Calico Life Sciences Receives FDA Orphan Designation for ADPKD Treatment

Calico Life Sciences Granted Orphan Drug Designation for ABBV-CLS-628

Calico Life Sciences LLC, a pioneering biotechnology firm established by Alphabet Inc. and Arthur D. Levinson, announced on November 5, 2025, that its investigational treatment, ABBV-CLS-628, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). This designation underlines the therapy's potential to address the pressing needs of the ADPKD community.

ABBV-CLS-628 is an anti-PAPP-A monoclonal antibody that has been developed in collaboration with AbbVie Inc. Alongside its recent Fast Track Designation from the FDA, this new status emphasizes the urgent treatment gap faced by individuals living with ADPKD, a condition that significantly deteriorates kidney function over time, often leading to dialysis or transplant by the age of 60 for over half of its patients.

Arthur D. Levinson, CEO of Calico, expressed his enthusiasm about the ODD, stating, “We are pleased that ABBV-CLS-628 has received both Fast Track and Orphan Drug designations, underscoring the urgent unmet need facing the ADPKD community. People living with this disease face a high risk of rapid disease progression, and we will continue to advance this program with the goal of improving the treatment trajectory for patients.”

Understanding ADPKD and Treatment Goals

ADPKD stands as the most prevalent inherited kidney disease globally, where cysts filled with fluid develop in both kidneys, causing significant renal complications. The progressive nature of the disease means that individuals can experience rapid decline in kidney function, making the development of effective therapies critical.

The Orphan Drug Act, established by Congress in 1983, incentivizes the development of treatments for rare diseases affecting fewer than 200,000 individuals in the U.S. By granting the ODD, the FDA signifies that ABBV-CLS-628 shows promise in treating ADPKD, which qualifies it for benefits such as tax credits for related clinical trials, protocol assistance, and an extended market exclusivity period post-approval.

The Clinical Journey of ABBV-CLS-628

Research surrounding ABBV-CLS-628 has been substantial. A Phase 1 safety study involving healthy volunteers demonstrated that the drug was safe and well-tolerated, with no significant adverse events reported. Currently, the therapy is progressing through a Phase 2 clinical trial (NCT06902558) that is set to enroll patients across about 95 sites globally. Participants will receive treatments intravenously every four weeks over a 92-week period, with safety follow-up planned for up to 15 weeks. This trial aims to assess both the safety and effectiveness of ABBV-CLS-628 in slowing the progression of ADPKD.

Calico Life Sciences: Pioneering Age-Related Research

Calico, based in San Francisco, focuses on advancing our understanding of the biology behind human aging and age-related diseases. The company aims to utilize groundbreaking technologies and model systems to devise innovative interventions that allow individuals to enjoy longer, healthier lives. For more information about Calico and its research initiatives, visit Calico's website and follow @calico on LinkedIn, X, and YouTube.

As we stand at the forefront of medical research and development, the groundwork laid by companies like Calico not only helps to bridge treatment gaps for conditions such as ADPKD but also inspires hope for many suffering from rare diseases worldwide. With ABBV-CLS-628, the future looks promising as clinical trials continue to explore its full potential.