Table 1.

Summary of Included Products and Key Characteristics

Product Name (Brand)	INN	Drug or Biologic	Indication	Rare Disease	Gene Therapy	FDA			EMA
						Date of Approval (Gray if Not FDA Approved in Study Period)	Orphan Designation	Priority Review	Date of Authorization (Gray if Not EMA Approved in Study Period)	Orphan Designation	Accelerated Assessment
Camzyos [20]	mavacamten	Drug	Adults with (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM)	Yes	No	4/28/2022	Yes	Yes
Amvuttra [21, 22]	vutisiran	Drug	Polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults	Yes	No	6/13/2022	Yes	No	9/15/2022	Yes	No
Pyrukynd [23, 24]	mitapivat	Drug	Hemolytic anemia in adults with pyruvate kinase (PK) deficiency	Yes	No	2/17/2022	Yes	Yes	11/9/2022	Yes	No
Enjaymo [25, 26]	sutimlimab	Drug	Hemolysis in adults with cold agglutinin disease (CAD)	Yes	No	2/4/2022	Yes	Yes	11/15/2022	Yes	no
Nextstellis [27]	etonorgestrel and estetrol	Drug	Females of reproductive potential to prevent pregnancy	No	No	4/15/2021	No	No
Nulibry [28, 29]	fosdenopterin	Drug	Molybdenum cofactor deficiency (MoCD) Type A	Yes	No	2/26/2021	Yes	Yes	9/15/2022	Yes	No
Imcivree [30, 31]	setmelanotide	Drug	Obesity due to proopiomelanocortin (POMC) or proprotein convertase subtilisin/kexin type 1 (PCSK1) deficiency	Yes	No	11/25/2020	Yes	Yes	7/16/2021	Yes	No
Zokinvy [32, 33]	lonafarnib	Drug	Processing-deficient Progeroid Laminopathies with either heterozygous LMNA mutation with progerin-like protein accumulation or ZMPSTE24 mutations	Yes	No	11/20/2020	Yes	Yes	7/18/2022	Yes	No
Dojolvi [34]	triheptanoin	Drug	Pediatric and adult patients with long-chain fatty acid oxidation disorders (LC-FAOD)	Yes	No	6/30/2020	Yes	No
Pretomanid/Dovprela [35, 36]	pretomanid	Drug	Pulmonary extensively drug resistant (XDR) or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB)	No	No	8/14/2019	Yes	Yes	7/31/2020	Yes	No
Vyondys 53 [37]	golodirsen	Drug	Duchenne muscular dystrophy (DMD) with amenable to exon 53 skipping	Yes	No	12/12/2019	Yes	Yes
Egaten [38]	triclabendazole	Drug	Fascioliasis in patients 6 years of age and older	No	No	2/13/2019	Yes	Yes
Skysona [39]	elivaldogene autotemcel	Biologic	Boys 4–17 years of age with early, active cerebral adrenoleukodystrophy (CALD)	Yes	Yes	16-Sep-22	Yes	Yes
Zynteglo [40]	betibeglogene autotemce	Biologic	ß-thalassemia	Yes	Yes	19-Aug-22	Yes	Yes
Rethymic [41]	allogeneic processed thymus tissue–agdc	Biologic	Congenital athymia	Yes	No	10/8/2021	Yes	Yes
Ryplazim [42]	plasminogen, human-tvmh	Biologic	Plasminogen deficiency type 1 (hypoplasminogenemi)	Yes	No	6/4/2021	Yes	Yes
Xembify [43]	immune globulin subcutaneous human-klhw	Biologic	Primary Humoral Immunodeficiency (PI) in patients 2 years of age and older	Yes	No	7/3/2019	No	No
Zolgensma [44, 45]	Onasemnogene abeparvovec	Biologic	Spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene	Yes	Yes	5/24/2019	Yes	Yes	5/18/2020	Yes	No
Asceniv [46]	mmune globulin intravenous, human – slra	Biologic	Primary humoral immunodeficiency in adults and adolescents (12 to 17 years of age)	Yes	No	4/1/2019	No	No
Esperoct [47, 48]	turoctocog alfa pegol	Biologic	Adults and children with hemophilia A	Yes	No	2/19/2019	No	No	6/20/2019	No	No
Mycapssa [49]	betibeglogene autotemce	Drug	Adult patients with acromegaly who have responded to and tolerated treatment with somatostatin analogues	Yes	No				12/2/2022	Yes	No
Roctavian [50]	valoctocogene roxaparvovec	Biologic	Severe haemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).,	Yes	Yes				6/23/2022	Yes	No
Upstaza [51]	eladocagene exuparvovec	Biologic	L amino acid decarboxylase (AADC) deficiency with a severe phenotype	Yes	Yes				5/19/2022	Yes	No
Voraxaze [52]	glucarpedase	Biologic	Adults and children (aged 28 days and older) with delayed methotrexate elimination or at risk of methotrexate toxicity	No	No				11/11/2021	Yes	No
Lydisilka [53]	drospirenone and estestrol	Drug	Oral contraception	No	No				3/25/2021	Yes	No
Libmeldy [54]	atidarsagene autotemcel	Biologic	Metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene	Yes	Yes				10/15/2020	Yes	No
Idefirix [55]	imlifidase	Biologic	Highly sensitised adult kidney transplant patients with positive crossmatch against an available deceased donor	Yes	No				6/25/2020	Yes	No
Cufence [56]	trientine dihydrochloride	Drug	Wilson’s disease in patients intolerant to D-Penicillamine therapy	Yes	No				5/29/2019	Yes	No