This section highlights FDA-related milestones and regulatory updates for drugs developed by Arcturus Therapeutics (ARCT).
Over the past two years, Arcturus Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ARCT-032, ARCT-810, ARCT-2304, ARCT-154, and LUNAR-H5N1. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
ARCT-032 FDA Regulatory Timeline and Events
ARCT-032 is a drug developed by Arcturus Therapeutics for the following indication: For The Treatment Of Cystic Fibrosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ARCT-032
- Announced Date:
- October 22, 2025
- Indication:
- For The Treatment Of Cystic Fibrosis
Announcement
Arcturus Therapeutics Holdings Inc announced interim results from its ongoing Phase 2 clinical trial of ARCT-032, an investigational inhaled mRNA therapy for people with cystic fibrosis.
AI Summary
Arcturus Therapeutics announced interim results from its Phase 2 trial of ARCT-032, an inhaled mRNA therapy for people with cystic fibrosis. The trial tests daily 10 mg doses in six Class I CF adults over 28 days.
The treatment was generally safe and well tolerated. Mild adverse events seen in Phase 1 appeared early and then resolved. One serious adverse event occurred after dosing ended but was judged unrelated by the Data Monitoring Committee.
AI-enhanced CT scans showed that four of six participants had meaningful reductions in mucus plug number and volume after 28 days of treatment. These imaging results suggest ARCT-032 has biological activity in the lungs.
An exploratory lung function analysis indicated a modest average increase of 3.8% in FEV₁ at Day 42 compared to baseline, though these findings are preliminary.
Arcturus plans to enroll a third cohort at 15 mg and begin a 12-week safety and efficacy study in up to 20 CF participants in the first half of 2026.
Read Announcement- Drug:
- ARCT-032
- Announced Date:
- March 6, 2025
- Indication:
- For The Treatment Of Cystic Fibrosis
Announcement
Arcturus Therapeutics Holdings Inc provided corporate updates.
AI Summary
Arcturus Therapeutics Holdings Inc. provided corporate updates during its Q4 2024 financial results call. The company highlighted steady progress in its rare disease programs, noting that the mRNA therapeutic candidate ARCT-032, designed for cystic fibrosis, and ARCT-810, targeting ornithine transcarbamylase deficiency, have both begun dosing participants. Interim Phase 2 data for both programs is expected by the end of Q2 2025, marking a significant milestone in the company’s clinical development efforts. Additionally, Arcturus emphasized positive advances with its STARR® sa-mRNA vaccines pipeline, reflecting its strong commitment to developing innovative mRNA therapies. The updates also mentioned ongoing collaboration efforts to support regulatory filings and expand manufacturing capabilities, illustrating the company’s focus on strengthening its presence in the mRNA medicine field and its potential impact on treating rare and infectious diseases.
Read Announcement- Drug:
- ARCT-032
- Announced Date:
- January 6, 2025
- Indication:
- For The Treatment Of Cystic Fibrosis
Announcement
Arcturus Therapeutics Holdings Inc announced that the first CF and OTC deficiency participants initiated dosing in December 2024, in the Company's Phase 2 multiple ascending dose studies.
AI Summary
Arcturus Therapeutics Holdings Inc. announced significant progress in its Phase 2 multiple ascending dose studies in December 2024. The company began dosing the first participants in two separate studies targeting cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency. In the CF study (NCT06747858), each enrolled participant will receive daily treatments of ARCT-032 over a period of 28 days. Meanwhile, for the OTC deficiency study, the first participant in the United States began receiving a 0.5 mg/kg dose of ARCT-810, with plans for five intravenous infusions over two months.
These developments highlight Arcturus’ commitment to addressing severe unmet needs in both CF and OTC deficiency patients. Interim data from these Phase 2 mRNA therapeutic studies is expected in the first half of 2025, marking an important milestone in their ongoing clinical programs.
Read Announcement- Drug:
- ARCT-032
- Announced Date:
- September 3, 2024
- Indication:
- For The Treatment Of Cystic Fibrosis
Announcement
Arcturus Therapeutics Holdings Inc announced that the U.S. Food and Drug Administration (FDA) has issued a "Study May Proceed" notification for the Company's Investigational New Drug (IND) application, ARCT-032, to treat cystic fibrosis (CF).
AI Summary
Arcturus Therapeutics announced that the U.S. Food and Drug Administration (FDA) has given a "Study May Proceed" notification for its Investigational New Drug (IND) application for ARCT-032, a potential treatment for cystic fibrosis (CF). This notification clears the way for the company to start a Phase 2 multiple ascending dose study. In this phase, ARCT-032 will be tested to determine its safety, tolerability, and effectiveness in people with CF who do not respond well to existing CFTR modulator therapies.
Using its novel LUNAR® technology, ARCT-032 is designed as an inhaled mRNA therapeutic aimed at restoring normal CFTR function in the lungs. This milestone represents an important step for Arcturus as it works to bring a new treatment option to address the critical needs of patients suffering from this life-threatening condition.
Read Announcement- Drug:
- ARCT-032
- Announced Date:
- May 28, 2024
- Indication:
- For The Treatment Of Cystic Fibrosis
Announcement
Arcturus Therapeutics Holdings Inc. announced Arcturus will be presenting Phase 1 results in healthy volunteers and new Phase 1b interim data in CF patients for ARCT-032, an inhaled investigational mRNA therapeutic to treat CF, at the 47th European Cystic Fibrosis Conference on June 7, 2024.
AI Summary
Arcturus Therapeutics Holdings Inc. will present new clinical trial data for ARCT-032 at the 47th European Cystic Fibrosis Conference on June 7, 2024. The company announced that ARCT-032, an inhaled investigational mRNA therapeutic, is being studied to treat cystic fibrosis by delivering CFTR mRNA to the lungs. In the Phase 1b study, early results from four cystic fibrosis patients showed a promising average improvement of 4% in FEV1, a measure of lung function, after just two inhaled doses. Additionally, the company will share Phase 1 results from healthy volunteers, which supported the safety and tolerability of the treatment with no serious adverse events reported. These encouraging early findings highlight ARCT-032’s potential to improve lung function in cystic fibrosis patients and add to the ongoing clinical development of this novel therapeutic approach.
Read Announcement
ARCT-810 FDA Regulatory Events
ARCT-810 is a drug developed by Arcturus Therapeutics for the following indication: To treat ornithine transcarbamylase deficiency.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ARCT-810
- Announced Date:
- June 23, 2025
- Indication:
- To treat ornithine transcarbamylase deficiency
Announcement
Arcturus Therapeutics Holdings Inc announced that it will host a virtual KOL presentation of ARCT-810 Phase 2 interim data for Ornithine Transcarbamylase (OTC) deficiencyon Monday, June 30, 2025, at 12:00 p.m. ET.
AI Summary
Arcturus Therapeutics Holdings Inc. announced it will host a virtual KOL presentation to review ARCT-810 Phase 2 interim data for Ornithine Transcarbamylase (OTC) deficiency. The event is scheduled for Monday, June 30, 2025, at 12:00 p.m. ET, with a press release summarizing the data to be issued beforehand at 11:00 a.m. ET. During this presentation, key clinical data will be discussed, focusing on the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescents and adults with OTC deficiency. The session will feature participation from leading experts in the field, including renowned professionals like Dr. Marshall Summar and Dr. Johannes Häberle, who bring extensive experience in managing and researching urea cycle disorders. This virtual presentation aims to provide valuable insights on ARCT-810’s progress as a potential transformative treatment for OTC deficiency.
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ARCT-2304 FDA Regulatory Events
ARCT-2304 is a drug developed by Arcturus Therapeutics for the following indication: For pandemic influenza disease caused by H5N1 virus.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ARCT-2304
- Announced Date:
- April 10, 2025
- Indication:
- For pandemic influenza disease caused by H5N1 virus.
Announcement
Arcturus Therapeutics Holdings Inc announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the self-amplifying mRNA (sa-mRNA) vaccine candidate, ARCT-2304, designed for active immunization to protect against disease caused by influenza A H5N1 subtype contained in the vaccine.
AI Summary
Arcturus Therapeutics Holdings Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its self-amplifying mRNA vaccine candidate, ARCT-2304. This vaccine is designed for active immunization to protect against diseases caused by the influenza A H5N1 subtype. The Fast Track Designation helps speed up the development and review process by offering enhanced communication with the FDA, priority review, and a rolling review process. This expedited review is important for addressing the unmet medical need of preventing serious conditions that could lead to a pandemic. The designation highlights the innovative approach of using the STARR® sa-mRNA platform combined with LUNAR® delivery technology to achieve effective protection at lower doses. Arcturus views this milestone as a significant step in its commitment to developing next-generation mRNA vaccines to protect global public health.
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ARCT-154 FDA Regulatory Events
ARCT-154 is a drug developed by Arcturus Therapeutics for the following indication: SARS-CoV-2 Delta Variant and Other Variants of Concern.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ARCT-154
- Announced Date:
- February 14, 2025
- Indication:
- SARS-CoV-2 Delta Variant and Other Variants of Concern
Announcement
Arcturus Therapeutics announced that the European Commission has granted marketing authorization for KOSTAIVE® (ARCT-154), a self-amplifying mRNA COVID-19 vaccine, for individuals 18 years and older. KOSTAIVE is the first sa-mRNA COVID-19 vaccine to receive approval from the European Commission (EC). KOSTAIVE is currently marketed in Japan against COVID-19.
AI Summary
The European Commission has granted marketing authorization for KOSTAIVE® (ARCT-154), a self-amplifying mRNA COVID-19 vaccine developed by Arcturus Therapeutics. Approved for individuals aged 18 and older, this decision makes KOSTAIVE the first sa-mRNA COVID-19 vaccine to receive approval from the European Commission. The vaccine is already available in Japan, where it is used to combat COVID-19, and the European approval will allow its marketing across all EU and EEA countries.
This milestone reflects the potential advantages of self-amplifying mRNA technology, which can stimulate a stronger and longer-lasting immune response. The positive regulatory decision follows encouraging clinical data demonstrating robust immunogenicity and sustained antibody levels. KOSTAIVE’s approval highlights the ongoing efforts to expand the range of available COVID-19 vaccines in the fight against the global pandemic.
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LUNAR-H5N1 FDA Regulatory Events
LUNAR-H5N1 is a drug developed by Arcturus Therapeutics for the following indication: For H5N1 Flu Vaccine.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- LUNAR-H5N1
- Announced Date:
- January 10, 2025
- Indication:
- For H5N1 Flu Vaccine
Announcement
Arcturus Therapeutics Holdings Inc. announced the initiation of the Company's Phase 1 study of ARCT-2304, a self-amplifying mRNA (sa-mRNA) vaccine candidate, also known as LUNAR-H5N1, for active immunization to prevent pandemic influenza disease caused by H5N1 virus.
AI Summary
Arcturus Therapeutics announced the start of its Phase 1 study for ARCT-2304, also known as LUNAR-H5N1, a self-amplifying mRNA vaccine candidate designed to prevent pandemic influenza caused by the H5N1 virus. The trial is a randomized, placebo-controlled study being conducted at multiple sites in the United States and will enroll about 200 healthy adults across two age groups. Screening began in November 2024, and the first participant was inoculated in December 2024. The study will assess the vaccine’s safety and immune response by measuring key markers such as hemagglutination inhibition, virus microneutralization, and neuraminidase enzyme-linked lectin assays. This approach uses Arcturus’ LUNAR® delivery and STARR® mRNA platform technologies, which have previously shown strong immune responses at low doses. The trial is fully funded by the Biomedical Advanced Research and Development Authority (BARDA).
Read Announcement
