VivoSeq
Other

Noonan Syndrome

5

Pipeline Programs↗

3

Active Trials↗

4

Companies Involved↗

Noonan syndrome is an early-stage landscape with 5 programs across 4 companies. The most active players are BioMarin Pharmaceutical, Takeda, Novo Nordisk. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

30/100

Pipeline Velocity

0/100

Mech. Concentration

50/100

Completion Rate

80/100

Attrition Rate

13/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

BioMarin Pharmaceutical logo

BioMarin Pharmaceutical

1 program

Phase 2Takeda logo

Takeda

1 program

Phase 2Novo Nordisk logo

Novo Nordisk

1 program

PreclinicalEMD Serono logo

EMD Serono

1 program

Preclinical

Pipeline

Programs by Phase

Analysis

Modality Breakdown

80%

Small molecule

4 of 5 classified programs — the dominant modality here.

20%ALL OTHERSPeptide120%

Small molecule leads; the ring shows all 5 classified programs, each share of the total. Modality classified for 100% of 6 pipeline programs.

Analysis

Trial Status

8

clinical trials

Completed4 · 50%
Recruiting2 · 25%
Terminated1 · 13%
Enrolling by invitation1 · 13%

Clinical Trials

Trial Activity

8 trials

Constitution of a Biological Collection to Study the Pathophysiology in Noonan Syndrome
Recruiting·UUniversity Hospital, Toulouse·N/A·NCT05202210·100 enrolled
A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 2·NCT06668805·54 enrolled
French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
Enrolling by invitation·Novo Nordisk logoNovo Nordisk·N/A·NCT05308927·221 enrolled
Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
Completed·Novo Nordisk logoNovo Nordisk·N/A·NCT03435627·71 enrolled
Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan Syndrome
Completed·UUniversity Hospital, Toulouse·Phase 3·NCT02713945·53 enrolled
Somatropin Effect on Linear Growth and Final Height in Subjects With Noonan Syndrome
Completed·Novo Nordisk logoNovo Nordisk·Phase 3·NCT01529840·24 enrolled
Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome
Completed·Novo Nordisk logoNovo Nordisk·Phase 3·NCT01927861·51 enrolled
Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome
Terminated·IInsmed Incorporated·Phase 2·NCT00351221·24 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)