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Table 1 Characteristics of Funded Grants in FY 2007—2011 that Led to FDA Approvals, (N = 9)

From: FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development

Year Approved
(First Funded)
Generic (Trade Name) Disease (Therapeutic Area) Study Phase at Funding PI Institution Single or Multiple Study Sites Non-Academic Collaboration at Application
2012 (2007) Ivacaftor (Kalydeco) Cystic Fibrosis Subjects with G551D (Pulmonary) Phase 2 Industry Multiple- 8 sites Patient group
2013 (2008) Topical nitrogen mustard, Meclorethamine (Valchlor) Mycosis Fungoides (Oncology/Hematology) Phase 2 Academic Multiple- 2 sites Industry
2015 (2011) Asfotase alfa (Strensiq) Hypophosphatasia (Endocrinology) Phase 2 Industry Multiple- 7 sites None
2015 (2008) Parthyroid Hormone (Natpara) Hypoparathyroidism (Endocrinology) Phase 3 Academic Single Industry
2015 (2007) Sirolimus (Rapamune) Lymphangioleiomyomatosis (Pulmonary) Phase 3 Academic Multiple- 8 sites Patient group & Industry
2016 (2011) Cheatham Platinum Stent System Aortic Wall Injury Associated with Aortic Coarctation (Cardiovascular) Phase 3 Academic Multiple- 19 sites Industry
2017 (2009) Berlin Heart EXCOR Pediatric Ventricular Assist Device Bridge-to-Heart Transplantation in Children (Cardiovascular) Phase 2 Academic Multiple- 13 sites None
2018 (2008) Fish Oil Triglycerides (Omegaven) Reversal of Parenteral Nutrition-Associated Cholestasis (Gastrointestinal) Phase 2 Academic Single None
2019 (2008) Tafamidis meglumine/ /Tafamidis free acid (Vyndaqel/Vyndamax) Familial Amyloid Polyneuropathy (Neurology) Phase 3 Industry Multiple- 8 sites None