RARE DISEASE
Invasive scopulariopsis infection
Invasive scopulariopsis infection
Invasive scopulariopsis infection
Drug discovery
3
drugs
With orphan designations
Overview
Invasive Scopulariopsis infection is a rare but life-threatening opportunistic fungal disease caused primarily by Scopulariopsis brevicaulis and related species. These dematiaceous or hyaline molds predominantly affect immunocompromised patients, causing disseminated or localized deep tissue infections (e.g., pneumonia, endocarditis, brain abscesses) [1][4][7]. Diagnosis is challenging due to nonspecific clinical presentations, and treatment is complicated by intrinsic resistance to most antifungals [1][3][11]. Mortality remains high despite aggressive interventions [4][7].
Burden
High mortality (up to 85–100% in disseminated cases) due to diagnostic delays and antifungal resistance [4][7][10].
Rising incidence in immunocompromised populations, with increasing reports of breakthrough infections post-transplant [4][7].
6%-17% of superficial mycosis cases involve Scopulariopsis, but invasive forms account for <1% of systemic fungal infections [2][14].
Therapies
Antifungals: Limited efficacy; posaconazole-terbinafine or amphotericin B-caspofungin combinations show partial synergy in vitro [3][11][15]. Liposomal amphotericin B and voriconazole may modestly improve outcomes [7][10].
Surgical intervention: Debridement or excision of necrotic tissue is critical for localized infections [11][15].
Experimental agents: Olorofim (a novel antifungal) shows promise in refractory cases [10].
Categories: rare infectious diseases, rare transplant-related disorders
Research Papers
71 drug discovery papers about Invasive scopulariopsis infection, with 2 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
71 drug discovery papers about Invasive scopulariopsis infection, with 2 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
categories:
Small molecules
small molecules
2026-06-01 | Invasive Scopulariopsis brevicaulis infection associated with CARD9 mutation: Clinical and therapeutic insights
Hyalohyphomycosis caused by Scopulariopsis species is an uncommon but clinically significant entity, particularly in immunocompromised individuals. Our case describes a rare presentation of chronic, progressive cutaneous hyalohyphomycosis due to S. brevicaulis in a young patient with a confirmed CARD9 mutation. The prolonged disease course, resistance to conventional antifungal therapy, and extensive local destruction highlight the diagnostic and therapeutic challenges associated with this organism. This report is unique in demonstrating the association between S. brevicaulis infection and CARD9 deficiency, which has been rarely documented. Additionally, it emphasizes the role of combination antifungal therapy, including voriconazole, terbinafine, and saturated solution of potassium iodide, resulting in clinical improvement after prior treatment failure.
2026-03-25 | Past, present, and emerging antifungals against Fusarium and other rare non-Aspergillus hyalohyphomycetes: a narrative review.
Invasive fungal infections caused by Fusarium and other rare hyalohyphomycetes, such as Scopulariopsis and Paecilomyces species, can lead to devastating localized and disseminated infections in vulnerable patient populations. Currently employed antifungal agents exhibit limited effectiveness for these molds. Over the past few years, the approval of new antifungals and the ongoing development of others in clinical trials offer promising new therapeutic options for treating these infections. In this review, utilizing PubMed literature review, we delve into selected hyalohyphomycoses and provide an overview of the pharmacokinetics and pharmacodynamics of rezafungin, ibrexafungerp, olorofim, and fosmanogepix. We examine the available in-vitro and in-vivo efficacy data for these antifungals. Rezafungin and ibrexafungerp inhibit different steps in the synthesis of (1,3) β-d-glucan, and rezafungin is a notably long-acting echinocandin; however, both have primarily been studied against candidiasis. Olorofim and fosmanogepix, in contrast, are novel antifungals in development with unique mechanisms of action with activity against hyalohyphomycoses. Both rezafungin and ibrexafungerp have limited efficacy and clinical experience in treating mold infections. Fosmanogepix and olorofim hold promise for treating invasive mold infections, including hyalohyphomycoses; however, given in-vitro antifungal susceptibility variability against hyalohyphomycetes, and lack of clinical trial information, future studies are needed.
2026-01-01 | P-473. Posaconazole therapeutic drug monitoring: the key to an effective antifungal prophylaxis
Abstract Background Posaconazole suspension (POSAsusp) is widely used for the prophylaxis of invasive fungal infections (IFI) in children, however, the optimal pediatric dosing regimen remains undefined. Furthermore, due to its low bioavailability and high variability, therapeutic drug monitoring (TDM) is necessary. Our aim is to describe TDM of POSAsusp, identify factors that affect pharmacokinetics and evaluate its safety and effectiveness. Methods Retrospective observational study including patients ≤18 years of age that received prophylactic POSAsusp during ≥1 week and with ≥1 TDM between 2017-2023. Posaconazole plasma trough concentrations (Ctrough) were targeted at 0.7-2.5 μg/ml. Results Seventy-one patients were included, of whom 48 (67.6%) were male. The median age was 4.8 years (IQR 2.4-8.9 years). The most frequent underlying causes were primary immunodeficiencies (32.4%) and acute lymphoblastic leukemia (19.7%). Fifty-five (77.5 %) patients received an HSCT. A total of 373 Ctrough were obtained, and only 178 (47.8%) fell within the established therapeutic range. Of these, 187 (50.1%) were considered sub-therapeutic, while the remaining 8 (2.1%) were supra-therapeutic. Although there was no significant correlation with age group, the dose required in patients under 2 years of age was higher than with the rest of age groups (17.6 mg/kg/day vs. 12.8 mg/kg/day). Gastrointestinal disorders, concomitant use of PPIs and HSCT were significantly associated with subtherapeutic Ctrough. There were six patients (8.5%) with breakthrough IFI (2 A.fumigatus, 2 Candida spp., 1 Scopulariopsis spp., 1 without microbiological identification), and they were associated with subtherapeutic Ctrough (70.8% vs. 48.7%, p< 0,05). 2/6 patients died due to breakthrough IFI. Drug-related toxicity resulted in drug discontinuation in 4 patients (5.6%) (hypertransaminasemia, vomiting, erythroderma), 1/4 had supratherapeutic Ctrough. Conclusion TDM is essential in patients receiving POSAsusp as antifungal prophylaxis as it ensures that patients obtain target Ctrough in order to prevent breakthrough IFI. It is especially necessary in patients with risk factors such as gastrointestinal disorders or PPI use. Further research is required to establish optimal dosing for patients under 2 years of age. Disclosures Natalia A. Mendoza-Palomar, n/a, Gilead Sciences: Honoraria|Gilead Sciences: Inscription, travel and accomodation for infectious diseases meetings|Pfizer: Advisor/Consultant|Pfizer: Honoraria Pere Soler-Palacín, MD, PhD, MSc, Astellas: Research grants|CLS Behring: Grant/Research Support|Gilead: Grant/Research Support|Grifols: Grant/Research Support|Pfizer: Grant/Research Support|Takeda: Grant/Research Support
2025-09-29 | Endocarditis caused by Scopulariopsis brevicaulis with disseminated emboli and multiple vascular aneurysms: A case report and literature review.
Scopulariopsis brevicaulis, a saprophytic fungus often associated with onychomycosis, has seldom been reported to cause deep-tissue infections in immunocompromised patients. To review the antifungal susceptibility of S. brevicaulis and management strategies for related endocarditis and other invasive infections. A literature search was conducted using PubMed until September 2024. This review presents a case of S. brevicaulis prosthetic valve endocarditis, emphasizing the diagnostic challenges and clinical implications of disseminated infection.A summary of published cases of endocarditis and other deep-tissue infections is included along with a discussion on the antifungal susceptibility of S. brevicaulis and current treatment strategies. S. brevicaulis presents a significant treatment challenge due to its rare and opportunistic nature, along with frequent multi-drug resistance. Although there is no single optimal therapy for this mold, a practical approach combines medical and surgical strategies customized to individual cases, employing a variety of agents based on in vitro sensitivity testing. Determining minimum inhibitory concentrations (MICs) is essential for guiding treatment decisions, even in the absence of established clinical breakpoints for this pathogen. Olorofilm, a novel oral antifungal, demonstrates remarkable potential for treating S. brevicaulis.
2024-11-11 | DETERMINATION OF ANTIFUNGAL ACTIVITY OF POPLAR BUDS AND PROPOLIS AGAINST COVID-19 STRAINS OF POSTCOID INFECTIONS
The new coronavirus infection is currently still an important problem for humanity as well as the global medical community. The COVID-19 virus is dangerous because it causes direct damage to the epithelium of the respiratory tract, thereby contributing to the penetration of bacteria and fungi into the tissues of the body. Invasive mycoses are a serious complication and cause a high percentage of deaths in hospitalized patients. The main strains of coinfections in COVID-19 are: Aspergillus, Mucorales and Candida. The issue of treatment and the possibility of prevention of secondary fungal infections in coronavirus infection is particularly acute. The aim of the work was to determine the antifungal activity of poplar (Populus L.) buds and propolis with antimicrobial activity against fungal strains isolated from patients with a new coronavirus infection. The paper reflects the results of the study of antifungal activity of the investigated samples of extracts of Populus nigra L. and Populus rubrinervis Hort. Alb. buds (70 and 96% ethyl alcohol) against clinical strains of Aspergillus fumigatus, Aspergillus flavus, Aspergillus niger, Scopulariopsis brevicaulis, Mucor spp. Propolis tincture and alcoholic solution of the reference sample (RS) of pinostrobin were the comparison preparations. The greatest activity of extracts from Populus rubrinervis Hort. Alb. buds against Aspergillus fumigatus (the content of active substances in 96% ethyl alcohol extraction – 0.0022%), Aspergillus flavus (70% ethyl alcohol extraction – 0.0019%), Aspergillus niger (70% ethyl alcohol extraction – 0.0019%), Scopulariopsis brevicaulis (70% ethyl alcohol extraction – 0.0009%). Propolis tincture showed the lowest fungicidal activity, has activity against A. niger and Scopulariopsis brevicaulis strains. The comparison preparation – alcoholic solution of RS pinostrobin showed antifungal activity against A. niger strain. Against Mucor spp. the tested extracts of poplar buds and comparison preparations did not show any pronounced fungicidal activity. The presence of fungicidal activity against strains of postcoid fungi of extracts of poplar bud extracts is presumably associated with the presence of the sum of phenolic compounds: flavonoids and phenylpropanoids.
small molecules
2026-06-01 | Invasive Scopulariopsis brevicaulis infection associated with CARD9 mutation: Clinical and therapeutic insights
Hyalohyphomycosis caused by Scopulariopsis species is an uncommon but clinically significant entity, particularly in immunocompromised individuals. Our case describes a rare presentation of chronic, progressive cutaneous hyalohyphomycosis due to S. brevicaulis in a young patient with a confirmed CARD9 mutation. The prolonged disease course, resistance to conventional antifungal therapy, and extensive local destruction highlight the diagnostic and therapeutic challenges associated with this organism. This report is unique in demonstrating the association between S. brevicaulis infection and CARD9 deficiency, which has been rarely documented. Additionally, it emphasizes the role of combination antifungal therapy, including voriconazole, terbinafine, and saturated solution of potassium iodide, resulting in clinical improvement after prior treatment failure.
2026-03-25 | Past, present, and emerging antifungals against Fusarium and other rare non-Aspergillus hyalohyphomycetes: a narrative review.
Invasive fungal infections caused by Fusarium and other rare hyalohyphomycetes, such as Scopulariopsis and Paecilomyces species, can lead to devastating localized and disseminated infections in vulnerable patient populations. Currently employed antifungal agents exhibit limited effectiveness for these molds. Over the past few years, the approval of new antifungals and the ongoing development of others in clinical trials offer promising new therapeutic options for treating these infections. In this review, utilizing PubMed literature review, we delve into selected hyalohyphomycoses and provide an overview of the pharmacokinetics and pharmacodynamics of rezafungin, ibrexafungerp, olorofim, and fosmanogepix. We examine the available in-vitro and in-vivo efficacy data for these antifungals. Rezafungin and ibrexafungerp inhibit different steps in the synthesis of (1,3) β-d-glucan, and rezafungin is a notably long-acting echinocandin; however, both have primarily been studied against candidiasis. Olorofim and fosmanogepix, in contrast, are novel antifungals in development with unique mechanisms of action with activity against hyalohyphomycoses. Both rezafungin and ibrexafungerp have limited efficacy and clinical experience in treating mold infections. Fosmanogepix and olorofim hold promise for treating invasive mold infections, including hyalohyphomycoses; however, given in-vitro antifungal susceptibility variability against hyalohyphomycetes, and lack of clinical trial information, future studies are needed.
2026-01-01 | P-473. Posaconazole therapeutic drug monitoring: the key to an effective antifungal prophylaxis
Abstract Background Posaconazole suspension (POSAsusp) is widely used for the prophylaxis of invasive fungal infections (IFI) in children, however, the optimal pediatric dosing regimen remains undefined. Furthermore, due to its low bioavailability and high variability, therapeutic drug monitoring (TDM) is necessary. Our aim is to describe TDM of POSAsusp, identify factors that affect pharmacokinetics and evaluate its safety and effectiveness. Methods Retrospective observational study including patients ≤18 years of age that received prophylactic POSAsusp during ≥1 week and with ≥1 TDM between 2017-2023. Posaconazole plasma trough concentrations (Ctrough) were targeted at 0.7-2.5 μg/ml. Results Seventy-one patients were included, of whom 48 (67.6%) were male. The median age was 4.8 years (IQR 2.4-8.9 years). The most frequent underlying causes were primary immunodeficiencies (32.4%) and acute lymphoblastic leukemia (19.7%). Fifty-five (77.5 %) patients received an HSCT. A total of 373 Ctrough were obtained, and only 178 (47.8%) fell within the established therapeutic range. Of these, 187 (50.1%) were considered sub-therapeutic, while the remaining 8 (2.1%) were supra-therapeutic. Although there was no significant correlation with age group, the dose required in patients under 2 years of age was higher than with the rest of age groups (17.6 mg/kg/day vs. 12.8 mg/kg/day). Gastrointestinal disorders, concomitant use of PPIs and HSCT were significantly associated with subtherapeutic Ctrough. There were six patients (8.5%) with breakthrough IFI (2 A.fumigatus, 2 Candida spp., 1 Scopulariopsis spp., 1 without microbiological identification), and they were associated with subtherapeutic Ctrough (70.8% vs. 48.7%, p< 0,05). 2/6 patients died due to breakthrough IFI. Drug-related toxicity resulted in drug discontinuation in 4 patients (5.6%) (hypertransaminasemia, vomiting, erythroderma), 1/4 had supratherapeutic Ctrough. Conclusion TDM is essential in patients receiving POSAsusp as antifungal prophylaxis as it ensures that patients obtain target Ctrough in order to prevent breakthrough IFI. It is especially necessary in patients with risk factors such as gastrointestinal disorders or PPI use. Further research is required to establish optimal dosing for patients under 2 years of age. Disclosures Natalia A. Mendoza-Palomar, n/a, Gilead Sciences: Honoraria|Gilead Sciences: Inscription, travel and accomodation for infectious diseases meetings|Pfizer: Advisor/Consultant|Pfizer: Honoraria Pere Soler-Palacín, MD, PhD, MSc, Astellas: Research grants|CLS Behring: Grant/Research Support|Gilead: Grant/Research Support|Grifols: Grant/Research Support|Pfizer: Grant/Research Support|Takeda: Grant/Research Support
2025-09-29 | Endocarditis caused by Scopulariopsis brevicaulis with disseminated emboli and multiple vascular aneurysms: A case report and literature review.
Scopulariopsis brevicaulis, a saprophytic fungus often associated with onychomycosis, has seldom been reported to cause deep-tissue infections in immunocompromised patients. To review the antifungal susceptibility of S. brevicaulis and management strategies for related endocarditis and other invasive infections. A literature search was conducted using PubMed until September 2024. This review presents a case of S. brevicaulis prosthetic valve endocarditis, emphasizing the diagnostic challenges and clinical implications of disseminated infection.A summary of published cases of endocarditis and other deep-tissue infections is included along with a discussion on the antifungal susceptibility of S. brevicaulis and current treatment strategies. S. brevicaulis presents a significant treatment challenge due to its rare and opportunistic nature, along with frequent multi-drug resistance. Although there is no single optimal therapy for this mold, a practical approach combines medical and surgical strategies customized to individual cases, employing a variety of agents based on in vitro sensitivity testing. Determining minimum inhibitory concentrations (MICs) is essential for guiding treatment decisions, even in the absence of established clinical breakpoints for this pathogen. Olorofilm, a novel oral antifungal, demonstrates remarkable potential for treating S. brevicaulis.
2024-11-11 | DETERMINATION OF ANTIFUNGAL ACTIVITY OF POPLAR BUDS AND PROPOLIS AGAINST COVID-19 STRAINS OF POSTCOID INFECTIONS
The new coronavirus infection is currently still an important problem for humanity as well as the global medical community. The COVID-19 virus is dangerous because it causes direct damage to the epithelium of the respiratory tract, thereby contributing to the penetration of bacteria and fungi into the tissues of the body. Invasive mycoses are a serious complication and cause a high percentage of deaths in hospitalized patients. The main strains of coinfections in COVID-19 are: Aspergillus, Mucorales and Candida. The issue of treatment and the possibility of prevention of secondary fungal infections in coronavirus infection is particularly acute. The aim of the work was to determine the antifungal activity of poplar (Populus L.) buds and propolis with antimicrobial activity against fungal strains isolated from patients with a new coronavirus infection. The paper reflects the results of the study of antifungal activity of the investigated samples of extracts of Populus nigra L. and Populus rubrinervis Hort. Alb. buds (70 and 96% ethyl alcohol) against clinical strains of Aspergillus fumigatus, Aspergillus flavus, Aspergillus niger, Scopulariopsis brevicaulis, Mucor spp. Propolis tincture and alcoholic solution of the reference sample (RS) of pinostrobin were the comparison preparations. The greatest activity of extracts from Populus rubrinervis Hort. Alb. buds against Aspergillus fumigatus (the content of active substances in 96% ethyl alcohol extraction – 0.0022%), Aspergillus flavus (70% ethyl alcohol extraction – 0.0019%), Aspergillus niger (70% ethyl alcohol extraction – 0.0019%), Scopulariopsis brevicaulis (70% ethyl alcohol extraction – 0.0009%). Propolis tincture showed the lowest fungicidal activity, has activity against A. niger and Scopulariopsis brevicaulis strains. The comparison preparation – alcoholic solution of RS pinostrobin showed antifungal activity against A. niger strain. Against Mucor spp. the tested extracts of poplar buds and comparison preparations did not show any pronounced fungicidal activity. The presence of fungicidal activity against strains of postcoid fungi of extracts of poplar bud extracts is presumably associated with the presence of the sum of phenolic compounds: flavonoids and phenylpropanoids.
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Drug Discovery Landscape
3 orphan drug designations for Invasive scopulariopsis infection.
3 orphan drug designations for Invasive scopulariopsis infection.
Drug | Therapy type | Orphan designation | Clinical trial status | Sponsor |
|---|---|---|---|---|
Olorofim | small molecules | EMA | — | Shionogi B.V. |
olorofim | small molecules | FDA | F2G Limited | |
Amphotericin B lipid complex | — | FDA | — | The Liposome Company, Inc. |
“Terminated” – programs with discontinued clinical trials, or with no updates reported for 5 years after completion or 12 years after the last trial started.
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