Sebelipase alpha is a recombinant form of the enzyme lysosomal acid lipase . Received the status of orphan drug and approved for use: USA (2015) [1] .
It is made from egg protein of genetically modified chicken [2] [3] .
Indications
Lysosomal acid lipase deficiency [4] .
Contraindications
Not. Allergies to egg products should be considered [4] .
Notes
- ↑ New Molecular Entity and New Therapeutic Biological Product Approvals for 2015 . FDA
- ↑ Balwani M, Breen C, Enns GM, Deegan PB, Honzik T, Jones S, Kane JP, Malinova V, Sharma R, Stock EO, Valayannopoulos V, Wraith JE, Burg J, Eckert S, Schneider E, Quinn AG. Clinical Effect and Safety Profile of Recombinant Human Lysosomal Acid Lipase in Patients with Cholesteryl Ester Storage Disease (English) // Hepatology. - September 2013. - Vol. 58 , no. 3 . - P. 950–957 .
- ↑ US government approves transgenic chicken .
- ↑ 1 2 KANUMA-sebelipase alfa injection, solution, concentrate .