kidney disease Archives - BioPharma Global

Regulus Therapeutics Announces Receipt of FDA Orphan Drug Designation for RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

July 20, 2022

SAN DIEGO, June 21, 2022 /PRNewswire/ — Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the “Company” or “Regulus”), today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD).

“Patients living with ADPKD currently have limited treatment options and approximately half of patients develop end-stage renal disease by age 60 requiring dialysis or transplantation,” commented Jay Hagan, CEO of Regulus Therapeutics. “ADPKD is a disease of high unmet need affecting nearly 160,000 Americans. We look forward to advancing RGLS8429 through the clinic with the goal of improving future treatment options for patients in need.”

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan designation status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

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BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

FDA grants Calliditas Therapeutics Accelerated Approval of TARPEYO™ to Reduce Proteinuria in IgA Nephropathy

December 17, 2021

by administrator BPG Blog

Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) (“Calliditas”) today announced that the US Food and Drug Administration (FDA) has approved TARPEYO (budesonide) delayed release capsules to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5g/g. This indication is approved under accelerated approval. It has not been established whether TARPEYO slows kidney function decline in patients with IgAN. Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial.¹

This approval marks the successful transition for Calliditas to a commercial-stage biopharmaceutical company.

“We are very excited to bring the first and only FDA-approved treatment to reduce proteinuria in IgAN to market,” said Renée Aguiar-Lucander, Chief Executive Officer of Calliditas. “TARPEYO represents an FDA approved product to help these patients who are at risk of rapid disease progression.”

TARPEYO is approved under accelerated approval based on achieving its primary endpoint of reduction in proteinuria in Part A of the NeflgArd pivotal Phase 3 study, an ongoing, randomized, double-blind, placebo-controlled, multicenter study conducted to evaluate the efficacy and safety of TARPEYO 16 mg once daily vs placebo in adult patients with primary IgAN.¹ The effect of TARPEYO was assessed in patients with biopsy-proven IgAN, eGFR ≥35 mL/min/1.73 m², and proteinuria (defined as either ≥1 g/day or UPCR ≥0.8 g/g) who were on a stable dose of maximally-tolerated RAS inhibitor therapy.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Rare Disease Highlight: Alport Syndrome

December 16, 2021

by administrator Rare Disease Highlight

Alport syndrome (AS) is a rare disease caused by mutations passed down from parents to their children ¹. It is estimated 30,000-60,000 individuals are affected by AS in the United States ². The mutations target a specific type of collagen in the kidneys, ears, and eyes, which impair its normal function of structural support ^1,3. As is primarily characterized by a continuous decline in kidney function, which usually starts with blood in the urine that is only visible using a microscope. The decrease in kidney function is related to the amount of damage in the kidneys and may eventually progress to kidney failure and end-stage kidney disease (ESKD) ¹. Depending on the severity of the disease, patients may also lose hearing in both ears and/or develop eye abnormalities ³. An early diagnosis of AS is crucial due to the lifetime risk of ESKD, and although a standard process is not established to diagnose AS, observing a positive family history of AS, persistent blood in the urine, hearing loss, and/or eye abnormalities should be enough for the physician to consider the possibility of AS ^4,5. Angiotensin-converting enzyme II inhibitors (ACE-I) are the current standard-of-care (SOC), but only delay the decline in kidney function, so many patients will inevitably progress to ESKD ^1,6. Dialysis or kidney transplant are other options for AS patients, but the patient survival is not improved ⁷. Thus, based on the gaps in the SOC, a disease-modifying therapy for individuals with AS remain a significant unmet medical need.

References used

1. Torra R, Furlano M. New therapeutic options for Alport syndrome. Nephrol Dial Transplant. 2019;34(8):1272-1279.

2. NORD. Alport Syndrome. NORD. https://rarediseases.org/rare-diseases/alport-syndrome/. Published 2020. Updated 2020. Accessed 06/16, 2020.

3. Hudson BG, Tryggvason K, Sundaramoorthy M, Neilson EG. Alport’s syndrome, Goodpasture’s syndrome, and type IV collagen. N Engl J Med. 2003;348(25):2543-2556.

4. Kashtan CE. Alport Syndrome. In: Adam MP, Ardinger HH, Pagon RA, et al., eds. GeneReviews(®). Seattle (WA)2019.

5. Kashtan CE. Alport Syndrome: Achieving Early Diagnosis and Treatment. Am J Kidney Dis. 2021;77(2):272-279.

6. Gross O, Licht C, Anders HJ, et al. Early angiotensin-converting enzyme inhibition in Alport syndrome delays renal failure and improves life expectancy. Kidney Int. 2012;81(5):494-501.

7. Temme J, Kramer A, Jager KJ, et al. Outcomes of male patients with Alport syndrome undergoing renal replacement therapy. Clin J Am Soc Nephrol. 2012;7(12):1969-1976.