The Final Charge , Phase I/II MND Results & FDA ODD Approval Catalyst, page-865

Great News Quiltman , some serious sales made in the ODD Space , Table Below

Orphan drugs refer to the pharmaceuticals which are used for the diagnosis, prevention, and treatment of rare medical disorders. Rare diseases are a health condition which generally affects small number of people compared with other prevalent diseases in general population. It is estimated that globally around 6000-8000 rare diseases exist with new rare diseases being reported in medical literature regularly. As rare diseases have low prevalence and individually rare, collectively they affect around 6-8% of the population. These diseases include genetic disorders, rare cancers, infectious topical diseases, and degenerative diseases. Around 80% of the rare diseases are genetic in nature and therefore mainly affects children.

The last few years have seen rapid influx in approval of orphan drugs which is mainly attributed to the restless efforts by scientists and rising awareness among population

Key Orphan Drug Sales 2022

	Drug	Company	FDA Approval	Indication	2021 Sales
1	Keytruda	Merck	2014	melanoma, NSCLC, HNSCC, classical HL, primary mediastinal large B-cell lymphoma, urothelial carcinoma, CRC, gastric cancer, esophageal cancer, cervical cancer, hepatocellular carcinoma, Merkel cell carcinoma, renal cell carcinoma, endometrial carcinoma, tumor mutational burden-high (TMB-H) cancer, cutaneous squamous cell carcinoma, and TNBC.	US$ 17.18 Billion
2	Revlimid	Bristo Myers Squibb	2005	Multiple myeloma, Follicular Lymphoma, myelodysplastic syndromes, mantle cell lymphoma, marginal zonal lymphoma	US$ 12.8 Billion
3	Opdivo	Bristo Myers Squibb	2014	NSCLC, malignant pleural mesothelioma, renal cell carcinoma, classical Hodgkin lymphoma, HNSCC, urothelial carcinoma, CRC, hepatocellular carcinoma, esophageal cancer, gastric cancer, and gastroesophageal junction cancer.	US$ 7.52 Billion
4	Trikafta	Vertex Pharmaceutical	2019	Cystic Fibrosis	US$ 5.69 Billion
5	Imbruvica	AbbVie	2015	Mantle Cell Lymphoma; Chronic Lymphocytic Leukemia; Waldenström’s Macroglobulinemia; Small Lymphocytic Lymphoma; Marginal Zone Lymphoma	US$ 5.4 Billion
6	Hemlibra	Genentech	2017	Hemophilia A	US$ 2.35 Billion
7	Lynparza	AstraZeneca	2014	Ovarian Cancer, Fallopian Tube Cancer, Peritoneal Cancer, Breast Cancer, Pancreatic Cancer, Prostate Cancer	US$ 2 Billion
8	Vyndaqel	Pfizer	2019	Cardiomyopathy of Transthyretin-Mediated Amyloidosis	US$ 2 Billion
9	Darzalex	Janssen Biotech	2015	Multiple Myeloma	US$ 1.64 Billion
10	Soliris	Alexion Pharmaceuticals	2007	Paroxysmal Nocturnal Hemoglobinuria, Hemolytic Uremic Syndrome, Myasthenia Gravis	US$ 1 Billion
11	Venclexta	Abbvie	2016	Chronic Lymphocytic Leukemia, Acute Myeloid Leukemia	US$ 934 Million
12	Jakafi	Incyte Corporation	2011	Myelofibrosis	US$ 592 Million