Q&A with OPKO Health, Inc., Akhtar Ashfaq MD, FACP, FASN
OPKO Health is a fully-integrated healthcare company focused on delivering next-generation solutions for serious diseases across established global markets. Led by an accomplished management team whose drug development experience is unmatched in the industry and partnered with other leading pharmaceutical companies, OPKO Health is dedicated to advancing proven technologies to transform patients’ lives. The Renal Division of OPKO Health, Inc. focused on developing proprietary products to treat secondary hyperparathyroidism (SHPT) associated with chronic kidney disease (CKD). In addition, the Renal Division is developing novel therapies to treat elevated blood phosphorus levels(hyperphosphatemia) in order to improve the control of SHPT in CKD patients.
Tell us about your first experience with a kidney patient—personally and professionally—and what impressions that left on you as a fellow human being.
I didn’t have to look far to meet a kidney patient, one lives in my own home. One in seven adults in the U.S. has chronic kidney disease (CKD), and tragically, 90% of them don’t even know it until it’s far advanced.
I’ve witnessed close family members live through CKD’s gradual decline, progressing to kidney failure and ultimately dialysis. They never imagined their kidneys would fail, until they did. Now, they can’t stop wondering if there was something they could have done differently to prevent it. That experience became the driving force behind my decision to become a nephrologist.
I see them as fighters, real survivors. For anyone unfamiliar with life on dialysis, I wish they could spend just one day alongside these patients. Only then would they grasp the daily battle to stay alive, to be well, and to move forward despite overwhelming odds.
They are a symbol of resilience. Whether it’s rain, snow, hurricanes, or blazing sun, they show up, often with a smile. And they keep showing up. People who live with kidney failure, sometimes for years, waiting and hoping for a transplant? Their perseverance humbles and inspires me every single day.
Can you tell our AAKP national and global audiences why/how OPKO Health entered the kidney disease space, and about the company’s commitment to kidney patients and their families?
OPKO Health entered the kidney space in 2007 with a clear mission: to address secondary hyperparathyroidism (SHPT), one of the most serious and under-appreciated complications of CKD. At the time, available treatments either failed to overcome vitamin D hormone deficiency, particularly in patients with obesity or advanced CKD, or posed serious side effects due to their pharmacological approach to replacing vitamin D.
The OPKO Renal Division has a steadfast commitment to continue developing safe, effective therapies that address the needs of patients with CKD and their families.
Why did OPKO Renal develop Rayaldee, an FDA-approved treatment for SHPT in CKD stage 3 or 4 patients?
We recognized that metabolic bone disease is a silent but serious, and potentially life-threatening condition. Like high cholesterol, it’s often ignored until it causes real damage. This lack of urgency stems from several factors: absence of a defined PTH target in non-dialysis CKD, concerns about vitamin D toxicity at higher serum levels, fear of adynamic bone disease from low PTH, and risks of elevated calcium and phosphorus.
We started developing Rayaldee in 2007 to directly address these concerns. It raises serum 25- hydroxyvitamin D (25D) levels gradually and predictably, allowing physiological increases in 1,25-dihydroxyvitamin D and safe reductions in PTH. It does so without causing hypercalcemia, hyperphosphatemia, hypercalciuria, or increases in fibroblast growth factor 23 (FGF23).
How did Rayaldee fill a treatment gap for SHPT in CKD/non-dialysis patients?
Until Rayaldee’s FDA approval in June 2016, treatment options for SHPT in non-dialysis CKD patients were limited to:
• Dietary vitamin D supplements (D2 or D3), which require conversion in the liver and are often ineffective in advanced CKD or in patients with obesity and are not approved for treatment of SHPT in patients with CKD.
• Active vitamin D analogs (e.g., calcitriol, paricalcitol), which lower PTH but can’t raise 25D levels and often cause hypercalcemia, hyperphosphatemia, hypercalciuria, and elevates FGF23. Moreover, these active analogs may have been associated with faster kidney function decline.
Recognizing the specific issue of hypercalcemia, the applicable 2017 KDIGO CKD-MBD guideline advised against routine use of active vitamin D analogs in CKD stages 3-4, limiting their use to severe, progressive SHPT.
Rayaldee fills this gap by bypassing the need for hepatic conversion, providing stable and sustained increases in 25D, enabling extra-renal production of calcitriol, and reducing PTH without the side effects associated with older therapies. Clinical studies show that achieving serum 25D levels ≥50 ng/mL is essential for effective and sustained PTH suppression, something Rayaldee can accomplish consistently, even in overweight CKD patients when dietary vitamin D supplements (D2 or D3) are unable to do so.
How can patients determine if Rayaldee is the right treatment option for them?
We believe Rayaldee is not just an option, it’s a necessity for patients with CKD stages 3-4, SHPT, and vitamin D insufficiency. Unlike traditional D2 or D3 supplements, Rayaldee can reliably raise serum 25D to levels to ≥ 50 ng/mL, required for meaningful and sustained PTH reduction (≥30%). Without this, clinicians are often forced to prematurely introduce active vitamin D analogs, despite their known risks.
Since its approval, Rayaldee has been prescribed by more than 6,000 physicians across the U.S., Europe, and China, benefiting around 50,000 patients worldwide. We encourage patients to talk to their nephrologists or peers who have experience with Rayaldee. For more information and patient resources, visit Rayaldee.com.
Why is patient access and choice so critical in CKD care, especially with newer innovations?
CKD is complex and burdensome, not just for patients, but for families, healthcare systems, and society. Innovation in this space is not a luxury, it’s a necessity.
We can develop the best therapies in the world, but if they don’t reach patients, they’re meaningless. In today’s resource-constrained environment, patients and caregivers must advocate for access to the best available treatments. That begins with being informed about both existing and new therapies, and understanding that innovation arises only when there’s a need to improve upon current standards.
If we want to take ownership of our health, we must participate in our care. That means being educated, involved, and vocal, not just for ourselves, but for all those living with CKD.
As a healthcare leader, how do you stay driven and optimistic, especially during the most difficult moments in drug development?
Drug development is not for the faint of heart. It takes resilience, perseverance, and unwavering belief and commitment. Even when a therapy passes every clinical milestone, approval is never guaranteed. On average, only 1 out of every 5,000–10,000 compounds screened in the lab makes it to market, and only 10% of those entering human trials are approved.
Despite the odds, we press on, because innovation in kidney disease is not optional. The cost of inaction is simply too high. Mortality for dialysis patients remains stubbornly around 20% and hasn’t improved meaningfully in decades. A landmark 2018 paper by Foreman et al. ranked CKD 16th among causes of death in 2016 above, breast, colorectal and liver cancers. By 2040 morality from CKD will climb to #5 just below ischemic heart disease, stroke, lower respiratory infections and COPD.
This simply cannot be accepted, and this trajectory must change. That’s what motivates our team at OPKO every day. We’re driven by the chance to shift outcomes by developing improved therapies and ultimately give CKD patients and their families a better future.
Final question – AAKP patients say this last question reveals a lot about a person: Who is one of your heroes, and why?
For someone who has spent more than 30 years in nephrology, as a clinician, scientist, patient, and advocate, my answer is easy. My heroes are the patients with CKD and the families who care for them.
They are the reasons we do what we do. They show us what strength looks like. They endure, persevere, and keep moving forward. Their courage and resilience inspire us to keep searching for better therapies, so they can live longer, live better, and remain valued members of their families and communities. They are the true heroes in this journey.
Dr. Akhtar Ashfaq is an Internist and Nephrologist by training and is board certified in both internal medicine and nephrology.
During his career, Dr. Ashfaq has published in Major Nephrology Journals, has contributed to Internal Medicine and Nephrology text books; he has been a reviewer for most of the Nephrology journals.
In addition, Dr. Ashfaq has been on the board of trustees of the American Kidney Fund and is also an active member of the Medical Advisory Board of American Association of Kidney Patients.
His major area of interest is patient awareness and education and improving the life of patients with chronic kidney disease.
