RARE DISEASE
Chronic graft versus host disease
Chronic graft versus host disease
Chronic graft versus host disease
Drug discovery
16
drugs
With orphan designations
Overview
Chronic graft-versus-host disease (cGVHD) is a systemic immune-mediated disorder occurring in 30–70% of allogeneic hematopoietic stem cell transplant (HSCT) recipients, typically emerging ≥100 days post-transplant. It manifests as autoimmune-like inflammation and fibrosis across multiple organs (e.g., skin, eyes, liver, lungs), driven by donor T-cells attacking host tissues. cGVHD is a leading cause of late non-relapse mortality and long-term morbidity due to organ dysfunction, infections, and immunosuppressive therapy toxicity [1][3][5][10].
Burden
Morbidity: 26–40% of patients with active cGVHD cannot work; high rates of pain, depression, and physical disability [2][9][14].
Mortality: Leading cause of non-relapse mortality (8–38%), driven by infections and organ failure [3][10][17].
Economic: Prolonged immunosuppression increases healthcare costs (e.g., $21,000 annual outpatient visits) and caregiver dependency [14][17][18].
Therapies
First-line: Systemic corticosteroids ± calcineurin inhibitors (tacrolimus, cyclosporine) [3][8][13].
Steroid-refractory options: JAK inhibitors (ruxolitinib), BTK inhibitors (ibrutinib), and ROCK2 inhibitors (belumosudil) [3][8][15].
Prophylaxis: Post-transplant cyclophosphamide, T-cell depletion (ATG, CD34+ selection), and graft engineering (naive T-cell depletion) [3][8][11].
Categories: rare immunological diseases, rare transplant-related disorders
Research Papers
2,397 drug discovery papers about Chronic graft versus host disease, with 1 first-in-class and 16 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2,397 drug discovery papers about Chronic graft versus host disease, with 1 first-in-class and 16 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-14 | Ruxolitinib-Associated Improvement in Post-transplant Air-Leak Syndrome Complicating Steroid-Dependent Organizing Pneumonia: A Case Report.
Air-leak syndrome (ALS), including pneumothorax, pneumomediastinum, and subcutaneous emphysema, is a rare but life-threatening complication after allogeneic hematopoietic stem cell transplantation (HSCT). However, no standard treatment has yet been established. Here, we report the case of a 60-year-old man with acute myeloid leukemia who underwent allogeneic HSCT. He developed chronic graft-versus-host disease (GVHD) and cryptogenic organizing pneumonia (COP) on day 126 after transplantation. Although initial corticosteroid therapy improved the COP, the relapse occurred during tapering, and repeated steroid treatment failed to achieve sustained disease control. The patient subsequently developed pneumomediastinum, consistent with ALS. Ruxolitinib treatment was initiated on day 265 for steroid-dependent chronic GVHD and organizing pneumonia. Following treatment, oxygenation improved, pneumomediastinum improved, and corticosteroids were successfully tapered and discontinued without COP recurrence. Serum Krebs von den Lungen-6 (KL-6) levels decreased in parallel with clinical and radiological improvement. The introduction of ruxolitinib is associated with improvements in ALS and enabled the discontinuation of steroids. Adverse events included cytopenia and mild liver dysfunction, consistent with known safety profiles. The patient died of acute respiratory failure after transfusion, which was not considered a direct complication of the ruxolitinib treatment. This case suggests that ruxolitinib may facilitate corticosteroid tapering and radiographic improvement of organizing pneumonia-associated ALS after allogeneic transplantation.
2026-08-11 | Phenotype-Specific Associations Between Graft‑Versus‑Host Disease and Post‑Transplant Outcomes.
Disease relapse remains the leading cause of failure following allogeneic hematopoietic cell transplantation (HCT). As novel prophylactic strategies increasingly aim to universally eliminate all forms of graft‑versus‑host disease (GVHD), how these approaches may inadvertently sacrifice graft‑versus‑leukemia (GVL) activity in the post‑transplant cyclophosphamide (PTCy) era is unclear. To evaluate the associations between time‑updated GVHD phenotypes and clinical outcomes, including relapse, non‑relapse mortality (NRM), and overall survival (OS), in patients undergoing PTCy‑based haploidentical or mismatched unrelated donor (MMUD) HCT. This was a retrospective cohort study using the Center for International Blood and Marrow Transplant Research registry. Participants included 7,055 patients with hematologic malignancies who received a first haploidentical or MMUD HCT with PTCy from 2013-2021. Associations with relapse, NRM and OS were evaluated using multi‑state time‑dependent Cox proportional hazards models and a multi‑state random survival forest (MS‑RSF). Time‑updated acute and chronic GVHD phenotypes included isolated grade II acute GVHD (aGVHD), grade III-IV aGVHD, mild chronic GVHD (cGVHD), and immunosuppressive therapy-requiring (IST‑requiring) cGVHD. IST‑requiring cGVHD without antecedent aGVHD was associated with a lower modeled relapse hazard compared with remaining GVHD‑free (Hazard Ratio [HR], 0.74; 95% confidence interval [CI], 0.62-0.89; False Discovery Rate (FDR)-adjusted p (q)=0.006) and lower overall mortality (HR 0.62; 95% CI, 0.53-0.73; q < 0.001). In contrast, grade III-IV aGVHD was associated with significantly higher modeled NRM (HR, 3.15; 95% CI, 2.57-3.84; q < 0.001). Mild cGVHD and isolated grade II aGVHD showed intermediate patterns without consistent associations. These associations were directionally consistent across multiple analytic approaches, including standard time‑dependent Cox regression, dynamic and fixed landmark analyses, and MS‑RSF. In this large PTCy‑treated mismatched donor cohort, IST‑requiring cGVHD was the GVHD phenotype most consistently associated with lower relapse incidence, whereas severe aGVHD was associated with higher NRM. These findings highlight heterogeneity in GVHD phenotypes and suggest that strategies distinguishing toxic acute GVHD from chronic alloreactivity patterns may better balance morbidity and long‑term disease control. Given that relapse remains the predominant cause of post‑transplant mortality, approaches aiming to universally eliminate all GVHD warrant careful reconsideration.
2026-08-11 | Comorbid conditions after allogeneic haematopoietic stem cell transplantation in young adults in Germany-A prospective, multicentre trial.
Young adults (YA) are particularly affected by comorbid conditions following allogeneic haematopoietic stem cell transplantation (alloHSCT), as they have to live with them for many years. This is the first multicentre prospective study investigating sequelae after alloHSCT in YA in Germany. In total, 107 patients who survived relapse-free until day 100 after alloHSCT were enrolled. Median patient age was 29.3 years. Chronic graft-versus-host disease (cGvHD) occurred in 40.2%. Comorbidities were captured at days 100, 180, 365 and 910 by applying the post-transplant multimorbidity index (PTMI). Considering all time points, 12.5%-23.5% of patients had no comorbidity, while 39.2%-49.5% had 1-3, 26.3%-33.0% had 4-6 and 5.7%-8.4% had >6 PTMI-defined comorbidities respectively. The most prevalent PTMI-based comorbidities were iron overload, hypogonadism, arterial hypertension, infections, hepatic dysfunction and osteopenia/osteoporosis. However, the spectrum of PTMI-recorded comorbidities and those concurrently self-reported by patients differed substantially. Factors linked to a higher PTMI were total body irradiation and active cGvHD. Investigating single PTMI-defined comorbidities, we found significant associations of osteopenia/osteoporosis with cGvHD, ongoing immunosuppression and hypogonadism, which was more common among female survivors. These results provide a detailed analysis of the occurrence of a vast spectrum of PTMI-defined comorbidities in YA after alloHSCT.
2026-08-10 | Reduced Cumulative Post-Transplant Cyclophosphamide Exposure Is Associated with Increased Acute and Chronic Graft-versus-Host Disease After Allogeneic Hematopoietic Cell Transplantation.
Post-transplant cyclophosphamide (PTCy) is conventionally administered at a cumulative dose of 100 mg/kg for graft-versus-host disease (GVHD) prophylaxis after allogeneic hematopoietic cell transplantation (allo-HCT). In clinical practice, cumulative PTCy exposure may be reduced because of institutional protocols, physician-directed dose modifications, or body weight-adjusted dosing strategies. However, the clinical consequences of receiving cumulative PTCy doses below the standard 100 mg/kg remain poorly defined. We retrospectively evaluated 251 consecutive allo-HCT recipients transplanted between 2015 and 2025. Among them, 180 patients receiving PTCy-based GVHD prophylaxis were stratified according to cumulative PTCy exposure (<100 vs. ≥100 mg/kg), irrespective of the reason for dose reduction. Acute GVHD, chronic GVHD, relapse, non-relapse mortality (NRM), and overall survival (OS) were analyzed using logistic regression, Cox proportional hazards models, and competing-risk methods. Among the 180 PTCy recipients, 71 received <100 mg/kg and 109 received ≥100 mg/kg; the median cumulative dose in the reduced-exposure group was approximately 80 mg/kg. Reduced cumulative PTCy exposure (<100 mg/kg) was associated with a significantly increased incidence of acute GVHD compared with standard exposure (57.7% vs. 37.0%; OR 2.31, 95% CI 1.21-4.49; p=0.009). Patients receiving <100 mg/kg also experienced a higher burden of clinically significant GVHD, including grade II-IV acute GVHD (25.4% vs. 12.8%; OR 2.30, 95% CI 1.07-5.08; p=0.035), moderate-to-severe chronic GVHD (21.1% vs. 7.3%; OR 3.48, 95% CI 1.29-10.12; p=0.010), and greater use of second-line GVHD-directed therapy with ruxolitinib and/or extracorporeal photopheresis (34.4% vs. 19.8%; OR 2.12, 95% CI 1.05-4.31; p=0.036). In multivariable analyses, reduced cumulative PTCy exposure remained independently associated with acute GVHD (OR 2.29, 95% CI 1.18-4.53; p=0.016) and moderate-to-severe chronic GVHD (OR 4.07, 95% CI 1.52-11.63; p=0.005). Competing-risk analysis confirmed a higher cumulative incidence of acute GVHD (SHR 1.83, 95% CI 1.18-2.83; p=0.007). No significant differences were observed in relapse, non-relapse mortality, or overall survival. In this cohort, cumulative PTCy exposure below 100 mg/kg was associated with increased acute GVHD, a greater burden of clinically significant chronic GVHD, and increased use of second-line GVHD-directed therapies, without significant differences in relapse, non-relapse mortality, or overall survival. These findings suggest that effective GVHD prophylaxis with PTCy may depend on achieving adequate cumulative drug exposure and support prospective studies to define the optimal cumulative PTCy dose for GVHD prophylaxis.
2026-08-09 | Loss of MGAT3 Amplifies TGF-β Signaling to Promote Bronchiolitis Obliterans Progression.
Bronchiolitis obliterans syndrome (BOS), a severe complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT), presents as chronic graft-versus-host disease characterized by inflammation and fibrosis of the small airway epithelium. This progressive fibrosis obstructs bronchiolar airways, leading to respiratory distress in patients. Due to the lack of effective treatments, a deeper understanding of the underlying mechanisms is crucial. This study explored the molecular mechanisms underlying the abnormal glycosylation regulation in BOS, aiming to provide new insights for early diagnosis and treatment of this disease. Bronchoalveolar lavage fluid (BALF) was collected from patients with and without BOS following allo-HSCT. N-glycans from the BALF were enriched using a solid-phase extraction method. Matrix-assisted laser desorption/ionization time-of-flight mass spectrometry (MALDI-TOF/TOF-MS) was used to detect N-glycosylation characteristics in the BALF of BOS patients. This phenomenon was validated using a graft-versus-host disease (GVHD) mouse model. The impact of MGAT3 knockdown on the biological functions of airway epithelial cells (BEAS-2B) was then examined using lentivirus transfection. The molecular mechanism by which the loss of MGAT3 enhances TGF-β signaling was explored using western blotting, enzyme-linked immunosorbent assay (ELISA), and immunofluorescence (IF). Our study investigated N-glycosylation changes in BALF of BOS patients and identified a strong correlation between the loss of bisecting-GlcNAc N-glycans and BOS progression. We found a novel mechanism where MGAT3, the enzyme synthesizing bisecting-GlcNAc structures, critically regulates TGF-β signaling. MGAT3 and bisecting-GlcNAc deficiency significantly enhance TGF-β signaling by increasing TGF-β storage through upregu-lation of latent TGF-β binding protein 1 (LTBP1) and by increasing the availability of TGF-β receptors. Our discovery highlights the crucial role of aberrant glycosylation in BOS progression and holds significant promise for developing novel biomarkers for early diagnosis, identifying new therapeutic targets, and ultimately improving the quality of life for BOS patients.
2026-08-14 | Ruxolitinib-Associated Improvement in Post-transplant Air-Leak Syndrome Complicating Steroid-Dependent Organizing Pneumonia: A Case Report.
Air-leak syndrome (ALS), including pneumothorax, pneumomediastinum, and subcutaneous emphysema, is a rare but life-threatening complication after allogeneic hematopoietic stem cell transplantation (HSCT). However, no standard treatment has yet been established. Here, we report the case of a 60-year-old man with acute myeloid leukemia who underwent allogeneic HSCT. He developed chronic graft-versus-host disease (GVHD) and cryptogenic organizing pneumonia (COP) on day 126 after transplantation. Although initial corticosteroid therapy improved the COP, the relapse occurred during tapering, and repeated steroid treatment failed to achieve sustained disease control. The patient subsequently developed pneumomediastinum, consistent with ALS. Ruxolitinib treatment was initiated on day 265 for steroid-dependent chronic GVHD and organizing pneumonia. Following treatment, oxygenation improved, pneumomediastinum improved, and corticosteroids were successfully tapered and discontinued without COP recurrence. Serum Krebs von den Lungen-6 (KL-6) levels decreased in parallel with clinical and radiological improvement. The introduction of ruxolitinib is associated with improvements in ALS and enabled the discontinuation of steroids. Adverse events included cytopenia and mild liver dysfunction, consistent with known safety profiles. The patient died of acute respiratory failure after transfusion, which was not considered a direct complication of the ruxolitinib treatment. This case suggests that ruxolitinib may facilitate corticosteroid tapering and radiographic improvement of organizing pneumonia-associated ALS after allogeneic transplantation.
2026-08-11 | Phenotype-Specific Associations Between Graft‑Versus‑Host Disease and Post‑Transplant Outcomes.
Disease relapse remains the leading cause of failure following allogeneic hematopoietic cell transplantation (HCT). As novel prophylactic strategies increasingly aim to universally eliminate all forms of graft‑versus‑host disease (GVHD), how these approaches may inadvertently sacrifice graft‑versus‑leukemia (GVL) activity in the post‑transplant cyclophosphamide (PTCy) era is unclear. To evaluate the associations between time‑updated GVHD phenotypes and clinical outcomes, including relapse, non‑relapse mortality (NRM), and overall survival (OS), in patients undergoing PTCy‑based haploidentical or mismatched unrelated donor (MMUD) HCT. This was a retrospective cohort study using the Center for International Blood and Marrow Transplant Research registry. Participants included 7,055 patients with hematologic malignancies who received a first haploidentical or MMUD HCT with PTCy from 2013-2021. Associations with relapse, NRM and OS were evaluated using multi‑state time‑dependent Cox proportional hazards models and a multi‑state random survival forest (MS‑RSF). Time‑updated acute and chronic GVHD phenotypes included isolated grade II acute GVHD (aGVHD), grade III-IV aGVHD, mild chronic GVHD (cGVHD), and immunosuppressive therapy-requiring (IST‑requiring) cGVHD. IST‑requiring cGVHD without antecedent aGVHD was associated with a lower modeled relapse hazard compared with remaining GVHD‑free (Hazard Ratio [HR], 0.74; 95% confidence interval [CI], 0.62-0.89; False Discovery Rate (FDR)-adjusted p (q)=0.006) and lower overall mortality (HR 0.62; 95% CI, 0.53-0.73; q < 0.001). In contrast, grade III-IV aGVHD was associated with significantly higher modeled NRM (HR, 3.15; 95% CI, 2.57-3.84; q < 0.001). Mild cGVHD and isolated grade II aGVHD showed intermediate patterns without consistent associations. These associations were directionally consistent across multiple analytic approaches, including standard time‑dependent Cox regression, dynamic and fixed landmark analyses, and MS‑RSF. In this large PTCy‑treated mismatched donor cohort, IST‑requiring cGVHD was the GVHD phenotype most consistently associated with lower relapse incidence, whereas severe aGVHD was associated with higher NRM. These findings highlight heterogeneity in GVHD phenotypes and suggest that strategies distinguishing toxic acute GVHD from chronic alloreactivity patterns may better balance morbidity and long‑term disease control. Given that relapse remains the predominant cause of post‑transplant mortality, approaches aiming to universally eliminate all GVHD warrant careful reconsideration.
2026-08-11 | Comorbid conditions after allogeneic haematopoietic stem cell transplantation in young adults in Germany-A prospective, multicentre trial.
Young adults (YA) are particularly affected by comorbid conditions following allogeneic haematopoietic stem cell transplantation (alloHSCT), as they have to live with them for many years. This is the first multicentre prospective study investigating sequelae after alloHSCT in YA in Germany. In total, 107 patients who survived relapse-free until day 100 after alloHSCT were enrolled. Median patient age was 29.3 years. Chronic graft-versus-host disease (cGvHD) occurred in 40.2%. Comorbidities were captured at days 100, 180, 365 and 910 by applying the post-transplant multimorbidity index (PTMI). Considering all time points, 12.5%-23.5% of patients had no comorbidity, while 39.2%-49.5% had 1-3, 26.3%-33.0% had 4-6 and 5.7%-8.4% had >6 PTMI-defined comorbidities respectively. The most prevalent PTMI-based comorbidities were iron overload, hypogonadism, arterial hypertension, infections, hepatic dysfunction and osteopenia/osteoporosis. However, the spectrum of PTMI-recorded comorbidities and those concurrently self-reported by patients differed substantially. Factors linked to a higher PTMI were total body irradiation and active cGvHD. Investigating single PTMI-defined comorbidities, we found significant associations of osteopenia/osteoporosis with cGvHD, ongoing immunosuppression and hypogonadism, which was more common among female survivors. These results provide a detailed analysis of the occurrence of a vast spectrum of PTMI-defined comorbidities in YA after alloHSCT.
2026-08-10 | Reduced Cumulative Post-Transplant Cyclophosphamide Exposure Is Associated with Increased Acute and Chronic Graft-versus-Host Disease After Allogeneic Hematopoietic Cell Transplantation.
Post-transplant cyclophosphamide (PTCy) is conventionally administered at a cumulative dose of 100 mg/kg for graft-versus-host disease (GVHD) prophylaxis after allogeneic hematopoietic cell transplantation (allo-HCT). In clinical practice, cumulative PTCy exposure may be reduced because of institutional protocols, physician-directed dose modifications, or body weight-adjusted dosing strategies. However, the clinical consequences of receiving cumulative PTCy doses below the standard 100 mg/kg remain poorly defined. We retrospectively evaluated 251 consecutive allo-HCT recipients transplanted between 2015 and 2025. Among them, 180 patients receiving PTCy-based GVHD prophylaxis were stratified according to cumulative PTCy exposure (<100 vs. ≥100 mg/kg), irrespective of the reason for dose reduction. Acute GVHD, chronic GVHD, relapse, non-relapse mortality (NRM), and overall survival (OS) were analyzed using logistic regression, Cox proportional hazards models, and competing-risk methods. Among the 180 PTCy recipients, 71 received <100 mg/kg and 109 received ≥100 mg/kg; the median cumulative dose in the reduced-exposure group was approximately 80 mg/kg. Reduced cumulative PTCy exposure (<100 mg/kg) was associated with a significantly increased incidence of acute GVHD compared with standard exposure (57.7% vs. 37.0%; OR 2.31, 95% CI 1.21-4.49; p=0.009). Patients receiving <100 mg/kg also experienced a higher burden of clinically significant GVHD, including grade II-IV acute GVHD (25.4% vs. 12.8%; OR 2.30, 95% CI 1.07-5.08; p=0.035), moderate-to-severe chronic GVHD (21.1% vs. 7.3%; OR 3.48, 95% CI 1.29-10.12; p=0.010), and greater use of second-line GVHD-directed therapy with ruxolitinib and/or extracorporeal photopheresis (34.4% vs. 19.8%; OR 2.12, 95% CI 1.05-4.31; p=0.036). In multivariable analyses, reduced cumulative PTCy exposure remained independently associated with acute GVHD (OR 2.29, 95% CI 1.18-4.53; p=0.016) and moderate-to-severe chronic GVHD (OR 4.07, 95% CI 1.52-11.63; p=0.005). Competing-risk analysis confirmed a higher cumulative incidence of acute GVHD (SHR 1.83, 95% CI 1.18-2.83; p=0.007). No significant differences were observed in relapse, non-relapse mortality, or overall survival. In this cohort, cumulative PTCy exposure below 100 mg/kg was associated with increased acute GVHD, a greater burden of clinically significant chronic GVHD, and increased use of second-line GVHD-directed therapies, without significant differences in relapse, non-relapse mortality, or overall survival. These findings suggest that effective GVHD prophylaxis with PTCy may depend on achieving adequate cumulative drug exposure and support prospective studies to define the optimal cumulative PTCy dose for GVHD prophylaxis.
2026-08-09 | Loss of MGAT3 Amplifies TGF-β Signaling to Promote Bronchiolitis Obliterans Progression.
Bronchiolitis obliterans syndrome (BOS), a severe complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT), presents as chronic graft-versus-host disease characterized by inflammation and fibrosis of the small airway epithelium. This progressive fibrosis obstructs bronchiolar airways, leading to respiratory distress in patients. Due to the lack of effective treatments, a deeper understanding of the underlying mechanisms is crucial. This study explored the molecular mechanisms underlying the abnormal glycosylation regulation in BOS, aiming to provide new insights for early diagnosis and treatment of this disease. Bronchoalveolar lavage fluid (BALF) was collected from patients with and without BOS following allo-HSCT. N-glycans from the BALF were enriched using a solid-phase extraction method. Matrix-assisted laser desorption/ionization time-of-flight mass spectrometry (MALDI-TOF/TOF-MS) was used to detect N-glycosylation characteristics in the BALF of BOS patients. This phenomenon was validated using a graft-versus-host disease (GVHD) mouse model. The impact of MGAT3 knockdown on the biological functions of airway epithelial cells (BEAS-2B) was then examined using lentivirus transfection. The molecular mechanism by which the loss of MGAT3 enhances TGF-β signaling was explored using western blotting, enzyme-linked immunosorbent assay (ELISA), and immunofluorescence (IF). Our study investigated N-glycosylation changes in BALF of BOS patients and identified a strong correlation between the loss of bisecting-GlcNAc N-glycans and BOS progression. We found a novel mechanism where MGAT3, the enzyme synthesizing bisecting-GlcNAc structures, critically regulates TGF-β signaling. MGAT3 and bisecting-GlcNAc deficiency significantly enhance TGF-β signaling by increasing TGF-β storage through upregu-lation of latent TGF-β binding protein 1 (LTBP1) and by increasing the availability of TGF-β receptors. Our discovery highlights the crucial role of aberrant glycosylation in BOS progression and holds significant promise for developing novel biomarkers for early diagnosis, identifying new therapeutic targets, and ultimately improving the quality of life for BOS patients.
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Drug Discovery Landscape
16 orphan drug designations for Chronic graft versus host disease, including 2 approved therapies.
16 orphan drug designations for Chronic graft versus host disease, including 2 approved therapies.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
sterile processed derived multiple allogeneic proteins paracrine signaling | cell therapies | FDA | 2026-05-20 | — | Regenerative Ocular Immunobiologics LLC |
rituximab | antibodies | FDA | 2026-05-12 | — | ODDIFACT SAS |
Pegtarazimod | peptides | EMA | 2025-08-22 | — | Clinipace GmbH |
axatilimab-csfr [Niktimvo] | antibodies | FDA | 2021-03-29 | 2024-08-14 | Incyte Corporation |
entospletinib | small molecules | FDA | 2017-08-10 | — | Kronos Bio, Inc. |
Ibrutinib [Imbruvica] | small molecules | EMA | 2016-11-18 | — | Janssen Cilag International |
Avdoralimab | antibodies | EMA | 2016-08-29 | — | [INACTIVE] Alexion Europe |
ibrutinib [Imbruvica] | small molecules | FDA | 2016-06-23 | 2017-08-02 | Pharmacyclics, LLC |
Rimiducid [AP1903] | small molecules | EMA | 2016-05-30 | — | Bellicum Pharma GmbH |
Mocravimod [KRP203] | small molecules | EMA | 2015-12-14 | — | Novartis Europharm Limited |
Efprezimod alfa | proteins | EMA | 2015-11-11 | — | Merck Sharp & Dohme B.V. |
Allogeneic peripheral blood mononuclear cells induced to an early apoptotic state [ApoCell] | cell therapies | EMA | 2015-01-15 | — | [INACTIVE] Richardson Associates Regulatory Affairs Limited |
Donor lymphocyte preparation depleted of functional alloreactive T-cells [Luxceptar] | cell therapies | EMA | 2008-09-05 | — | [INACTIVE] Kiadis Pharma Netherlands B.V. |
autologous lymphocytes depleted ex vivo of immunoreactive T cells using 4,5 | cell therapies | FDA | 2008-04-03 | — | Kiadis Pharma Netherlands B.V. |
Gavilimomab | antibodies | EMA | 2002-11-14 | — | [INACTIVE] SangStat UK Limited |
Thalidomide [Thalidomide Laphal 50 mg Hard capsules] | small molecules | EMA | 2001-07-09 | — | [INACTIVE] Pharmion France |
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