TABLE 3.

Pediatric Orphan Indications for Drugs Receiving FDA Breakthrough Designation

Generic Name	Trade Name	Marketing Approval Date	Category	Disease Population(s) Targeted by Indication
Canakinumab	Ilaris	September 23, 2016	Non-novel, rare disease only	Tumor necrosis factor receptor–associated periodic syndrome
Canakinumab	Ilaris	September 23, 2016	Non-novel, rare disease only	Hyperimmunoglobulin D syndrome; mevalonate kinase deficiency
Canakinumab	Ilaris	September 23, 2016	Non-novel, rare disease only	Familial Mediterranean fever
Ivacaftor	Kalydeco	February 21, 2014	Non-novel, rare disease only	Patients with cystic fibrosis aged ≥6 y with certain CFTR mutations (eg, G1224E, G1349D)
Ivacaftor	Kalydeco	December 29, 2014	Non-novel, rare disease only	Patients with cystic fibrosis aged ≥6 y with certain R117H CFTR mutation
Ivacaftor	Kalydeco	March 17, 2015	Non-novel, rare disease only	Patients with cystic fibrosis aged 2–5 y with certain CFTR mutations (eg, G1224E, G1349D)
Lumacaftor–ivacaftor	Orkambi	September 28, 2016	Non-novel, rare disease only	Patients with cystic fibrosis aged 6–11 y homozygous for the F508del CFTR mutation
Lumacaftor–ivacaftor	Orkambi	August 7, 2018	Non-novel, rare disease only	Patients with cystic fibrosis aged 2–5 y homozygous for the F508del CFTR mutation
Blinatumomab	Blincyto	December 3, 2014	Novel drug	Philadelphia chromosome–negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia
Cerliponase alfa	Brineura	April 27, 2017	Novel drug	Patients aged ≥3 y with tripeptidyl peptidase 1 deficiency
Burosumab-twza	Crysvita	April 17, 2018	Novel drug	X-linked hypophosphatemia in patients aged ≥1 y
Emapalumab-lzsg	Gamifant	November 20, 2018	Novel drug	Hemophagocytic lymphohistiocytosis
Emicizumab-kxwh	Hemlibra	November 16, 2017	Novel drug	Hemophilia A
Sebelipase alfa	Kanuma	December 8, 2015	Novel drug	Lysosomal acid lipase
Uridine triacetate	n/a	September 4, 2015	Novel drug	Hereditary orotic aciduria
Inotuzumab ozogamicin	n/a	August 17, 2017	Novel drug	Relapsed or refractory B-cell precursor acute lymphoblastic leukemia
Tezacaftor and ivacaftor	n/a	February 12, 2018	Novel drug	Patients with cystic fibrosis aged ≥12 y homozygous for the F508del CFTR mutation
Lumacaftor–ivacaftor	Orkambi	July 2, 2015	Novel drug	Patients with cystic fibrosis aged ≥12 y homozygous for the F508del CFTR mutation
Asfotase alfa	Strensiq	October 23, 2015	Novel drug	Perinatal-, infantile-, and juvenile-onset hypophosphatasia
Lanadelumab-flyo	Takhzyro	August 23, 2018	Novel drug	Patients with hereditary angioedema aged ≥12 y

n/a, not applicable.