Table 1 Application characteristics of NDAs and BLAs included in the systematic review
Therapy– Generic (US Brand name) | Approved Indication | Epidemiology of Disease | NDA/BLA | Review Type | Approval date |
|---|---|---|---|---|---|
Elivaldogene autotemcel (Skysona) [20] | Early cerebral adrenoleukodystrophy (CALD) | US prevalence: 35–40% of the 1:20,000 males affected with X-ALD | BLA | Priority | Sept 16, 2022* |
Vutrisiran (Amvuttra) [21] | Polyneuropathy (PN) of hereditary transthyretin mediated (hATTR) amyloidosis in adults | US incidence: 1/100,000 in U.S. Caucasians; US prevalence of hATTR-PN: 100 to 2,500 individuals | NDA | Standard | Jun 13, 2022 |
Vosoritide (Voxzogo) [22] | Increase in linear growth in children with achondroplasia ages 5 and older with open epiphyses | Global incidence: 1 in 25,000 births | NDA | Priority | Nov 19, 2021* |
Allogeneic processed thymus tissue- agdc (Rethymic) [23] | Immune reconstitution in pediatric patients with congenital athymia | US incidence: 20 newborns p.a. | BLA | Priority | Oct 8, 2021 |
Fosdenopterin (Nulibry) [24] | Molybdenum cofactor deficiency (MoCD) type A | US prevalence: 45 to 54 patients, all under 10 years of age; US incidence: 0.24–0.29 per 100,000 infants [25] | NDA | Priority | Feb 26, 2021 |
Lonafarnib (Zokinvy) [26] | Hutchinson-Gilford progeria syndrome (HGPS) and processing deficient progeroid laminopathies (PL) | HGPS Global incidence: 1 in 4Â million births; Global prevalence: 1 in 20Â million PL US prevalence: 1 in 25Â million. | NDA | Priority | Nov 20, 2020 |
Viltolarsen (Viltepso) [27] | Duchenne muscular dystrophy | Global incidence: 1 in 5,000 live male births; US prevalence: 16 cases per 100,000 live male births | NDA | Priority | Aug 12, 2020* |
Risdiplam (Evrysdi) [28] | Spinal muscular atrophy | Global incidence: 8.5 to 10.3 per 100,000 live births | NDA | Priority | Aug 7, 2020 |
Triheptanoin (Dojolvi) [29] | A source of calories and fatty acids in the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) | US, Germany, Australia incidence: 1 in 9,300 individuals | NDA | Standard | Jun 30, 2020 |
Pretomanid Tablet (Pretomanid) [30] | Pulmonary extensively drug-resistant (XDR) and treatment-intolerant/nonresponsive (TI/NR) multidrug-resistant (MDR) tuberculosis in adults | US reported cases in 2020 [31]: MDR-TB: 56 cases; XDR TB: 1 case | NDA | Priority | Aug 14, 2019 |
Onasemnogene abeparvovec-xioi (Zolgensma) [32] | Spinal muscular atrophy (SMA) | SMA type 1 global incidence: 1 in 10,000 live births; Global prevalence: 1–2 per 100,000 | BLA | Priority | May 24, 2019 |
Triclabendazole (Egaten) [33] | Fascioliasis | Global prevalence: 2.4 to 17Â million individuals | NDA | Priority | Feb 13, 2019 |
Stiripentol (Diacomit) [34] | Dravet syndrome | US incidence: 1 in 40,000 infants [35] | NDA | Priority | Aug 20, 2018 |
Fish oil triglycerides inj. Emulsion (Omegaven) [36] | Parenteral nutrition-associated cholestasis (PNAC) | Global incidence: 28.2% In children who received PN for > = 14 days | NDA | Priority | Jul 27, 2018 |
Burosumab (Crysvita) [37] | X-linked hypophosphatemia (XLH) | Global incidence: 1 in 20,000 live births; US prevalence: 3,000 pediatric and 12,000 adult patients | BLA | Priority | Apr 17, 2018 |
Voretigene neparvovec (Luxturna) [38] | Biallelic RPE65 mutation-associated retinal dystrophy | US prevalence: 1,000 to 2,000 individuals [39] | BLA | Priority | Dec 19, 2017 |
Emicizumab-kxwh (Hemlibra) [40] | Hemophilia A (congenital factor VIII deficiency) | US incidence:20,000 live births; Global incidence: 400,000 live births | BLA | Priority | Nov 16, 2017 |
Vestronidase alfa-vjbk (Mepsevii) [41] | Mucopolysaccharidosis type 7 (MPS VII) | Global prevalence: 1 in 250,000 | BLA | Priority | Nov 15, 2017 |
Cerliponase alfa (Brineura) [42] | Neuronal ceroid lipofuscinosis type 2 (CLN2) | US & Europe incidence: 0.56-4 pts per 100,000 live births | BLA | Priority | Apr 27, 2017 |
Thiotepa (Tepadina) [43] | Class 3 β-thalassemia | Global incidence: 100,000 children p.a. | NDA | Standard | Jan 26, 2017 |