TABLE 2.
Examples of Pediatric Orphan Indications for Non-Novel Drugs
| Generic Name (Trade Name) | Pediatric Orphan Indication | Date of Initial FDA Approval for Drug’s Active Moiety | Orphan Drug Exclusivity Start Date | Years Between Initial FDA Approval and Orphan Drug Exclusivity (Vintage) | Approved Only To Treat Rare Diseases When Orphan Drug Exclusivity Began | Examples of Previous FDA-Approved Indications When Orphan Drug Exclusivity Began |
|---|---|---|---|---|---|---|
| Lumacaftor–ivacaftor (Orkambi) | Cystic fibrosis in children aged 6–11 y homozygous for the F508del CFTR mutation | July 2, 2015 | September 28, 2016 | 1.2 | Yes | Cystic fibrosis in adults and children aged ≥12 y homozygous for the F508del CFTR mutation |
| Lumacaftor–ivacaftor (Orkambi) | Cystic fibrosis in children aged 2–5 y homozygous for the F508del CFTR mutation | July 2, 2015 | August 7, 2018 | 3.1 | Yes | Cystic fibrosis in adults and children aged ≥6 y homozygous for the F508del CFTR mutation |
| Ivacaftor (Kalydeco) | Cystic fibrosis in children aged ≥2 y with specific CFTR mutations (eg, 711+3A G, E831X) | January 31, 2012 | July 31, 2017 | 5.5 | Yes | Cystic fibrosis in children aged ≥2 y with other CFTR mutations (eg, E56K, P67L, R74W) |
| Canakinumab (Ilaris) | Tumor necrosis factor receptor–associated periodic syndrome in pediatric and adult patients | June 17, 2009 | September 23, 2016 | 7.3 | Yes | Cryopyrin-associated periodic syndromes in adults and children aged ≥4 y; systemic juvenile idiopathic arthritis in adults and children aged ≥2 y |
| Ledipasvir–sofosbuvir (Harvoni) | Chronic hepatitis C in children aged 12 and older or who weigh ≥35 kg | October 10, 2014 | April 7, 2017 | 2.5 | No | Chronic hepatitis C in adults |
| Adalimumab (Humira) | Juvenile idiopathic arthritis in children aged 2–3 y | December 31, 2002 | September 30, 2014 | 11.8 | No | Adults with autoimmune diseases such as rheumatoid arthritis, ankylosing spondylitis, Crohn disease; children aged ≥4 y with juvenile idiopathic arthritis; children ≥6 y with Crohn disease |
| AbobotulinumtoxinA (Dysport)a | Lower limb spasticity in children aged ≥2 y with cerebral palsy | December 9, 1991 | July 29, 2016 | 24.7 | No | Adults with cervical dystonia; cosmesis in non-elderly adults with glabellar lines |
| Propranolol hydrochloride (Hemangeol) | Proliferating infantile hemangioma | November 13, 1967 | March 14, 2014 | 46.4 | No | Hypertension, arrhythmias migraine, myocardial infarctionb |
| Hydroxyurea (Siklos) | Children ≥2 y with sickle cell anemia | December 7, 1967 | December 21, 2017 | 50.1 | No | Adults with certain types of head and neck cancer; adults with sickle cell anemiab |
a
AboboulinumtoxinA is a specific formulation of botulinum toxin type A, which was first FDA approved in 1991.
b
Siklos and Hemangeol have no other indications other than the pediatric orphan indication listed but are not new molecular entities because their active moieties had previously been approved by the FDA for other indications (examples of which are listed in the last column).