NDA Application for Drug to Treat HRS-1 Announced
Mallinckrodt has announced a rolling submission of a New Drug Application (NDA) to the US FDA for terlipressin, an agent being investigated for the treatment of hepatorenal syndrome type 1 (HRS-1). The global biopharmaceutical company has submitted its clinical data package to the FDA. The rolling submission will allow for submission of portions of the regulatory applications to the FDA as they are completed. Mallinckrodt expects to complete the NDA submission in the coming months.
According to a press release, HRS-1 is an acute and life-threatening complication characterized by acute kidney failure in patients with cirrhosis. If left untreated, median survival time among patients with HRS-1 is less than 2 weeks and the mortality rate is more than 80% within three months of diagnosis. Currently, there are no approved drug therapies for HRS-1 in the United States; HRS-1 affects an estimated 30,000 to 40,000 patients annually in the United States.
Steven Romano, MD, executive vice president and chief scientific officer at Mallinckrodt, said, “This is an important milestone that brings us closer to potentially delivering the first FDA-approved treatment in the US for HRS-1, a life-threatening, difficult-to-treat condition. We are grateful to the patients, their families and caregivers, and the researchers who made this achievement possible.”
The NDA for terlipressin is based, in part, on results from CONFIRM, a phase 3 prospective study conducted among patients with HRS-1. The trial met its primary end point of verified HRS reversal, defined as improvement in renal function, avoidance of dialysis therapy, and short-term survival. Serious adverse events were reported in 65% of participants in the terlipressin group and 60.6% of those in the placebo group; there were no new or unexpected adverse events reported. Initial results of the CONFIRM trial were reported at The Liver Meeting® 2019, the annual meeting of the American Association for the Study of Liver Diseases.
FMCNA Announces Telehealth Solution
In a recent press release, Fresenius Medical Care North America (FMCNA) announced the planned rollout of a new telehealth solution for its home dialysis patients. FMCNA is a leading provider of kidney care products and services and its telehealth program is designed to improve collaboration among patients, the clinical care team, and physicians to facilitate the rapid growth of home therapies for patients with end-stage renal disease.
Following implementation of a series of pilot programs in 2019, FMCNA signed an agreement to seamlessly integrate telehealth into theHub, the company’s connect health platform.
Joe Turk, president of home and critical care therapies at FMCNA, said, “We are excited to bring this user-friendly telehealth solution to our home dialysis patients, part of our effort to improve the experience for patients managing their own treatments. This solution will help people living with kidney disease better connect with their care team, including nephrologists, nurses, social workers, and dietitians.”
The press release noted that as a result of legislation at the federal level, under certain conditions, Medicare allows for a home dialysis patient to meet with their nephrologist and care team for their monthly visit via telehealth. Telehealth is also supported by many private insurers. Telehealth can improve access to care while reducing travel burden for patients, particularly for patients residing in rural areas. Telehealth also provides a more frequent visual touchpoint with the care team, increasing the rates of successful home therapies.
Ahmad Sharif, MD, chief medical information officer for FMCNA, said, “We have rapidly accelerated development of new connected health technologies to improve the patient, physician, and staff experience. When home patients are more connected to their care team through remote monitoring, recent studies show fewer hospital admissions and less technique failure. This new telehealth solution will be critical to expanding the adoption of home dialysis for people living with kidney failure.”
The new telehealth program will launch initially in select markets and roll out more widely later this year, the press release said.
American Kidney Fund Grants Aid Patients with Kidney Failure
In 2019, the number of kidney transplants reached an all-time high: more than 24,000. Of those, according to a press release from the American Kidney Fund (AKF), more than one in 20 transplants were made possible with financial assistance from the AKF. The press release announced that 1400 of the AKF grant recipients living with kidney failure received a kidney transplant and post-transplant care in 2019.
Patients who received the life-saving transplants were living in 48 states and Puerto Rico and ranged from 13 to 80 years of age. More than 60% of the AKF grant recipients who received a transplant last year are members of racial and ethnic minority groups; racial and ethnic minorities are disproportionately affected by kidney disease, yet generally receive fewer kidney transplants than white Americans, according to the press release.
LaVarne A. Burton, president and CEO of AKF, said, “Patients face so many obstacles on the road to getting a transplant, and the American Kidney Fund helps them overcome one very significant hurdle—the financial barriers to maintaining comprehensive health coverage necessary for transplant. Our charitable premium assistance program is a lifeline that helps low-income patients access the full range of healthcare services needed to treat this complex condition and stay as healthy as possible—allowing them to qualify for the transplant waiting list, go through the transplant workup, have the surgery itself, and receive post-transplant care. Our assistance continues for the full insurance plan year post-transplant, providing support to patients as they recover and adjust to post-transplant living.”
The press release noted that AKF is also working to make it easier to become a living kidney donor. In 2019, nine states enacted legislation spearheaded by AKF that will protect living kidney donors from insurance discrimination and make it possible for them to have job-protected leave. This year, efforts to champion such legislation in 15 additional states are being led by AKF; the Fund also actively supports the federal Living Donor Protection Act of 2019.
Public Awareness Campaign Launched
The National Kidney Foundation (NKF), the American Society of Nephrology (ASN), and the US Department of Health and Human Services (HHS) collaborated to create a nationwide public awareness campaign highlighting the risk for kidney disease. The campaign was launched as part of National Kidney Month in March. The campaign focused on the 33% of Americans at risk for kidney disease, including those with diabetes, heart disease, hypertension, obesity, and a family history of the disease.
In a joint press release, Kevin Longino, CEO of NKF and a kidney transplant recipient, said, “Look around the next time you’re sitting in a school auditorium or even in a giant, professional sports stadium; one-third of every adult in there with you is at risk of developing kidney disease. We believe it’s essential to reach the more than 80,000,000 American adults at risk, because if you can diagnose and treat kidney disease early, you can help stave off its life-threatening complications.”
The Are You the 33%? Campaign, cosponsored by the NKF, HHS, and ASN, is part of a public awareness initiative of the Advancing American Kidney Health plan. ASN president, Anupam Agarwal, MD, FASN, said, “More than 90% of the 37,000,000 Americans and 850,000,000 individuals worldwide affected with kidney disease are unaware that they are even sick. This silent epidemic often strikes without symptoms. Millions of people won’t know they have kidney disease until their kidneys stop working and it’s too late.”
The campaign seeks to change those odds by urging every American adult to take a simple, one-minute quiz at MinuteForYourKidneys.org. HHS Secretary Alex Azar said, “My own family has known the burdens of kidney disease, including how taxing dialysis can be. Helping Americans understand their risk for kidney disease and take steps to prevent it is and must be a top public health priority.”
Lillian Pryor Begins Term as ANNA President
Lillian Pryor, MSN, RN, CNN, has begun her 16-month term as president of the American Nephrology Nursing Association (ANNA). According to a press release from ANNA, Ms. Pryor began her term on January 1. She will work with the ANNA board of directors and Tamara Kear, PhD, RN, CNN, FAAN, executive director to oversee all operations and strategic planning for the 8300 member organization.
Ms. Pryor has been involved with ANNA since 1990, including serving as president-elect, ANNA director, chairperson of the ANNA awards & scholarships committee, and representative to the Kidney Health Initiative Patient Perspective Task Force. She has also published articles in and served as a peer reviewer for the Nephrology Nursing Journal.
“It is an honor to serve as ANNA president, and I look forward to upholding our mission and vision,” she said. “Many changes are on the horizon for nephrology care delivery, and nurses have the crucial responsibility of advocating for patients and improving care. We’ll work diligently to ensure nurses’ and patients’ voices are heard.”
Vifor Pharma Gains BBB- Credit Rating
In a March press release, Vifor Pharma announced that Fitch Ratings has initiated coverage of Vifor Pharma with an investment grade BBB- credit rating and a stable outlook. According to the release, the credit rating is “supported by Vifor Pharma’s leading position in growing niche markets, solid organic growth prospects, and its strategic partnership with Fresenius Medical Care, as well as the Group’s solid financial profile.”
“We are very pleased with this outcome as it represents a significant step towards gaining access to the US unsecured corporate debt market,” Colin Bond, chief financial officer at Vifor Pharma, said. “This investment grade credit rating from Fitch is a product of Vifor Pharma’s continued focus on the execution of its three strategic growth drivers and the delivery of the milestone 2020 plan. This second investment grade credit rating will further support us in our development as a global specialty pharmaceuticals company.”