Sebelipase Alfa

Sold as Kanuma. Every FDA application for this active ingredient.

As of Nov 15, 2026, FDA lists 1 application for Sebelipase Alfa (sold as Kanuma), first approved Dec 8, 2015, with 1 new use approved and 0 published complete response letters.

First approved: Dec 8, 2015
Applications: 1
New uses approved: 1
Complete response letters: 0
Medicare spending, 2025: $801K
First approved in the EU: Aug 28, 2015
First approved in Japan: Mar 28, 2016

Medicare Spending

CMS Part D and Part B, 2025, before manufacturer rebates; matched by generic name

Product	Part	Spending, 2025	Change	Beneficiaries
Kanuma	Part D	$801K	New	0

Orphan Designations (FDA)

One designation, 1 approved for the designated use; see all orphan designations

Treatment of lysosomal acid lipase deficiencyAlexion Pharmaceuticals · designated Jul 1, 2010 · approved Dec 8, 2015

Approvals in the EU (EMA)

First approved in the EU 3 months before the US. Centrally authorised medicines, matched by ingredient; see all EU approvals

Kanuma, new substanceAlexion Europe SAS · Aug 28, 2015

Approvals in Japan (PMDA)

First approved in Japan 4 months after the US. PMDA's English list since 2008, matched by ingredient; see all Japanese approvals

Kanuma, new ingredientA drug with a new active ingredient indicated for the treatment of lysosomal acid lipase deficiency. · Alexion Pharma G.K. · Mar 28, 2016 · Review report

Clinical Trials

1 open or ongoing study names this ingredient as an intervention, including as a comparator; newest first. See all

Study	Phase	Status	Posted
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)MPS I, MPS II · University of California, San Francisco	Phase 1	Recruiting	Aug 31, 2020

Applications

NDAs, BLAs and generic ANDAs for this ingredient, oldest first; each opens its full history

Approved	Application	Sponsor	Supplements	Marketing
Dec 8, 2015	KanumaBLA 125561 · New molecular entity	Alexion Pharm	2	Marketed

Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA