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Kanuma, sebelipase alfa (formerly SBC-102)

Also known as: rhLAL

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Company Alexion Pharmaceuticals Inc.
DescriptionRecombinant human lysosomal acid lipase (LAL) enzyme replacement therapy (ERT)
Molecular Target
Mechanism of ActionEnzyme replacement therapy
Therapeutic ModalityBiologic: Enzyme
Latest Stage of DevelopmentRegistration
Standard IndicationGlycosphingolipid storage disorders
Indication DetailsTreat early onset LAL deficiency (Wolman disease); Treat LAL deficiency (Wolman disease); Treat late onset LAL deficiency (cholesteryl ester storage disease)
Regulatory Designation U.S. - Breakthrough Therapy (Treat early onset LAL deficiency (Wolman disease));
U.S. - Fast Track (Treat early onset LAL deficiency (Wolman disease));
U.S. - Orphan Drug (Treat early onset LAL deficiency (Wolman disease));
U.S. - Orphan Drug (Treat LAL deficiency (Wolman disease));
U.S. - Priority Review (Treat LAL deficiency (Wolman disease));
EU - Orphan Drug (Treat LAL deficiency (Wolman disease));
EU - Orphan Drug (Treat early onset LAL deficiency (Wolman disease));
Japan - Orphan Drug (Treat early onset LAL deficiency (Wolman disease));
Switzerland - Orphan Drug (Treat early onset LAL deficiency (Wolman disease));
Switzerland - Orphan Drug (Treat late onset LAL deficiency (cholesteryl ester storage disease))
Partner

 Product Details
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Total Number of DealsTotal Deal ValueTotal Upfront CashTotal Milestone Payments

2

$8,788.4M

$4,473.5M

0


 Deals Details
DateCompanies InvolvedDeal HeadlineTotal Deal ValueTotal Upfront CashTotal Milestone
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