AI Drug Discovery for Pharma and Biotech

Drug discovery

2

drugs

With orphan designations

Overview

Legg-Calvé-Perthes disease is a juvenile osteonecrosis of the femoral head caused by transient vascular disruption, leading to bone collapse and remodeling. It typically affects children aged 4–12 years (peak 5–7), with a 4:1 male predominance. Bilateral involvement occurs in 10–24% of cases. Prognosis depends on age of onset, femoral head containment, and preservation of hip mobility. Long-term sequelae include early osteoarthritis in 50% of patients with significant femoral head deformity [1][5][10].

Population

  • Incidence: 0.4–29.0/100,000 children <15 years globally, with racial disparities (highest in Caucasians: 5.69/100,000) [2][6][14]

  • Sex/age: 80% male, peak diagnosis at 5–7 years [1][10]

  • Risk factors: Extreme obesity (OR=3.41), coagulopathies (50% of cases), and latitude-related environmental factors [2][5][7]

Burden

  • 50% require total hip replacement by age 50–60 if femoral head deformity persists [10][11]

  • Early osteoarthritis risk correlates with residual asphericity (HR=12.4 for severe deformity) [7][16]

  • Socioeconomic impact: Higher hospitalization rates in deprived populations (incidence up to 33.6/100,000) [6][8]

Therapies

  • Non-surgical: NSAIDs, activity restriction (crutches/wheelchair), bracing (Petrie cast), and physical therapy for hip mobility <6 years [1][3][14]

  • Surgical: Femoral/acetabular osteotomies for containment in older children (>8 years) or lateral pillar B/C involvement [1][11][14]

  • Prognostic tools: Herring classification guides intervention timing; <6 years generally favorable outcomes [12][14]

Categories: rare bone diseases, rare genetic diseases, rare systemic and rheumatological diseases

Research Papers

907 drug discovery papers about Legg-Calvé-Perthes disease, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

907 drug discovery papers about Legg-Calvé-Perthes disease, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-06-12 | [Extracorporeal magnetotransduction therapy in juvenile Perthes' disease : A case report].

Morbus Perthes is an aseptical osteonecrosis in childhood that has great variablity in theextent, course and healing results. A spontaneous resolution is possible, which, however, mandates high patience for both the affected child and the caring parents. Regarding the lack of conservative treatment methods, a new non-invasive therapy approach using novel extracorporeal magnetotransduction therapy (EMTT) was performed on a 4-year-old patient with Perthes disease. A significant pain reduction was achieved within 6 weeks in line with MRI improvements with a total of 9 EMTT sessions. EMTT represents a promising non-invasive treatment option for children with Perthes disease, and future controlled studies in this special patient cohort are pending and warranted.

Open article ↗



2026-06-01 | The Role of Hip Arthrodiastasis in Management of Late-Onset Legg-Calvé-Perthes Disease: A Prospective Case Series.

Late-onset Legg-Calvé-Perthes disease (LCPD), defined as onset after the age of 8 years or younger, is associated with a poor prognosis and limited remodeling potential. The role of hip arthrodiastasis in this age group remains controversial. This prospective case series enrolled patients consecutively and collected data at predefined follow-up intervals. It included 24 patients with late-onset LCPD treated with articulated hip arthrodiastasis using a hinged Ilizarov external fixator combined with percutaneous adductor tenotomy. This reflects a deliberate institutional shift in treatment strategy rather than selective case allocation, which may reduce selection bias but limits the availability of a contemporary control group. All patients were older than 8 years at disease onset and classified as Herring lateral pillar B or C. Clinical outcomes were assessed using the Harris Hip Score (HHS) and range of motion (ROM). Radiographic evaluation included the Sharp acetabular angle, Reimer uncoverage index, epiphyseal index, and modified Stulberg classification at final follow-up. The mean follow-up was 24 months (range: 18 to 30). The mean HHS improved significantly from 47.2 preoperatively to 86.7 at final follow-up (P<0.001). Significant improvements were observed in hip flexion, abduction, and internal rotation (all P<0.001). Radiographically, mean femoral head uncoverage improved from 33.3% to 25.0% (P<0.001), and the Sharp acetabular angle decreased from 42.3 to 37.6 degrees (P<0.001). According to the modified Stulberg classification, 19 hips (79%) had good outcomes (Stulberg II to III), while 5 hips (21%) had poor outcomes (Stulberg IV to V). Complications included pin-tract infections managed with local care and oral antibiotics, as well as a small number of patients requiring additional secondary procedures. Articulated hip arthrodiastasis with adductor tenotomy resulted in favorable short-term clinical and radiographic outcomes in most patients with late-onset LCPD. This technique improved hip motion and radiographic parameters suggestive of improved femoral head containment, without the need for femoral or pelvic osteotomy, and may represent a useful treatment option in this challenging patient population. Level IV-therapeutic case series.

Open article ↗



2026-05-14 | TOTAL HIP ARTHROPLASTY IN LEGG-CALVÉ-PERTHES DISEASE: A STUDY FROM THE AUSTRALIAN ORTHOPAEDIC ASSOCIATION NATIONAL JOINT REPLACEMENT REGISTRY

Background Legg-Calve-Perthes disease (LCPD) is a paediatric hip disorder characterised by idiopathic ischaemic necrosis and early osteoarthritis. There is limited evidence on revision outcomes for total hip arthroplasty (THA) in LCPD compared to primary osteoarthritis (OA). Methods Data from the Australian Orthopaedic Association National Joint Replacement Registry (AOANJRR) was analysed to determine cumulative percent revision rate (CPR), revision indication and functional outcome scores for THA performed for patients undergoing THA for Perthes disease and primary OA between 2003 and 2023. Results There were 1159 THAs for LCPD and 521,603 for primary OA available for analysis. Patients with LCPD incurred a higher CPR in the first two weeks postoperatively, but no difference was observed beyond this time. The CPR adjusted for age, head size, sex and femoral fixation for patients with LCPD at 18 years was 8.5% (95% CI 5.6,12.8) compared to 7.1% (95% CI 7.0, 7.3) for patients with primary OA. There was no difference in the reason for revision. Functional outcome scores were similar, although those patients with LCPD had slightly lower improvement in the Oxford Hip Score. Conclusion Revision rates following THA are comparable between patients with LCPD and primary OA at eighteen years post-operatively. The higher early revision rate in LCPD patients warrants further investigation into contributing factors including surgical planning, types of prostheses, and the influence of previous surgery.

Open article ↗



2026-06-12 | [Extracorporeal magnetotransduction therapy in juvenile Perthes' disease : A case report].

Morbus Perthes is an aseptical osteonecrosis in childhood that has great variablity in theextent, course and healing results. A spontaneous resolution is possible, which, however, mandates high patience for both the affected child and the caring parents. Regarding the lack of conservative treatment methods, a new non-invasive therapy approach using novel extracorporeal magnetotransduction therapy (EMTT) was performed on a 4-year-old patient with Perthes disease. A significant pain reduction was achieved within 6 weeks in line with MRI improvements with a total of 9 EMTT sessions. EMTT represents a promising non-invasive treatment option for children with Perthes disease, and future controlled studies in this special patient cohort are pending and warranted.

Open article ↗



2026-06-01 | The Role of Hip Arthrodiastasis in Management of Late-Onset Legg-Calvé-Perthes Disease: A Prospective Case Series.

Late-onset Legg-Calvé-Perthes disease (LCPD), defined as onset after the age of 8 years or younger, is associated with a poor prognosis and limited remodeling potential. The role of hip arthrodiastasis in this age group remains controversial. This prospective case series enrolled patients consecutively and collected data at predefined follow-up intervals. It included 24 patients with late-onset LCPD treated with articulated hip arthrodiastasis using a hinged Ilizarov external fixator combined with percutaneous adductor tenotomy. This reflects a deliberate institutional shift in treatment strategy rather than selective case allocation, which may reduce selection bias but limits the availability of a contemporary control group. All patients were older than 8 years at disease onset and classified as Herring lateral pillar B or C. Clinical outcomes were assessed using the Harris Hip Score (HHS) and range of motion (ROM). Radiographic evaluation included the Sharp acetabular angle, Reimer uncoverage index, epiphyseal index, and modified Stulberg classification at final follow-up. The mean follow-up was 24 months (range: 18 to 30). The mean HHS improved significantly from 47.2 preoperatively to 86.7 at final follow-up (P<0.001). Significant improvements were observed in hip flexion, abduction, and internal rotation (all P<0.001). Radiographically, mean femoral head uncoverage improved from 33.3% to 25.0% (P<0.001), and the Sharp acetabular angle decreased from 42.3 to 37.6 degrees (P<0.001). According to the modified Stulberg classification, 19 hips (79%) had good outcomes (Stulberg II to III), while 5 hips (21%) had poor outcomes (Stulberg IV to V). Complications included pin-tract infections managed with local care and oral antibiotics, as well as a small number of patients requiring additional secondary procedures. Articulated hip arthrodiastasis with adductor tenotomy resulted in favorable short-term clinical and radiographic outcomes in most patients with late-onset LCPD. This technique improved hip motion and radiographic parameters suggestive of improved femoral head containment, without the need for femoral or pelvic osteotomy, and may represent a useful treatment option in this challenging patient population. Level IV-therapeutic case series.

Open article ↗



2026-05-14 | TOTAL HIP ARTHROPLASTY IN LEGG-CALVÉ-PERTHES DISEASE: A STUDY FROM THE AUSTRALIAN ORTHOPAEDIC ASSOCIATION NATIONAL JOINT REPLACEMENT REGISTRY

Background Legg-Calve-Perthes disease (LCPD) is a paediatric hip disorder characterised by idiopathic ischaemic necrosis and early osteoarthritis. There is limited evidence on revision outcomes for total hip arthroplasty (THA) in LCPD compared to primary osteoarthritis (OA). Methods Data from the Australian Orthopaedic Association National Joint Replacement Registry (AOANJRR) was analysed to determine cumulative percent revision rate (CPR), revision indication and functional outcome scores for THA performed for patients undergoing THA for Perthes disease and primary OA between 2003 and 2023. Results There were 1159 THAs for LCPD and 521,603 for primary OA available for analysis. Patients with LCPD incurred a higher CPR in the first two weeks postoperatively, but no difference was observed beyond this time. The CPR adjusted for age, head size, sex and femoral fixation for patients with LCPD at 18 years was 8.5% (95% CI 5.6,12.8) compared to 7.1% (95% CI 7.0, 7.3) for patients with primary OA. There was no difference in the reason for revision. Functional outcome scores were similar, although those patients with LCPD had slightly lower improvement in the Oxford Hip Score. Conclusion Revision rates following THA are comparable between patients with LCPD and primary OA at eighteen years post-operatively. The higher early revision rate in LCPD patients warrants further investigation into contributing factors including surgical planning, types of prostheses, and the influence of previous surgery.

Open article ↗



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Drug Discovery Landscape

2 orphan drug designations for Legg-Calvé-Perthes disease.

2 orphan drug designations for Legg-Calvé-Perthes disease.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

clodronate + simvastatin

small molecules

FDA

2024-12-23

VSI Composites, Inc.

N-[(2S)-1-[(3aS,6R,6aR)-6-Ethynyl-3-oxohexahydro-2H-furo[3,2-b]pyrrol-4-yl]-4-methyl-1-oxopentan-2-yl]-4-[5-fluoro-2-(4-methylpiperazin-1-yl)-1,3-thiazol-4-yl]benzamide hydrochloride

small molecules

FDA

2024-04-15

OsteoCat Therapeutics AB

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New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.