Sequana Medical submits Premarket Approval application to US FDA for alfapump® in recurrent or refractory ascites due to liver cirrhosis
Sequana Medical NV (Euronext Brussels: SEQUA) (the "Company" or "Sequana Medical"), a pioneer in the treatment of fluid overload in liver disease, heart failure and cancer, today announces that it has submitted a Premarket Approval (PMA) application to the US Food and Drug Administration (FDA) for alfapump, the Company’s fully implantable, wirelessly charged device for patients with recurrent or refractory ascites due to liver cirrhosis. The alfapump received breakthrough device designation from the US FDA in 2019.
Recurrent or refractory ascites is a severe condition characterized by the accumulation of fluid in the abdomen. 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. If approved by the FDA, the alfapump could become the first active implantable medical device in the US that automatically and continuously removes ascites from the abdomen into the bladder, where it is naturally eliminated through urination.
Timur Resch, Global Vice President QM/QA/RA at Sequana Medical, commented: “The submission of our Premarket Approval application to the FDA is the result of an enormous team effort and a clear demonstration of our intensive preparation to fulfil US regulatory requirements. We have great confidence in the strength of our PMA submission and look forward to work in close collaboration with the FDA to facilitate a seamless and thorough review process intended to bring our breakthrough device to the US market as soon as possible.”
Ian Crosbie, Chief Executive Officer of Sequana Medical, added: “This is a key milestone for the alfapump and underscores our commitment to improving treatment options for patients with recurrent or refractory liver ascites. This overlooked patient group is forecast to grow strongly due to NASH / MASH and today’s limited treatment options often lead to poor clinical outcomes, severely reduced quality of life, a substantial burden on their caregivers and high costs to payors. Data from our North American pivotal study demonstrate the potential for alfapump to transform the lives of these patients by virtually eliminating the need for paracentesis and delivering clinicially important improvements in quality of life. We anticipate FDA approval in the second half of 2024 and look forward to introducing the alfapump through our own specialy salesforce focused on US liver transplant centers.”
The PMA filing is based on the successful execution of Sequana Medical’s pivotal POSEIDON study, a landmark study across 18 centers in the US and Canada with a total of 69 patients implanted with the alfapump. The primary effectiveness endpoints at six months post-implantation in the Pivotal Cohort (i) exceeded the predefined thresholds with statistical significance, and primary safety endpoint data was in line with expectations (ii). Data at 12 months post-implantation continued to show a strong and durable clinical profile, virtually eliminating the need for therapeutic paracentesis and delivering a clinically meaningful improvement in patients’ quality of life (iii).
Data from the patient preference study and a matched cohort analysis of the NACSELD (iv) registry with the POSEIDON Pivotal Cohort indicated that US patients have a strong preference for the alfapump vs standard paracentesis procedures and that the safety profile of the alfapump is comparable to standard of care.
The North American market of recurrent and refractory ascites due to liver cirrhosis is forecast to grow 6-7% per year, from 78,000 patients in 2025, reaching a market opportunity for alfapump of over $2.5 billion by 2035, with NASH being the major driver of growth (v). To date, over 1,000 alfapump systems have been implanted.
(i) The Pivotal Cohort is used for the primary effectiveness endpoints and consists of 40 patients implanted with the alfapump
(ii) Data reported in press release of 25 October 2022
(iii) Data reported in press release of 19 October 2023
(iv) NACSELD is the North American Consortium for the Study of End Stage Liver Disease. A matched cohort analysis was conducted by an independent group comparing outcomes of decompensated cirrhosis patients from the NACSELD-III registry to those from the POSEIDON study; see press release of 19 October 2023
(v) Based on US and Canada market assessment conducted by highly experienced international consulting group, estimating over 170,000 patients with recurrent or refractory ascites in North America by 2035, with estimated incidence of 60% and based on $25K for price of alfapump