What Is Olorofim?
Olorofim is a drug that has been investigated in clinical trials for its potential therapeutic effects. While the specific mechanism of action is not detailed in the provided trial descriptions, olorofim is being studied for its use in treating a range of serious conditions, primarily focusing on fungal infections. These include Invasive Fungal Infections, Coccidioidal Meningitis, and Invasive Aspergillosis.
Overall, 8 clinical trials have been conducted or are currently underway involving olorofim, with a total enrollment of 548 participants. The first trial began in 2018, and the latest trial is projected to conclude in 2026. In addition to studies in patients with specific infections, olorofim has also been evaluated in healthy volunteers and individuals with hepatic or renal impairment to assess its safety and how it is processed by the body. F2G Biotech GmbH has sponsored the majority of these investigations.
Uses and Conditions Under Study
Olorofim is being investigated across several clinical conditions, primarily focusing on serious fungal infections and pharmacokinetic studies in specific populations.
- Fungal Infections: Olorofim is under study for its potential to treat various fungal infections. This includes Invasive Fungal Infections, which are serious infections that can spread throughout the body. It is also being investigated for Invasive Aspergillosis, a severe lung infection caused by a common mold, and Coccidioidal Meningitis, a fungal infection that affects the brain and spinal cord. A total of 4 trials have explored olorofim's use in these specific fungal conditions.
- Pharmacokinetic Studies in Specific Populations: To understand how olorofim is absorbed, distributed, metabolized, and excreted, trials have been conducted in specific patient groups. This includes individuals with Hepatic Impairment (liver problems) and Renal Impairment (kidney problems). These studies help determine appropriate dosing for patients with compromised organ function. Each of these conditions has been the focus of 1 trial.
- Studies in Healthy Volunteers: Olorofim has also been administered to Healthy participants in 2 trials. These studies are typically conducted early in drug development to assess safety, tolerability, and basic pharmacokinetic properties in individuals without underlying health conditions.
Dosing
Olorofim has been studied in various dosage forms and strengths across its clinical trials. The drug has been administered as an oral medication, with dosage forms including intact tablets, and for administration via nasogastric (NG) tube. It has also been studied in intravenous (IV) form.
Investigational dosing regimens have included both single and multiple oral doses. For instance, one study involved a single oral dose of 150 mg or 200 mg. Another regimen described a loading dose of 5 tablets (totaling 150 mg) taken twice daily on Day 1, followed by a maintenance dose of 3 tablets (90 mg) twice daily from Day 2 until Day 84 (± 7 days).
Studies have also explored olorofim's pharmacokinetics under different conditions, such as oral administration in fasted or fed states, and in individuals with normal or impaired hepatic and renal function. Specific cohorts for these studies included those with severe renal impairment, normal renal function, mild hepatic impairment, moderate hepatic impairment, and normal hepatic function.
Side Effects
Information regarding the side effects of Olorofim was not provided in the clinical trial data.
Clinical Trial Results
Clinical trial results for Olorofim come from a study (NCT03583164) that evaluated its use in participants with invasive fungal infections who had limited treatment options. The study assessed various outcomes at Day 42 and Day 84.
Mortality Rates
- The all-cause mortality rate for patients treated with Olorofim was 11.9% at Day 42.
- This rate increased to 16.3% at Day 84.
Overall Response Rates
Overall response indicates a positive outcome based on a comprehensive assessment of the infection. At Day 42:
- The Data Review Committee (DRC) adjudicated overall response rate was 28.7%.
- The investigator-assessed overall response rate was 24.8%.
At Day 84:
- The DRC adjudicated overall response rate was 27.2%.
- The investigator-assessed overall response rate was 29.7%.
Clinical Response Rates
Clinical response refers to an improvement in the patient's signs and symptoms of infection. At Day 42:
- The DRC adjudicated clinical response rate was 59.9%.
- The investigator-assessed clinical response rate was 53.5%.
At Day 84:
- The DRC adjudicated clinical response rate was 54.0%.
- The investigator-assessed clinical response rate was 56.4%.
Mycological Response Rates
Mycological response indicates a reduction or elimination of the fungal pathogen. At Day 42:
- The DRC adjudicated mycological response rate was 18.3%.
- The investigator-assessed mycological response rate was 25.7%.
At Day 84:
- The DRC adjudicated mycological response rate was 22.5%.
- The investigator-assessed mycological response rate was 31.7%.
Overall Response by Fungal Species (Day 42)
The DRC adjudicated overall response at Day 42 varied by the type of fungal infection:
- For all Aspergillus species, the response rate was 34.7%.
- For Scedosporium species, the response rate was 36.4%.
- For Lomentospora Prolificans, the response rate was 42.3%.
- For other Olorofim-susceptible fungi, the response rate was 33.3%.
- No overall response was observed for Coccidioides species (0.0%).
Currently Recruiting Trials
At this time, there are no clinical trials for Olorofim actively recruiting new participants. Clinical trials are essential for evaluating new treatments, and while Olorofim has been studied in the past, there are no open opportunities to enroll in a study for this drug right now. We encourage you to check back periodically, as trial statuses can change, and new studies may open for enrollment in the future.
Where to Participate
Currently, there are no active clinical trial sites for Olorofim in any city or state. This means there are no locations where you can participate in a study for this investigational drug at present. When trials for Olorofim were active, they generally sought participants of all genders, but healthy volunteers were not eligible. Specific age requirements were not broadly specified, and trials were not open to children.
Development Timeline
The journey of Olorofim in clinical development began on July 11, 2018, with the initiation of its first clinical trial. Since then, a total of 8 clinical trials have been conducted, involving 548 participants. The primary driving force behind Olorofim's development has been F2G Biotech GmbH, which has sponsored 7 of these trials, with one additional trial sponsored by Fariba Donovan.
Olorofim's development has progressed through various phases, starting with 5 Phase 1 trials to assess safety and dosage. This was followed by 2 Phase 2 trials, which further evaluated the drug's effectiveness and side effects. The drug has also advanced to a Phase 3 trial, a crucial stage for confirming its benefits and monitoring side effects in a larger population.
The scope of conditions studied for Olorofim has also evolved over time. Initial investigations focused on conditions such as IBS-C and hyperphosphatemia. However, the development pipeline expanded significantly to include serious fungal infections and other medical conditions. Olorofim has since been studied for Coccidioidal Meningitis, Hepatic Impairment, Invasive Aspergillosis, and Renal Impairment, demonstrating a broader potential application for this investigational treatment.