Across the Kidneyverse: December 29, 2023

This week: Tarpeyo, Aldosterone, KPD Milestones & More!

Dec 29, 2023

Welcome to Signals, where we explore the frontiers of healthcare & technology, one [space] at a time. While many of us hit pause to celebrate the holidays, here’s one last dose of optimism from the Kidneyverse for 2023. Happy New Year, my fellow explorers!

IN THIS ISSUE

Headlines

Here’s what caught my eye over the past two weeks:

A team of researchers led by Dr. Katherine Tuttle believe they may have “broken the code” to fight kidney disease. (Seattle Times)
The FDA released draft guidance on use of Real-World Evidence for medical devices. (FDA)
Penn Medicine just set the world record with 100 kidney paired donation transplants in a single year. (Penn Medicine News)
Tarpeyo (Calliditas) becomes the first fully FDA-approved drug for IgA nephropathy based on a measure of kidney function. (Healio)
A recent study shows fruits, vegetables & cooking classes may improve kidney outcomes in high-risk populations. (Healio)
UAMS performs first kidney-pancreas transplant in Arkansas. (Magnolia)
18 kidney donors hiked three volcanoes in Guatemala to show potential donors what’s possible with one kidney. (WMUR Manchester)
Another study shows why dialysis clinics must prepare for future record-breaking weather and climate disasters based on data from 60,000 patients between 2001 to 2019. (ASN Kidney News)
Healio shared its lists of top stories and themes from the past year, including home dialysis, COVID & kidney management.

A Closer Look

A closer look at a few headlines and why they matter:

1. A global team of researchers led by Dr. Katherine Tuttle believes they may have "broken the code" to fight kidney disease.

Make no mistake, this treatment is ‘on par with the biggest breakthroughs in medicine,' and can potentially help millions of people. An 11,000-person study will start in 2024, though the drug regimen is not expected to be used in clinical practice for another four years.

Why it matters: Dr. Tuttle and her team are testing the addition of a third drug, called an aldosterone synthase inhibitor (ASi), that will be used in combination with two other inhibitors, ACEs + SGL2s. Their recent Providence trial enrolled 714 patients and saw a "clinically meaningful reduction" in damage to the kidney in 70% of patients when ASI was combined with the two other drugs.

2. The FDA released draft guidance on Use of Real-World Evidence for medical devices.

Best summed up by Marty Culjat:

"The new guidance is excellent in that it clarifies and expands on potential use cases where RWD can be applicable in regulatory submissions (including primary evidence in new submissions, and supplemental evidence in support of OUS data!), and explains its concerns with a variety of topics, including relevance of the RWD to the target population, data quality and integrity, methodologies for collection and analysis of RWD, and documentation provided in regulatory submissions."

Why it matters: Digital Medicine is an exciting frontier and future, but it's had its share of setbacks (here, here, and here). This news gives companies much needed clarity at a time when clinical rigor is paramount not only for FDA submission, but for payer coverage and eventual adoption.

3. Penn Medicine just set the world record with 100 kidney paired donation transplants in a single year.

Kidney paired donation (KPD) was first suggested in 1986, but it was not until 2000 when the first paired donation transplant was performed in the US.

Here’s National Kidney Registry CEO Garet Hil on the significance of this milestone:

"This will be like Roger Bannister breaking the 4 minute mile barrier – Penn proved it is possible and now many transplant centers will break the 100 KPD transplant threshold over the next decade."

Why it matters: More than 90,000 people in the United States are waiting to receive a kidney transplant, with average waits to receive a kidney from a deceased donor stretching to between five and eight years.

Kidney Exchange: An Operations Perspective | Microeconomic Insights — Microeconomic Insights

4. Calliditas wins over FDA for full approval, broader label on kidney disease drug Tarpeyo.

Two years after securing accelerated approval, Calliditas Therapeutics just became the first fully-approved treatment in the US for IgAN, also known as Berger’s disease.

As Fierce Pharma points out, “while some graduations from accelerated to full approval are ceremonial, this endorsement carries extra weight as it expands the label for Tarpeyo, allowing it to be accessed by all patients with IgAN. Previously, the FDA limited the med's use to those at risk for rapid disease progression.”

Since this is the first drug approved for this indication, the Calliditas team, led by CEO Renee Aguiar-Lucander, will continue building out its US commercial strategy from scratch in parallel with ongoing efforts in Europe and China.

Why it matters: There are 130,000 to 150,000 people who have immunoglobulin A nephropathy (IgAN) in the U.S. Other key pharma players in this space include Travere (sparsentan) and Novartis (Chinook’s atrasentan).

Visual of the week

Pictures say a thousand words. What does this one tell you about the future of kidney health?

Empagliflozin RCT. This figure from The Lancet shows results from the Phase 2 trial by Dr. Tuttle and her colleagues mentioned above that may prove to be a major medical breakthrough in the treatment of CKD. An 11,000-person Phase 3 trial will start in 2024.

Here’s what Dr. Peter Rossing, Chief Physician and Head of Research at Steno Diabetes Center in Copenhagen, had to say about the study results:

“We are moving more and more towards the fact that in the future we will treat people with kidney disease as we treat patients with heart disease, by giving a number of drugs that protect the kidneys by lowering albuminuria levels."

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