Ferring launches bladder cancer gene therapy Adstiladrin in US through early experience program

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Ferring, Adstiladrin, Gene therapy, bladder cancer, FDA, early experience program

Ferring Pharmaceuticals achieved a significant milestone by administering its groundbreaking gene therapy, Adstiladrin, to its first commercial patient. This innovative treatment offers hope to certain bladder cancer patients, potentially sparing them from the necessity of bladder removal.

The inaugural recipient of Adstiladrin is a resilient 78-year-old gentleman from the American Midwest. His treatment is part of Ferring’s early experience program, a compassionate initiative aimed at ensuring patients have access to this life-changing drug while the company scales up its manufacturing capabilities.

Developing Adstiladrin has been no small feat, as it relies on a complex adenovirus vector-based delivery system. Dr. Elizabeth Garner, Chief Scientific Officer at Ferring Pharmaceuticals, emphasized the company’s commitment to responsible distribution during this crucial manufacturing expansion.

The first patient dosed with Adstiladrin marks an incredible milestone for Ferring, the bladder cancer community and the patients we aim to serve. Making this novel and efficacious intravesical gene therapy commercially available for patients underscores our commitment to changing the trajectory of NMIBC. We will continue bringing available doses of Adstiladrin forward responsibly as we scale up manufacturing.”

– Shetal Vyas, Vice President, General Manager, Uro-Oncology at Ferring Pharmaceuticals

To meet the increasing demand for Adstiladrin, Ferring has strategically established two new production sites, one in Finland and another in New Jersey. By 2025, manufacturing is expected to reach full capacity, paving the way for Ferring to receive substantial milestone payments of up to $200 million from its partnership with Royalty Pharma, which has an overall deal value of $500 million.

Currently, the early experience program is accessible to investigators who conducted the phase 3 trial of Adstiladrin. Phase 3 will approximately enroll 800 patients in the ABLE-41 study. The program will gradually welcome more clinics as manufacturing output escalates, fostering accessibility for those in need.

One notable aspect of the program is its commitment to ensuring that patients who receive their first dose during the initial phase can complete their treatment regimen, which spans every three months over a minimum of one year, despite potential supply constraints. The first patient to undergo treatment has already scheduled his next appointment for December.

“Adstiladrin represents a major advancement in the current treatment landscape for people living with high-risk NMIBC who may be facing removal of the bladder. The ADSTILADRIN Early Experience Program and ABLE-41 US RWE Study allow us to address pressing patient needs while collecting data on its use in a real-world setting, further expanding on what we’ve learned in our Phase 3 clinical program.”

– Elizabeth Garner, MD, MPH, Chief Scientific Officer at Ferring Pharmaceuticals

Adstiladrin gained approval in December of the previous year for the treatment of high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer, either with or without papillary tumors. It represents a groundbreaking gene therapy, uniquely tailored for patients who no longer respond to standard therapies. Adstiladrin is directly administered into the bladder via a catheter, providing a targeted approach to treatment.

Notably, the recently treated patient has also been enrolled in Ferring’s real-world evidence study, known as ABLE-41. This study is designed to comprehensively evaluate the therapy’s effectiveness by assessing various factors such as durability, survival rates, and complete response rates. Additionally, it seeks to understand the experiences of patients, caregivers, and physicians through a series of questionnaires. This holistic approach will provide valuable insights into the impact of Adstiladrin on the quality of life for patients and their caregivers.

The ABLE-41 study is expected to span up to two years, allowing Ferring Pharmaceuticals to gain a deeper understanding of the real-world impact of Adstiladrin on the lives of those it serves. This groundbreaking gene therapy represents a beacon of hope for patients battling bladder cancer, and Ferring Pharmaceuticals is committed to making it accessible to all who can benefit from it.

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