AI Drug Discovery for Pharma and Biotech

Drug discovery

2

drugs

With orphan designations

Overview

Osteochondritis dissecans (OCD) is an acquired idiopathic lesion of subchondral bone, causing cartilage instability or detachment, most commonly in the knee (66.2% of cases), followed by elbow and ankle joints [1][4][14]. It primarily affects adolescents (10-20 years), with symptoms including activity-related pain, swelling, locking, and restricted motion [6][7][16]. Diagnosis combines radiographs and MRI to assess lesion stability [16][17]. Stable lesions in skeletally immature patients often heal with rest, while unstable or refractory cases require surgical intervention [1][5][13]. Untreated OCD increases osteoarthritis risk [2][11][16].

Population

  • Primarily adolescents aged 10-20 years, with male predominance (3:1 ratio); incidence rising in female athletes [4][9][14].

  • Bilateral involvement in 20-30% of cases; multifocal lesions in 11.2% [9][14].

  • Familial forms (10% of cases) linked to ACAN mutations causing cartilage defects [11][12].

Burden

  • Annual incidence: 15-30/100,000; knee OCD prevalence 2.3-31.6/100,000 [4][14][19].

  • 35% require surgery; 63% experience symptom recurrence post-microfracture at 10 years [13][18].

  • Economic impact: 6-month activity restriction reduces sports participation, while untreated cases accelerate osteoarthritis onset by 10-20 years [2][10][16].

Therapies

  • Conservative: Activity restriction (2-6 months), bracing, and physical therapy for stable lesions (50-67% success rate in juveniles) [1][13][18].

  • Surgical: Drilling (stable lesions), fixation with bioabsorbable screws (unstable lesions), or osteochondral grafts for detached fragments [5][13][16].

  • AAOS 2023 guidelines recommend early surgical consideration for salvageable unstable lesions in skeletally mature patients [5].

Categories: rare bone diseases, rare systemic and rheumatological diseases

Research Papers

1,558 drug discovery papers about Osteochondritis dissecans, with 2 first-in-class and 3 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

1,558 drug discovery papers about Osteochondritis dissecans, with 2 first-in-class and 3 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-08-15 | Arthroscopic Technique for Autologous Minced Cartilage Implantation for Focal Lesions of the Capitellum Humeri of the Elbow: A Technical Note.

Focal cartilage defects of the capitellum humeri are frequently underestimated, despite their substantial contribution to elbow pain and functional impairment in young and physically active patients. Arthroscopic autologous minced cartilage implantation is a single-stage biological cartilage repair technique that has shown favorable clinical and imaging outcomes in larger joints. Osteochondral lesions of the capitellum humeri of the elbow, particularly in the setting of osteochondritis dissecans, represent a challenging condition in young and physically active patients. However, detailed descriptions of the arthroscopic surgical technique and its application in the elbow joint remain limited. Therefore, the purpose of this Technical Note is to describe a step-by-step arthroscopic technique for autologous minced cartilage implantation in focal osteochondral lesions of the capitellum humeri. Indications, surgical pearls and pitfalls, and postoperative care are outlined, and the potential advantages of this technique in the treatment of elbow cartilage lesions are discussed.

Open article ↗



2026-07-01 | Autologous Chondrocyte Implantation in osteochondral defects of the capitulum: clinical and radiological outcome in a case series

Background Osteochondritis dissecans (OCD) of the capitellum is a cartilage and subchondral bone disorder predominantly affecting young, physically active individuals. While established surgical techniques such as microfracturing and osteochondral autograft transfer (OAT) offer acceptable outcomes, limitations in cartilage quality or procedural invasiveness remain. Autologous chondrocyte implantation (ACI) has been proposed as a less invasive, biologically restorative alternative, though clinical data on its use in the elbow are not available. Therefore, the aim of this pilot study was to evaluate the feasibility of ACI for the treatment of elbow OCD in a case series. Methods In this prospective observational study, six male patients (20.5 ± 5.2 years) with centralized ICRS grade IV OCD lesions of the capitellum were included and treated with ACI. Clinical evaluations were performed at 6 months (T1), 12 months (T2), and 34.8 ± 7.9 months (T3) postoperatively, including assessment of range of motion (ROM), subjective elbow value (SEV), visual analogue scale (VAS) for pain, Mayo Elbow Performance Score (MEPS), Kerlan-Jobe Orthopaedic Clinic (KJOC) score, and return to sport (RTS). MRI was conducted at T2 and T3, and cartilage repair tissue was analyzed using the MOCART 2.0 Ankle Score. Results Improvements were observed across all clinical outcome measures. SEV increased from 59 ± 18.5% preoperatively to 91.2 ± 4.2% at final follow-up, while VAS under load decreased from 6.5 ± 1.6 to 0.8 ± 0.7. MEPS and KJOC improved from 70.6 ± 17.7 to 97.5 ± 6 and from 38.3 ± 10 to 88.5 ± 8.4, respectively. MRI demonstrated defect filling and integration in all individuals, as assessed by the MOCART Score, which showed no deterioration between T2 and T3. Mean RTS time was 6.8 ± 3 months. No complications or need for revision surgery were recorded. Conclusion This pilot feasibility study provides preliminary insights into the treatment of centralized capitellar OCD lesions in a case series of young, active patients, with clinical and radiological findings at a mean 3-year follow-up (range, 25–42 months). These exploratory findings suggest that ACI may warrant further investigation for focal, symptomatic elbow defects with minimal or limited involvement of the subchondral bone. Larger controlled studies are needed to define the role of ACI relative to established treatment options.

Open article ↗



2026-05-30 | Return to Sport After Surgical Treatment of Capitellar Osteochondritis Dissecans Lesions in Baseball Players: A Systematic Review and Meta-analysis.

Because of the difficulty of baseball players with capitellar osteochondritis dissecans (OCD) lesions to return to sport (RTS), multiple operative strategies have been used for these lesions. However, there has yet to be a systematic review of RTS outcomes in specifically baseball players. The purpose of this study was to compare the RTS rates between surgical procedures for capitellar OCD lesions in youth baseball players, with a secondary purpose of comparing improvements in elbow range of motion (ROM) and improvements in Timmerman-Andrews scores between surgical procedures. The authors hypothesized that RTS rates would not differ based on the surgical procedure for capitellar OCD lesions in youth baseball players. Systematic review; Level of evidence, 4. The authors performed a literature search in February 2025 to identify studies evaluating postoperative outcomes for the treatment of capitellar OCD lesions in youth baseball players. Surgical procedures were grouped into debridement/loose-body removal ± microfracture, fixation procedures, bone peg grafting, and autologous osteochondral transplantation. Preoperative and postoperative elbow flexion and extension ROM, Timmerman-Andrews scores, and RTS rate and time were collected. Meta-analysis included pooled effect estimates for improvements in flexion ROM, extension ROM, and Timmerman-Andrews scores, as well as the proportion of RTS, applying a common effect model. Overall, 462 studies were screened and 16 studies were included. There were 389 total youth baseball players and 89% of lesions were unstable. RTS after autologous osteochondral transplantation (175/182; 96%) was more likely than after fixation (68/76; 89%), bone peg grafting (27/31; 87%), or debridement/loose-body removal ± microfracture (59/70; 84%) (P = .022). Overall, 31 of 60 (52%) pitchers were able to return to pitching. Fixation procedures were associated with greater improvements in elbow extension ROM (8° vs 3°; P = .013) than autologous osteochondral transplantation. Fixation procedures (score, 71) and autologous osteochondral transplantation (score, 58) also had greater improvements in Timmerman-Andrews scores than bone peg grafting (score, 26; P < .001); however, there was significant heterogeneity across studies and large differences in preoperative Timmerman-Andrews scores across groups. Overall, 92% of youth baseball players RTS after operative management of capitellar OCD lesions, with the highest RTS rates after autologous osteochondral transplantation; however, almost half of pitchers are unable to return to pitching. Clinicians can counsel injured youth baseball players that capitellar OCD lesions indicated for autologous osteochondral transplantation can have similar or better RTS outcomes than OCD lesions indicated for fixation or debridement ± microfracture.

Open article ↗



2026-05-15 | Osteochondritis Dissecans of the Capitellum in the Young Athlete.

Osteochondritis dissecans of the capitellum is an uncommon but notable cause of elbow pain in the young athlete. Although the exact pathogenesis of this condition is not completely understood, repetitive microtrauma, such as in throwing athletes or gymnasts, can predispose these groups to developing osteochondral lesions. Patients may present with laterally based elbow pain, swelling, or loss of range of motion. Initial screening radiographs can often detect these lesions, with MRI key to determining the exact lesion size, location, and stability. Treatment depends on multiple factors and may include a period of rest, particularly in stable lesions. Surgery is more often recommended in patients with unstable lesions or loose bodies or those who have persistent lesions despite a course of nonsurgical management. Historically, isolated osteochondritis dissecans removal and débridement yielded suboptimal results in the medium to long term, especially in larger and more laterally based lesions. Osteochondral autograft or allograft transplantation allows for more anatomic restoration of capitellar anatomy and has become the preferred treatment for larger, unstable defects with promising outcomes and return to activity.

Open article ↗



2026-05-06 | Osteochondral autograft transfer for capitellar osteochondritis dissecans in high-level adolescent athletes.

Osteochondritis dissecans (OCD) of the capitellum is a condition that predominantly affects high-performing adolescent athletes engaged in repetitive overhead motion and can lead to pain and mechanical symptoms that can interfere with the ability to perform in sports. Optimal surgical management of large OCD lesions remains inadequately studied. Prior studies have shown good short-term outcomes with the use of osteochondral autograft transfer (OAT). This study's primary objective is to evaluate the long-term outcomes of adolescent athletes undergoing OAT procedures for capitellar OCD. We retrospectively identified 28 patients with large (≥1 cm2) unstable capitellar OCD lesions who underwent OATs from the ipsilateral knee. The average age at the time of surgery was 14.95 years (range, 11-22 years). Time to return to play, post-operative and final level of play, Disabilities of the Arm, Shoulder and Hand (DASH) and Sport-Specific DASH questionnaires, pre-operative and post-operative elbow range of motion, post-operative elbow Single Assessment Numeric Evaluation score, and radiographic outcomes were collected and analyzed. Twenty patients were available for evaluation at an average of 62.4 months of follow-up (range 6 to 164.5 months). Chart review was conducted for the remaining patients. The average return to play timeline was 6.28 months (range, 3-13 months). The return to sport rate was 95%. Eighty percent of pitchers returned to pitching, all throwing by an average of 4 months. The average overall DASH score was 3.79 (95% confidence interval [CI]: 1.83-5.75), and the average Sport Specific DASH score was 7.11 (95% CI: 0.45-14.78). Elbow range of motion significantly improved, with improvement in flexion from a pre-operative average of 130.3° to 140.9° post-operatively (P < .001) and improvement in extension from a pre-operative average of 16.1° to 2.3° post-operatively (P < .001). Average post-operative elbow Single Assessment Numeric Evaluation scores collected from 12/20 patients were 85.8 (range, 60-100). Osseous integration of the graft site was complete in 24 of the patients by the 12-week post-operative visit. Treatment of large, unstable OCD lesions of the capitellum in adolescent athletes is safe, allows for a high rate of return to sport, and has good clinical outcomes with long-term follow-up.

Open article ↗



2026-08-15 | Arthroscopic Technique for Autologous Minced Cartilage Implantation for Focal Lesions of the Capitellum Humeri of the Elbow: A Technical Note.

Focal cartilage defects of the capitellum humeri are frequently underestimated, despite their substantial contribution to elbow pain and functional impairment in young and physically active patients. Arthroscopic autologous minced cartilage implantation is a single-stage biological cartilage repair technique that has shown favorable clinical and imaging outcomes in larger joints. Osteochondral lesions of the capitellum humeri of the elbow, particularly in the setting of osteochondritis dissecans, represent a challenging condition in young and physically active patients. However, detailed descriptions of the arthroscopic surgical technique and its application in the elbow joint remain limited. Therefore, the purpose of this Technical Note is to describe a step-by-step arthroscopic technique for autologous minced cartilage implantation in focal osteochondral lesions of the capitellum humeri. Indications, surgical pearls and pitfalls, and postoperative care are outlined, and the potential advantages of this technique in the treatment of elbow cartilage lesions are discussed.

Open article ↗



2026-07-01 | Autologous Chondrocyte Implantation in osteochondral defects of the capitulum: clinical and radiological outcome in a case series

Background Osteochondritis dissecans (OCD) of the capitellum is a cartilage and subchondral bone disorder predominantly affecting young, physically active individuals. While established surgical techniques such as microfracturing and osteochondral autograft transfer (OAT) offer acceptable outcomes, limitations in cartilage quality or procedural invasiveness remain. Autologous chondrocyte implantation (ACI) has been proposed as a less invasive, biologically restorative alternative, though clinical data on its use in the elbow are not available. Therefore, the aim of this pilot study was to evaluate the feasibility of ACI for the treatment of elbow OCD in a case series. Methods In this prospective observational study, six male patients (20.5 ± 5.2 years) with centralized ICRS grade IV OCD lesions of the capitellum were included and treated with ACI. Clinical evaluations were performed at 6 months (T1), 12 months (T2), and 34.8 ± 7.9 months (T3) postoperatively, including assessment of range of motion (ROM), subjective elbow value (SEV), visual analogue scale (VAS) for pain, Mayo Elbow Performance Score (MEPS), Kerlan-Jobe Orthopaedic Clinic (KJOC) score, and return to sport (RTS). MRI was conducted at T2 and T3, and cartilage repair tissue was analyzed using the MOCART 2.0 Ankle Score. Results Improvements were observed across all clinical outcome measures. SEV increased from 59 ± 18.5% preoperatively to 91.2 ± 4.2% at final follow-up, while VAS under load decreased from 6.5 ± 1.6 to 0.8 ± 0.7. MEPS and KJOC improved from 70.6 ± 17.7 to 97.5 ± 6 and from 38.3 ± 10 to 88.5 ± 8.4, respectively. MRI demonstrated defect filling and integration in all individuals, as assessed by the MOCART Score, which showed no deterioration between T2 and T3. Mean RTS time was 6.8 ± 3 months. No complications or need for revision surgery were recorded. Conclusion This pilot feasibility study provides preliminary insights into the treatment of centralized capitellar OCD lesions in a case series of young, active patients, with clinical and radiological findings at a mean 3-year follow-up (range, 25–42 months). These exploratory findings suggest that ACI may warrant further investigation for focal, symptomatic elbow defects with minimal or limited involvement of the subchondral bone. Larger controlled studies are needed to define the role of ACI relative to established treatment options.

Open article ↗



2026-05-30 | Return to Sport After Surgical Treatment of Capitellar Osteochondritis Dissecans Lesions in Baseball Players: A Systematic Review and Meta-analysis.

Because of the difficulty of baseball players with capitellar osteochondritis dissecans (OCD) lesions to return to sport (RTS), multiple operative strategies have been used for these lesions. However, there has yet to be a systematic review of RTS outcomes in specifically baseball players. The purpose of this study was to compare the RTS rates between surgical procedures for capitellar OCD lesions in youth baseball players, with a secondary purpose of comparing improvements in elbow range of motion (ROM) and improvements in Timmerman-Andrews scores between surgical procedures. The authors hypothesized that RTS rates would not differ based on the surgical procedure for capitellar OCD lesions in youth baseball players. Systematic review; Level of evidence, 4. The authors performed a literature search in February 2025 to identify studies evaluating postoperative outcomes for the treatment of capitellar OCD lesions in youth baseball players. Surgical procedures were grouped into debridement/loose-body removal ± microfracture, fixation procedures, bone peg grafting, and autologous osteochondral transplantation. Preoperative and postoperative elbow flexion and extension ROM, Timmerman-Andrews scores, and RTS rate and time were collected. Meta-analysis included pooled effect estimates for improvements in flexion ROM, extension ROM, and Timmerman-Andrews scores, as well as the proportion of RTS, applying a common effect model. Overall, 462 studies were screened and 16 studies were included. There were 389 total youth baseball players and 89% of lesions were unstable. RTS after autologous osteochondral transplantation (175/182; 96%) was more likely than after fixation (68/76; 89%), bone peg grafting (27/31; 87%), or debridement/loose-body removal ± microfracture (59/70; 84%) (P = .022). Overall, 31 of 60 (52%) pitchers were able to return to pitching. Fixation procedures were associated with greater improvements in elbow extension ROM (8° vs 3°; P = .013) than autologous osteochondral transplantation. Fixation procedures (score, 71) and autologous osteochondral transplantation (score, 58) also had greater improvements in Timmerman-Andrews scores than bone peg grafting (score, 26; P < .001); however, there was significant heterogeneity across studies and large differences in preoperative Timmerman-Andrews scores across groups. Overall, 92% of youth baseball players RTS after operative management of capitellar OCD lesions, with the highest RTS rates after autologous osteochondral transplantation; however, almost half of pitchers are unable to return to pitching. Clinicians can counsel injured youth baseball players that capitellar OCD lesions indicated for autologous osteochondral transplantation can have similar or better RTS outcomes than OCD lesions indicated for fixation or debridement ± microfracture.

Open article ↗



2026-05-15 | Osteochondritis Dissecans of the Capitellum in the Young Athlete.

Osteochondritis dissecans of the capitellum is an uncommon but notable cause of elbow pain in the young athlete. Although the exact pathogenesis of this condition is not completely understood, repetitive microtrauma, such as in throwing athletes or gymnasts, can predispose these groups to developing osteochondral lesions. Patients may present with laterally based elbow pain, swelling, or loss of range of motion. Initial screening radiographs can often detect these lesions, with MRI key to determining the exact lesion size, location, and stability. Treatment depends on multiple factors and may include a period of rest, particularly in stable lesions. Surgery is more often recommended in patients with unstable lesions or loose bodies or those who have persistent lesions despite a course of nonsurgical management. Historically, isolated osteochondritis dissecans removal and débridement yielded suboptimal results in the medium to long term, especially in larger and more laterally based lesions. Osteochondral autograft or allograft transplantation allows for more anatomic restoration of capitellar anatomy and has become the preferred treatment for larger, unstable defects with promising outcomes and return to activity.

Open article ↗



2026-05-06 | Osteochondral autograft transfer for capitellar osteochondritis dissecans in high-level adolescent athletes.

Osteochondritis dissecans (OCD) of the capitellum is a condition that predominantly affects high-performing adolescent athletes engaged in repetitive overhead motion and can lead to pain and mechanical symptoms that can interfere with the ability to perform in sports. Optimal surgical management of large OCD lesions remains inadequately studied. Prior studies have shown good short-term outcomes with the use of osteochondral autograft transfer (OAT). This study's primary objective is to evaluate the long-term outcomes of adolescent athletes undergoing OAT procedures for capitellar OCD. We retrospectively identified 28 patients with large (≥1 cm2) unstable capitellar OCD lesions who underwent OATs from the ipsilateral knee. The average age at the time of surgery was 14.95 years (range, 11-22 years). Time to return to play, post-operative and final level of play, Disabilities of the Arm, Shoulder and Hand (DASH) and Sport-Specific DASH questionnaires, pre-operative and post-operative elbow range of motion, post-operative elbow Single Assessment Numeric Evaluation score, and radiographic outcomes were collected and analyzed. Twenty patients were available for evaluation at an average of 62.4 months of follow-up (range 6 to 164.5 months). Chart review was conducted for the remaining patients. The average return to play timeline was 6.28 months (range, 3-13 months). The return to sport rate was 95%. Eighty percent of pitchers returned to pitching, all throwing by an average of 4 months. The average overall DASH score was 3.79 (95% confidence interval [CI]: 1.83-5.75), and the average Sport Specific DASH score was 7.11 (95% CI: 0.45-14.78). Elbow range of motion significantly improved, with improvement in flexion from a pre-operative average of 130.3° to 140.9° post-operatively (P < .001) and improvement in extension from a pre-operative average of 16.1° to 2.3° post-operatively (P < .001). Average post-operative elbow Single Assessment Numeric Evaluation scores collected from 12/20 patients were 85.8 (range, 60-100). Osseous integration of the graft site was complete in 24 of the patients by the 12-week post-operative visit. Treatment of large, unstable OCD lesions of the capitellum in adolescent athletes is safe, allows for a high rate of return to sport, and has good clinical outcomes with long-term follow-up.

Open article ↗



Access all drug discovery papers and probability of success in trials forecasts:

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Drug Discovery Landscape

2 orphan drug designations for Osteochondritis dissecans.

2 orphan drug designations for Osteochondritis dissecans.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

allogeneic human chondrocytes

cell therapies

FDA

2024-12-30

Vanarix SA

recombinant human platelet derived growth factor-BB

proteins

FDA

2010-08-06

Biomimetic Therapeutics, Inc.

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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.