Sequana Medical NV has received U.S. Food and Drug Administration (FDA) Premarket Approval (PMA) to market alfapump as a treatment for recurrent or refractory ascites due to liver cirrhosis.

The approval gives alfapump the distinction of being the first active implantable U.S. medical device that automatically and continuously removes ascites from the abdomen into the bladder. The company estimates there will be approximately 70,000 U.S. patients with recurrent or refractory ascites, representing a 2025 alfapump market opportunity worth more than $2 billion. The patient population is forecast to reach 130,000 patients by 2032, primarily driven by NASH/MASH and alcoholic liver disease.¹

“The approval of alfapump by the FDA opens the door to a breakthrough therapeutic option for patients with ascites,” commented H.E. Vargas, M.D., professor of Medicine for the Mayo Clinic College of Medicine (Phoenix), POSEIDON Study principal investigator. “The introduction of the system will be welcome by patients, families, and their care providers as this technology not only provides effective treatment,  but also has been shown to improve quality of life.”⁴

The company is preparing for the U.S. commercial launch of the alfapump System in H2 2025 through a focused specialty sales force concentrating on the 90 liver transplant centers that represent the large majority of target customers. The company has received FDA Breakthrough Device Designation for the alfapump System and has applied, and expects to receive, NTAP (new technology add-on payments) which should facilitate patient access to the device. In addition to the existing ICD-10 procedure codes for the alfapump procedure, the American Medical Association has granted six new CPT⁵ III reimbursement codes, available for use by healthcare professionals and payers since July 1, 2024, for procedures related to the alfapump System.

“With a standard of care that has seen minimal change in thousands of years, this marks a huge milestone for the large and growing liver ascites community in the U.S.,” Sequana Medical CEO Ian Crosbie stated. “Our vision is to transform the lives of these patients. Recurrent or refractory liver ascites is a devastating condition with a terrible impact on the lives of our patients and their caregivers, not only for the painful and burdensome paracentesis procedure itself but also in the weeks leading up to each drainage. The POSEIDON clinical study results demonstrate that the alfapump can virtually eliminate the need for therapeutic paracentesis, and improve patient quality of life, with a safety profile comparable to standard of care, delivering a 21st century solution that can allow these patients to take back their lives.^3,4 We wish to thank all the patients and investigators who made this possible by participating in the POSEIDON study. With over 1,000 alfapump systems implanted to date, we look forward to starting commercialization in H2 2025, bringing this solution to many more patients in need.”

Recurrent or refractory ascites is a severe condition characterized by the accumulation of abdominal fluid. The current standard treatment involves therapeutic paracentesis, an invasive and burdensome procedure that drains ascites from the abdomen using a large needle over an extended period. The alfapump is touted as the first active implantable medical device in the United States that automatically and continuously removes ascites from the abdomen into the bladder, where it is naturally eliminated through urination. To date, more than 1,000 alfapump systems have been implanted.

“We are highly encouraged by the enthusiastic feedback for the alfapump that we are receiving from the clinical community—there is a clear need for improved treatment options and we are putting in place the commercial team to deliver our novel therapy,” Sequana Medical Chief Commercial Officer Martijn Blom said. “Preparations for commercial launch are underway, and we are already working with target centers for the initial commercial launch planned for H2 2025. It is a huge privilege to bring such a breakthrough in therapy to the clinical and patient communities, and we are excited to work with them to establish alfapump as the new standard of care.”

The U.S. market of recurrent and refractory ascites due to liver cirrhosis is forecast to grow by 9% annually, from approximately 70,000 patients in 2025 to 130,000 patients by 2032, primarily driven by the increasing prevalence of NASH / MASH.^2,6 The total market opportunity for alfapump is estimated at more than $2 billion in 2025, including approximately $500 million from the company’s initial priority target market of patients requiring at least 12 paracenteses annually.

“The receipt of the PMA approval for our alfapump System is a major milestone for our company and reflects many years of diligent work by the Sequana team,” Sequana Medical Global Vice President of QM/QA/RA Timur Resch stated. “We wish to thank the FDA for its collaboration and support in bringing this device to U.S. patients who will be very grateful for this new treatment option.”

The FDA’s approval of the PMA is based on the successful execution of Sequana Medical’s pivotal POSEIDON study, a landmark study across 18 U.S. and Canadian centers with 69 total patients implanted with the alfapump. The primary effectiveness endpoints at six months post-implantation in the Pivotal Cohort⁷ exceeded the predefined thresholds with statistical significance, and primary safety endpoint data was in line with expectations.⁸ Data at 12 months post-implantation continued to show a strong and durable clinical profile, virtually eliminating the need for therapeutic paracentesis and delivering an improvement in quality of life (as defined by subjective physical health (assessed by SF-36 PCS) and ascites symptoms (assessed by Ascites Q)).⁹ At AASLD’s The Liver Meeting in November 2024, key POSEIDON investigators reported the alfapump virtually eliminated the need for large volume paracentesis at 24 months, with overall 62% survival.¹⁰

Data from the patient preference study and a matched cohort analysis of the NACSELD-III registry with the POSEIDON Pivotal Cohort indicated that US patients have a strong preference for the alfapump compared with standard paracentesis procedures and the alfapump’s safety profile is comparable to standard of care.¹¹

Sequana Medical NV is a pioneer in treating fluid overload, a serious and frequent clinical complication in patients with liver disease, heart failure and cancer. This causes major medical issues including increased mortality, repeated hospitalizations, severe pain, difficulty breathing and restricted mobility. Although diuretics are standard of care, they become ineffective, intolerable or exacerbate the problem in many patients. There are limited effective treatment options, resulting in poor clinical outcomes, high costs and a major impact on their quality of life. Sequana Medical aims to provide treatment options for this large and growing “diuretic resistant” patient population. alfapump and DSR are Sequana Medical’s proprietary platforms that work with the body to treat diuretic-resistant fluid overload, and are intended to deliver major clinical and quality of life benefits for patients, while reducing costs for healthcare systems.

Results of the Company’s RED DESERT and SAHARA proof-of-concept studies in heart failure published in the European Journal of Heart Failure in April 2024 support DSR’s mechanism of action as breaking the cardiorenal syndrome cycle. All three patients from the non-randomized cohort of MOJAVE, a U.S. randomized controlled multi-center Phase 1/2a clinical study, have been successfully treated with DSR, resulting in a dramatic improvement in diuretic response and virtual elimination of loop diuretic requirements.¹² The independent Data Safety Monitoring Board approved the start of the randomized MOJAVE cohort of up to a further 30 patients, which is planned after alfapump U.S. PMA approval.

Sequana Medical is listed on the regulated market of Euronext Brussels and headquartered in Ghent, Belgium.

The alfapump System is currently not approved in Canada. DSR therapy is still in development and is currently not approved in any country.

References
¹ Based on US and Canada market assessment conducted by highly experienced international consulting group
² NASH – Non-alcoholic steatohepatitis; MASH – Metabolic dysfunction-associated steatohepatitis
³ POSEIDON study results in SSED (PMA P230044, FDA approval letter on file) – 100% median reduction in number of therapeutic paracentesis per month in Pivotal Cohort
⁴ Quality of life as defined by subjective physical health (assessed by SF-36 PCS) and ascites symptoms (assessed by Ascites Q)
⁵ CPT copyright 2024 American Medical Association. All rights reserved. CPT® is a registered trademark of the American Medical Association.
⁶ Based on US and Canada market assessment conducted by highly experienced international consulting group
⁷ The Pivotal Cohort is used for the primary effectiveness endpoints and consists of 40 patients implanted with the alfapump
⁸ Data reported in press release of 25 October 2022
⁹ Data reported in press release of 19 October 2023
¹⁰ Based upon the pivotal cohort of the POSEIDON study, data reported in press release of 18 November 2024
¹¹ Data reported in press release of 19 October 2023; Patient Preference study conducted by RTI Health Solutions, and matched cohort analysis presented by Dr. Bajaj at EASL Congress 2024.
¹² Data reported in press release of 25 March 2024; mean increase of 326% in six-hour urinary sodium excretion at 3 months follow up vs baseline, and 95% reduction of loop diuretics over same period.