FDA Office of Orphan Products Development
72
3
5
58
Key Highlights
Risk & Performance
Pipeline Risk Assessment
Pipeline Risk Assessment
Based on historical performance
Moderate Risk
Score: 35/100
9.7%
7 terminated/withdrawn out of 72 trials
89.2%
+2.7% vs industry average
17%
12 trials in Phase 3/4
40%
23 of 58 completed trials have results
Key Signals
Enrollment Performance
Analytics
Activity Timeline
Global Presence
Clinical Trials (72)
SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
Role: collaborator
Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System
Role: collaborator
Safety of MT-401-OTS in Patients With Relapsed AML or MDS
Role: collaborator
Abatacept as GVHD Prophylaxis Phase 2
Role: collaborator
Interferon-γ (IFN-γ) With Donor Leukocyte Infusion to Treat Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes Post Allogeneic Hematopoietic Stem Cell Transplantation
Role: collaborator
Implanted Myoelectric Control for Restoration of Hand Function in Spinal Cord Injury
Role: collaborator
Daily Vitamin D for Sickle-cell Respiratory Complications
Role: collaborator
Cholic Acid for Hepatic Steatosis in Lipodystrophy
Role: collaborator
Safety and PK of Nikkomycin Z for Coccidioides Pneumonia Treatment
Role: collaborator
Comparison of Two Medications Aimed at Slowing Aortic Root Enlargement in Individuals With Marfan Syndrome
Role: collaborator
Efficacy and Safety of Sirolimus in LAM
Role: collaborator
Inhaled Nitric Oxide for Pediatric Painful Sickle Crisis
Role: collaborator
Phase I Study of Vasoactive Intestinal Peptide in Patients With Acute Respiratory Distress Syndrome and Sepsis
Role: collaborator
KPT-330 Plus RICE for Relapsed/Refractory Aggressive B-Cell Lymphoma
Role: collaborator
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Role: collaborator
L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
Role: collaborator
ABC294640 in Treating Patients With Advanced Solid Tumors
Role: collaborator
Alendronate in Juvenile Osteoporosis
Role: collaborator
A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)
Role: collaborator
Clinical Trial on Treatment of Intraventricular Hemorrhage
Role: collaborator