FDA Office of Orphan Products Development

Closed portfolio with an early-phase archive tilt

According to ClinicalTrials.gov-derived indexes, FDA Office of Orphan Products Development sits in the top decile of US sponsors by registered trial count (#1,331 of 15,742 sponsors by registered trial count).

10 total trials none currently recruiting 10 completed early-phase tilt top decile by trial count

FDA Office of Orphan Products Development: 10 sponsored US clinical trials, none currently recruiting.

FDA Office of Orphan Products Development sponsors 10 registered US clinical trials on ClinicalTrials.gov, none currently recruiting, and 10 completed. 0 of these trials are in Phase 3-4 (later-stage) and 8 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Neurofibromatosis 2, with 1 trials.

Top-decile sponsor footprint

FDA Office of Orphan Products Development ranks in the top decile of sponsors by registered trial count (#1,331 of 15,742 sponsors by registered trial count). Volume at this scale usually reflects a long institutional pipeline, read phase tilt and therapeutic areas below before treating it as current-year activity.

Trial completion reliability score

A 90/100

10 of 10 decided trials (Completed vs. Terminated on ClinicalTrials.gov) reached their planned completion (100.0%), vs. national p10 68.4% / p90 100% among 1,234 sponsors with at least 10 decided trials. Still-open trials (Recruiting, Active-not-recruiting, Not-yet-recruiting) are excluded since they have not reached an outcome yet.

Indexed trials (enrollment closed)

10 total, page 1 of 1

COMPLETED Phase 2

A Controlled Study of Olanzapine in Children With Autism

NCT00057408
COMPLETED Phase 2

Testing of ADI-PEG in Hepatocellular Carcinoma

NCT00056992
COMPLETED Phase 2

Assessment of Chronic Guillain-Barre Syndrome Improvement With Use of 4-aminopyridine

NCT00056810
COMPLETED Phase 1

An Implant for Hearing Loss Due to Removal of Neurofibromatosis 2 Tumors

NCT00030043
COMPLETED Phase 1

Keratinocyte Growth Factor to Prevent Acute GVHD

NCT00031148
COMPLETED Phase 1

Etanercept for Non-Infectious Lung Injury Following Bone Marrow Transplantation

NCT00029328
COMPLETED Phase 1

Minocycline in Patients With Huntington's Disease

NCT00029874
COMPLETED NA

Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)

NCT00027391
COMPLETED Phase 1

ADI-PEG in Patients With Metastatic Melanoma

NCT00029900
COMPLETED NA

Randomized Study of Recombinant Human Growth Hormone in Patients on Chronic Hemodialysis or Peritoneal Dialysis

NCT00004429

Phase Distribution

PhaseTrial count
Phase 1 5
Phase 2 3

Therapeutic Areas

Neurofibromatosis 2 1

How the FDA Office of Orphan Products Development early-phase archive is shaped

10 indexed studies, none listed as recruiting (0% open); 10 completed (100%).

Phase mix: 0 late (III/IV) vs 8 early (I/II) (early-phase heavy).

Top condition: Neurofibromatosis 2 (1 trial), 0 others listed.

How to read a closed sponsor archive

When recruiting_count is zero, the indexed set is completed or otherwise not open. Phase tilt still describes where archived studies sat on the investigational pathway. Re-check ClinicalTrials.gov before acting.

FDA Office of Orphan Products Development: archived-pipeline peers nationwide

Peers matched on 10 indexed studies with no current recruiting: volume and archive shape, not condition overlap.

FDA Office of Orphan Products Development sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together. Source: ClinicalTrials.gov (2026-08-08 snapshot). 2×2 strategic matrix plotting 9 entities by Total trials (X) and Recruiting share (Y), with a crosshair dividing the plot into four quadrants. Higher volume & more openFocused & more openHigher volume, mostly closedSmaller & mostly closed 051015 0%10%20%30%40% Total trials Recruiting share FDA Office of Orphan Products DevelopmentAdamas PharmaceuticalsAdverum BiotechnologiesAevi Genomic Medicine, LLC, a Cerecor companyAgouron Pharmaceuticals10xBio22nd Century Group23andMe3-C Institute for Social Development
FDA Office of Orphan Products Development sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together. Source: ClinicalTrials.gov (2026-08-08 snapshot).

FDA Office of Orphan Products Development sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together.

Sponsors near 10 archived trials

Sponsors with similar archive density (± trial count)

Related

Read our methodology - how this data is sourced, computed, and verified.

What to do with this FDA Office of Orphan Products Development page

10 registered trials is a research-attention snapshot, not an endorsement of FDA Office of Orphan Products Development or any single study.

  • None of FDA Office of Orphan Products Development's trials are currently recruiting in this registry; that can change as new trials post, or check the broader research index. Browse research indexes
  • Neurofibromatosis 2 is the condition FDA Office of Orphan Products Development studies most, compare against other sponsors active in the same condition before evaluating a specific study. See Neurofibromatosis 2 trials
  • Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

FDA Office of Orphan Products Development pipeline card: 10 registered trials from official public datasets.Report a data error.