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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