebelipase alfa
sebelipase alfa
sebelipase alfa

Clinical trials

NCT ID	Phase	Start date	Sponsor(s)
NCT02376751	N/A	Feb 25, 2015	Alexion
NCT02193867	Phase 2	Jun 1, 2014	Alexion
NCT02112994	N/A	May 1, 2014	Alexion
NCT01757184	Phase 3	Jan 1, 2013	Alexion
NCT01488097	Phase 2	Nov 1, 2011	Alexion
NCT01473875	Phase 2/Phase 3 (Phase 3)	Nov 1, 2011	Alexion
NCT01307098	Phase 1	May 1, 2011	Alexion
NCT01371825	Phase 2/Phase 3 (Phase 3)	May 1, 2011	Alexion

Showing 8 of 8 trials

Page 1 / 1

Organizations

Research & Development (1)

Organization	Org type	Trials	As lead sponsor	Phases	Earliest year
Alexion	For profit	8	8	4	2011

1 organizations

Page 1 / 1

Marketing (2)

Organization	Org type	Relationship	Date
Alexion	For profit	MKTG	Dec 8, 2015
Alexion	For profit	NDA	Dec 8, 2015