VivoSeq
Rare Disease

Familial Mediterranean Fever

9

Pipeline Programs↗

5

Active Trials↗

8

Companies Involved↗

Familial Mediterranean Fever is an early-stage landscape with 9 programs across 8 companies. The most active players are Mutual Pharmaceutical Company, Inc., Aristea Therapeutics, Inc., Kiniksa Pharmaceuticals (UK), Ltd.. 2 therapies are approved with 1 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

38/100

Pipeline Velocity

6/100

Mech. Concentration

40/100

Completion Rate

78/100

Attrition Rate

14/100

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.

M

Mutual Pharmaceutical Company, Inc.

1 program

Phase 1A

Aristea Therapeutics, Inc.

1 program

Phase 2K

Kiniksa Pharmaceuticals (UK), Ltd.

1 program

Phase 2Roche logo

Roche

1 program

Phase 2Amgen logo

Amgen

1 program

Phase 3M

Milestone Pharmaceuticals Inc.

1 program

Approved

Pipeline

Programs by Phase

Analysis

Modality Breakdown

70%

Small molecule

7 of 10 classified programs — the dominant modality here.

30%ALL OTHERSMonoclonal antibody220%Protein / enzyme110%

Small molecule leads; the ring shows all 10 classified programs, each share of the total. Modality classified for 100% of 11 pipeline programs.

Analysis

Trial Status

14

clinical trials

Completed7 · 50%
Recruiting4 · 29%
Withdrawn2 · 14%
Active, not recruiting1 · 7%

Clinical Trials

Trial Activity

14 trials

Observing the Efficacy and Safety of Different Drugs Used in Real-world Familial Mediterranean Fever (FMF) Cases
Recruiting·TTongji Hospital·N/A·NCT07077473·12 enrolled
Safety and Efficacy of RPH-104 Used to Prevent Recurrent Fever Attacks in Adult Patients With Colchicine Resistant or Colchicine Intolerant Familial Mediterranean Fever
Recruiting·—·Phase 2·NCT05190991·60 enrolled
Randomized Controlled Trial in Patients on Long-term Colchicine With Colchicine-resistant Familial Mediterranean Fever (FMF) to Evaluate the Efficacy of On-demand Anakinra Treatment for Painful Attacks in Patients Who Refuse Continuous Daily Therapy
Recruiting·AAssistance Publique - Hôpitaux de Paris·Phase 3·NCT06336733·50 enrolled
An Exploratory Study of the Genetics, Pathophysiology, and Natural History of Autoinflammatory Diseases
Recruiting·NNational Human Genome Research Institute (NHGRI)·N/A·NCT00001373·5,000 enrolled
Efficacy and Safety of RPH-104 for Resolution and Prevention of Recurring Attacks in Adult Subjects With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine
Active, not recruiting·—·Phase 2·NCT05092776·84 enrolled
Kineret (Anakinra), in Adult Patients With Colchicine-Resistant Familial Mediterranean Fever
Completed·SSheba Medical Center·Phase 3·NCT01705756·25 enrolled
Study of Colchicine Resistance in Familial Mediterranean Fever
Completed·AAssistance Publique - Hôpitaux de Paris·N/A·NCT05418686·30 enrolled
Rilonacept for Treatment of Familial Mediterranean Fever (FMF)
Completed·TThe Cleveland Clinic·Phase 2·NCT00582907·14 enrolled
Pharmacokinetics Study of Colchicine in Familial Mediterranean Fever (FMF) Patients
Completed·MMutual Pharmaceutical Company, Inc.·Phase 1·NCT01075906·75 enrolled
Tocilizumab for the Treatment of Familial Mediterranean Fever
Completed·UUniversity Hospital Tuebingen·Phase 2·NCT03446209·30 enrolled
Efficacy and Safety of Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever
Completed·Novartis logoNovartis·Phase 2·NCT01088880·10 enrolled
Evaluate the Safety and Efficacy of Canakinumab in Pediatric Patients With Colchicine Intolerant or Colchicine Resistant Familial Mediterranean Fever (FMF)
Completed·Novartis logoNovartis·Phase 2·NCT01148797·15 enrolled
A Study to Evaluate RIST4721 in Familial Mediterranean Fever (FMF)
Withdrawn·AAristea Therapeutics, Inc.·Phase 2·NCT05448391
A Prospective, Open-label, Single Arm, Multicenter, Post-authorization Efficacy and Safety Study of Subcutaneous Anakinra in Chinese Patients With Colchicine-resistant Familial Mediterranean Fever (FMF)
Withdrawn·SSwedish Orphan Biovitrum·Approved·NCT06666335

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