This section highlights FDA-related milestones and regulatory updates for drugs developed by Edgewise Therapeutics (EWTX).
Over the past two years, Edgewise Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
EDG-5506, EDG-7500, and sevasemten. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
EDG-5506 - FDA Regulatory Timeline and Events
EDG-5506 is a drug developed by Edgewise Therapeutics for the following indication: Duchenne and Becker muscular dystrophy (DMD and BMD).
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:
- EDG-5506
- Announced Date:
- October 1, 2024
- Indication:
- Duchenne and Becker muscular dystrophy (DMD and BMD)
Announcement
Edgewise Therapeutics, Inc announced its participation at the 29th International Annual Congress of the World Muscle Society (WMS) with an industry-sponsored symposium and the presentation of seven scientific posters..
AI Summary
Edgewise Therapeutics, Inc. announced its participation at the 29th International Annual Congress of the World Muscle Society (WMS) in Prague. The company will host an industry-sponsored symposium titled “Understanding disease progression and a potential novel agent to protect muscle,” featuring key opinion leaders including Dr. Luca Bello and Dr. Craig McDonald. In addition, Edgewise will present seven scientific posters that focus on sevasemten, an oral fast myosin inhibitor designed to reduce muscle damage in individuals with Becker muscular dystrophy. The posters will highlight research on reduced muscle damage biomarkers, functional stabilization, and the patient experience, drawing on findings from clinical studies such as DUNE, ARCH, and insights from the Phase 2 CANYON trial. This involvement at WMS underscores Edgewise’s commitment to advancing research in muscular dystrophies and sharing significant scientific progress with the global medical community.
Read Announcement- Drug:
- EDG-5506
- Announced Date:
- April 23, 2024
- Indication:
- Duchenne and Becker muscular dystrophy (DMD and BMD)
Announcement
Edgewise Therapeutics, Inc announced that the European Medicines Agency (EMA) has granted Orphan Drug Designations for sevasemten for the treatment of Becker muscular dystrophy (Becker) and for the treatment of Duchenne muscular dystrophy (Duchenne).
AI Summary
Edgewise Therapeutics announced that the European Medicines Agency (EMA) has granted Orphan Drug Designations for sevasemten for the treatment of Becker muscular dystrophy and Duchenne muscular dystrophy. This designation is a significant milestone, as it offers benefits like reduced regulatory fees and up to 10 years of market exclusivity in the European Union. Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage, a key factor in the progression of these debilitating conditions.
The EMA’s decision highlights a shared recognition of the urgent need for new therapeutic options for patients suffering from these rare muscle disorders. Edgewise is committed to advancing sevasemten through clinical development in both Becker and Duchenne, providing hope for improved management and quality of life for affected individuals and their caregivers.
Read Announcement- Drug:
- EDG-5506
- Announced Date:
- April 15, 2024
- Indication:
- Duchenne and Becker muscular dystrophy (DMD and BMD)
Announcement
Edgewise Therapeutics, Inc announced positive two-year topline results from the ARCH trial. ARCH is an open label, single-center study assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK) and functional measures with sevasemten (EDG-5506) in adults with Becker.
AI Summary
Edgewise Therapeutics, Inc. announced positive two-year topline results from the ARCH trial, a study focusing on sevasemten (EDG-5506) for adults with Becker Muscular Dystrophy. The open-label, single-center trial assessed safety, tolerability, muscle damage biomarkers, pharmacokinetics, and functional measures. Researchers found that the North Star Ambulatory Assessment (NSAA) remained stable over the two-year period, contrasting with the decline usually seen in Becker natural history studies.
In addition to stable NSAA scores, significant decreases were seen in key biomarkers such as creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2), indicating a reduction in muscle damage. Sevasemten was well-tolerated by all 12 participants, with no adverse events leading to dose adjustments or discontinuations. These promising results support further exploration of sevasemten as a treatment option for Becker Muscular Dystrophy.
Read Announcement- Drug:
- EDG-5506
- Announced Date:
- April 15, 2024
- Indication:
- Duchenne and Becker muscular dystrophy (DMD and BMD)
Announcement
Edgewise Therapeutics, Inc announced positive two-year topline results from the ARCH trial. ARCH is an open label, single-center study assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK) and functional measures with sevasemten (EDG-5506) in adults with Becker.
AI Summary
Edgewise Therapeutics, Inc. announced positive two-year topline results from the ARCH trial, an open-label, single-center study in adults with Becker muscular dystrophy. The study evaluated the safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics, and functional measures with sevasemten (EDG-5506). Over the two-year period, sevasemten was well-tolerated by all 12 participants with no dose reductions or discontinuations due to side effects.
The results showed that participants’ muscle function, measured using the North Star Ambulatory Assessment (NSAA), remained stable, which contrasts with the decline typically observed in natural history studies. Additionally, significant decreases in key muscle damage biomarkers, creatine kinase and fast skeletal muscle troponin I, were reported. These findings support sevasemten’s potential to reduce contraction-induced muscle damage and preserve muscle function in patients with Becker muscular dystrophy.
Read Announcement
EDG-7500 - FDA Regulatory Timeline and Events
EDG-7500 is a drug developed by Edgewise Therapeutics for the following indication: For Hypertrophic Cardiomyopathy (HCM) and Other Serious Diseases of Cardiac Diastolic Dysfunction.
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:
- EDG-7500
- Announced Date:
- April 2, 2025
- Indication:
- For Hypertrophic Cardiomyopathy (HCM) and Other Serious Diseases of Cardiac Diastolic Dysfunction
Announcement
Edgewise Therapeutics, Inc announced positive top-line data of EDG-7500 from the Phase 2 CIRRUS-HCM four-week trial in participants with obstructive or nonobstructive HCM.
AI Summary
Edgewise Therapeutics, Inc. announced encouraging top-line results from its Phase 2 CIRRUS-HCM trial evaluating EDG-7500 over four weeks in participants with both obstructive and nonobstructive hypertrophic cardiomyopathy (HCM). The study showed that EDG-7500, a novel oral cardiac sarcomere modulator, provided rapid and meaningful reductions in left ventricular outflow tract gradients in patients with obstructive HCM. At a 100 mg dosage, reductions reached 71% at rest, and similar improvements were seen post-Valsalva. The treatment also led to significant decreases in NT-proBNP, a key heart failure biomarker, and showed positive trends in diastolic function measures.
Additionally, participants experienced noticeable improvements in daily functioning and quality of life, as reflected by better Kansas City Cardiomyopathy Questionnaire scores and NYHA class ratings. Importantly, EDG-7500 was well-tolerated with no significant reductions in left ventricular ejection fraction, supporting its potential as a promising therapeutic option for patients with HCM.
Read Announcement- Drug:
- EDG-7500
- Announced Date:
- March 26, 2025
- Estimated Event Date Range:
- April 2, 2025 - April 2, 2025
- Target Action Date:
- April 02, 2025
- Indication:
- For Hypertrophic Cardiomyopathy (HCM) and Other Serious Diseases of Cardiac Diastolic Dysfunction
Announcement
Edgewise Therapeutics, Inc., announced that members of the management team will hold a live webcast to discuss top-line data of EDG-7500 from the Phase 2 CIRRUS-HCM 28-day trial in patients with obstructive or nonobstructive HCM on Wednesday, April 2, 2025, at 8:30 am ET.
AI Summary
Edgewise Therapeutics, Inc. announced that its management team will host a live webcast on Wednesday, April 2, 2025, at 8:30 am ET. During the webcast, they will discuss the top-line data from the Phase 2 CIRRUS-HCM 28-day trial, which studied the effects of EDG-7500 in patients with either obstructive or nonobstructive hypertrophic cardiomyopathy (HCM).
EDG-7500 is a new oral therapy designed to slow early contraction velocity and help improve cardiac relaxation in patients with HCM and other related conditions. The event is expected to provide insights from leading investigators and will include a slide presentation to help explain the trial results. Interested participants can register for the live webcast and access the replay on the company’s events page.
Read Announcement- Drug:
- EDG-7500
- Announced Date:
- September 19, 2024
- Indication:
- For Hypertrophic Cardiomyopathy (HCM) and Other Serious Diseases of Cardiac Diastolic Dysfunction
Announcement
Edgewise Therapeutics, Inc announced top-line data of EDG-7500 from the Phase 1 trial in healthy subjects and the single-dose arm of the Phase 2 CIRRUS-HCMtrial in patients with obstructive HCM.
AI Summary
Edgewise Therapeutics announced top-line data for its novel oral drug EDG-7500 from both a Phase 1 trial in healthy subjects and the single-dose arm of its Phase 2 CIRRUS-HCM trial in patients with obstructive hypertrophic cardiomyopathy (HCM). In the Phase 1 study, EDG-7500 was well tolerated at various dosages with no meaningful changes in left ventricle ejection fraction (LVEF). In the Phase 2 trial, patients receiving 100 or 200 mg doses experienced significant reductions in left ventricular outflow tract (LVOT) gradients—a 67% drop at rest and 55% during provocation—while maintaining stable LVEF. These results suggest that EDG-7500 may improve heart relaxation and reduce obstruction in HCM without compromising heart function. Edgewise is now dosing patients in a 28-day trial, with further data expected in early 2025 to continue assessing the drug’s potential benefits.
Read Announcement- Drug:
- EDG-7500
- Announced Date:
- September 17, 2024
- Indication:
- For Hypertrophic Cardiomyopathy (HCM) and Other Serious Diseases of Cardiac Diastolic Dysfunction
Announcement
Edgewise Therapeutics, Inc announced that members of the management team will hold a live webcast to discuss top-line data of EDG-7500 from the Phase 1 trial in healthy subjects and the single-dose arm of the Phase 2 CIRRUS-HCMtrial in patients with obstructive HCM on Thursday, September 19, 2024, at 8:30 am ET.
AI Summary
Edgewise Therapeutics, Inc. announced a live webcast on Thursday, September 19, 2024, at 8:30 am ET. During the webcast, members of the management team will discuss the top-line data from the Phase 1 trial of EDG-7500 in healthy subjects and the single-dose arm of the Phase 2 CIRRUS-HCM trial in patients with obstructive hypertrophic cardiomyopathy (HCM). The webcast will also feature insights from CIRRUS-HCM investigator Dr. Anjali T. Owens, who will share her views on EDG-7500 and its potential impact on HCM treatment.
An accompanying slide presentation will be available, offering further information on the data and study progress. Interested viewers can register for both the live event and the replay by visiting the Edgewise events page.
Read Announcement
sevasemten - FDA Regulatory Timeline and Events
sevasemten is a drug developed by Edgewise Therapeutics for the following indication: For Becker muscular dystrophy.
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:
- sevasemten
- Announced Date:
- June 26, 2025
- Indication:
- For Becker muscular dystrophy
Announcement
Edgewise Therapeutics, Inc today unveiled positive results in its sevasemten program for Becker and Duchenne muscular dystrophies.
AI Summary
Edgewise Therapeutics announced positive outcomes from its sevasemten program for Becker and Duchenne muscular dystrophies. In the Becker extension trial (MESA), patients showed sustained disease stabilization over three years. Data from MESA confirmed steady improvements and maintenance of North Star Ambulatory Assessment (NSAA) scores, echoing findings from previous trials. These results may help in slowing disease progression and improving motor function in Becker muscular dystrophy.
In addition, Phase 2 observations in Duchenne muscular dystrophy provided valuable insights into dosing strategies. The LYNX and FOX studies helped define a 10 mg dose that was well tolerated and showed potential in reducing functional decline. Edgewise plans to discuss these developments in an upcoming virtual investor event, underscoring the progress of sevasemten as a promising treatment option for patients suffering from these challenging muscle disorders.
Read Announcement- Drug:
- sevasemten
- Announced Date:
- December 16, 2024
- Indication:
- For Becker muscular dystrophy
Announcement
Edgewise Therapeutics, Inc announced positive topline results from the Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy.
AI Summary
Edgewise Therapeutics announced positive topline results from its Phase 2 CANYON trial evaluating sevasemten in individuals with Becker muscular dystrophy. In this largest interventional Becker study to date, sevasemten met the primary endpoint by reducing creatine kinase levels, a key biomarker for skeletal muscle damage. Additionally, patients treated with sevasemten showed stabilization of the North Star Ambulatory Assessment (NSAA), with trends toward improved motor function compared to placebo. The drug, a first-in-class fast skeletal myosin inhibitor, aims to protect muscle from contraction-induced damage. Sevasemten was well-tolerated, and no new safety concerns were observed during the trial, even in patients with advanced disease. Edgewise plans to further discuss these encouraging results during an upcoming virtual investor event, as the company continues to engage with the FDA and European Medicines Agency on future regulatory strategies.
Read Announcement
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