Praxis Precision Medicines Announces FDA Acceptance and Priority Review of New Drug Application for Relutrigine in Patients with SCN2A and SCN8A DEEs

Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for relutrigine, for the treatment of SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The FDA has set a target action date under the Prescription Drug User Fee Act (PDUFA) of September 27, 2026.

• FDA assigned PDUFA target action date of September 27, 2026

“Our first FDA acceptance of an NDA submission marks a significant milestone in our evolution to a commercial-stage company and an important step toward delivering innovative, precision neuroscience therapies to patients in need. SCN2A/8A DEEs have no currently approved targeted therapies and relutrigine, if approved, would be the first disease-modifying therapy for children suffering from these devastating and fatal conditions. We look forward to working closely with the FDA during the review process while continuing to advance our launch preparations,” said Marcio Souza, president and chief executive officer.

Relutrigine for treatment of SCN2A/8A DEEs

The NDA is supported by positive results from the EMBOLD study, which was stopped early for efficacy following a successful interim analysis and recommendation from the Data Monitoring Committee. Relutrigine has an Orphan Drug Designation, as well as a Rare Pediatric Disease Designation and a Breakthrough Therapy Designation. If granted approval, relutrigine will be the first FDA-approved therapy for SCN2A/8A DEE as well as be eligible for a Pediatric Review Voucher.

Relutrigine is also being investigated in broad DEEs through the EMERALD trial, which is expected to be completed by the end of 2026.

About Relutrigine

Relutrigine is a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathies (DEEs) as a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in severe DEEs. Relutrigine’s mechanism of precision sodium channel (NaV) modulation is consistent with superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of relutrigine have demonstrated dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A, SCN8A and other DEE mouse models. Relutrigine has been generally well-tolerated in three Phase 1 studies and has demonstrated biomarker changes indicative of NaV channel modulation. Data from cohort 1 of the Phase 2 EMBOLD study demonstrated a well-tolerated, robust, short- and long-term improvement in motor seizures in a heavily pre-treated population, alongside maintained seizure freedom in some patients with SCN2A- and SCN8A-DEE. Relutrigine has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA for the treatment of SCN2A-DEE, SCN8A-DEE and Dravet syndrome; as well as Breakthrough Therapy Designation (BTD), and ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE.

Praxis Precision Medicines Announces FDA Acceptance and Priority Review of New Drug Application for Relutrigine in Patients with SCN2A and SCN8A DEEs

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure

SQ Innovation, Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved its drug-device combination Lasix ONYU (furosemide injection) for the treatment of edema (due to fluid overload) in adult patients with chronic heart failure. Lasix ONYU was developed to enable subcutaneous infusion of furosemide outside the healthcare setting for selected patients, as prescribed by a clinician without the need for a healthcare professional to administer the drug.

About 6.7 million Americans suffer from heart failure, with the prevalence expected to rise to 8.7 million by 2030. Heart failure is a leading cause of hospitalizations for individuals aged 65 and older with approximately 1.2 million hospitalizations per year [1].

Lasix ONYU consists of a novel high-concentration formulation of furosemide combined with a state-of-the-art small Infusor for treatment at home. The innovative design includes a reusable unit that can be used for 48 treatments and a plastic sterile single-use unit that is discarded after treatment. The two-component design reduces manufacturing complexity and cost, allowing the product to be offered at a different, more favorable price point which is expected to reduce barriers to widespread adoption.

“Lasix ONYU has the potential to be transformative in the care of patients experiencing worsening heart failure due to fluid overload,” said Pieter Muntendam, MD, founder, President and CEO of SQ Innovation. “Treating selected patients at home offers important benefits to patients, health systems and payors. We look forward to launching Lasix ONYU with leading health systems in the 4th quarter of 2025.”

Bioavailability and diuretic response were determined in a clinical study in which Lasix ONYU demonstrated complete bioavailability (112%) resulting in similar diuresis (115%) and natriuresis (117%) when compared to the same dose given by IV bolus. The biphasic delivery of furosemide by the Infusor resulted in a tempered diuretic response while IV bolus administration led to a shorter period of more intense diuresis. The results of the study were published in a leading cardiovascular journal [2].

“Heart failure is the most common serious medical condition in the U.S. and affects about one in four Americans during their lifetime. The number of patients affected is expected to double over the next 20 years and we currently already often lack adequate resources to take care of the 6.7 million patients affected presently – there are not enough beds, clinicians and funds”, said Dr. Javed Butler, Professor of Medicine at University of Mississippi and President, Baylor Scott and White Research Institute. “The only two actionable solutions now are more widespread adoption of guideline directed medical therapy (GDMT) and treating more patients at home with products such as subcutaneous diuretics instead of hospitalization for intravenous diuretics.”

“Decongestion through use of IV diuretics has been the cornerstone of treatment for reducing edema and hypervolemia in heart failure patients for over five decades,” stated S. Craig Thomas, Immediate Past President of the American Association of Heart Failure Nurses (AAHFN), an organization dedicated to advancing nursing education, clinical practice, and research to improve outcomes for heart failure patients. “The availability of accessible, affordable, and novel options that do not require the presence of a healthcare professional allows for transformative new clinical care-delivery. This means patients who now would typically need to be hospitalized for several days of IV treatment can instead remain home, supported by periodic or remote monitoring. The significance of this shift away from inpatient care for patients, hospitals, and payers cannot be overstated.”

Starting this quarter, Lasix ONYU will be available from leading pharmaceutical distributors enabling timely availability at participating medical facilities and affiliated retail pharmacies.

SQ Innovation is hosting a Conference Call and Webcast on Thursday October 9, at 4:30pm ET to introduce the product and answer questions from the community. Participating in the conference call will be:

Pieter Muntendam, MD, President and CEO of SQ Innovation

Mustafa M. Ahmed, MD, Professor of Medicine, Section Chief, Heart Failure, University of Florida Health, Gainesville, FL

Craig Thomas, Nurse Practitioner, Advanced Heart Failure Center, University of Virginia Health System, Charlottesville, VA and Immediate Past President American Association of Heart Failure Nurses (AAHFN)

https://en.wikipedia.org/wiki/Furosemide

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure