Diseases & therapies
"I am sure we succeeded because we believed in the project and in ourselves, and because we come to work each day motivated to support our patients."
Senior Global Clinical Project Manager
Patients at risk of thrombosis are often treated with an anticoagulation agent such as warfarin. This can lead to an increased risk of bleeding when these patients require emergency surgery, which is typically managed by infusion of a four-factor prothrombin complex concentrate (4F-PCC) to restore the blood’s ability to clot.
At the request of the US Food and Drug Administration (FDA), Octapharma’s Clinical R&D team initiated the development of the LEX-209 clinical trial in 2016. The trial was designed to investigate the efficacy of the company’s 4F-PCC, compared to a similar product.
The study, Octapharma’s first head-to-head comparison, delivered strong results and was brought to an early conclusion in 2022, paving the way for the product to be offered to patients in the USA.
This success was not always guaranteed. From the beginning, recruitment of patients proved to be difficult as it is not easy to predict where or when in the world a patient treated on warfarin will need urgent surgery.
“We simply needed to engage a lot of hospitals and fish for patients,” says Dmitrii Matveev, VP and Head of Clinical R&D for Immunology & Critical Care. “In addition, it was also difficult to convince anyone to take part in such urgent settings.”
Romana Wesenauer, Director, Clinical Supply Chain, shares the same view. “My team was responsible for sourcing and planning for the LEX-209 study on a global level, and coordinated drug supplies for all study countries to ensure study sites were equipped at all times to be ready for emergency patients participating in the trial. Forecasting demand in a particular region was a challenge as emergency patients are hard to predict, but I’m proud to say we managed it successfully over the duration of the study.”
Looking back at the LEX-209 trial, Dmitrii recalls despairing moments when enrolment completely stalled. “Nobody had fully anticipated that it would be so difficult to enroll patients for this new study,” he admits. “This was a high-risk trial from the start. And during all these years, it has been a journey of persistence and strong belief.”
Nevertheless, the team persevered. “We have always been committed to providing the critical care community with the life-saving therapies they need,” Dmitrii explains. Each setback came with an insight that brought them one step closer to their goal.
Staying agile in a constantly changing and unpredictable environment was the only way to succeed for the LEX-209 team.
“Closing and reopening the study in several countries, dealing with the COVID-19 pandemic and the consequent lack of study site personnel, and the shutdown of logistic routes are just a few of the challenges we were faced with,” recalls Romana. “Close communication with all stakeholders was our key to running the study successfully and efficiently.”
By introducing local country champions, Dmitrii’s team was able to get closer to local physicians and practitioners. “At some point, we also switched to patients in Eastern Europe and did a lot of searching for patients ourselves at Octapharma,” says Dmitrii.
Furthermore, the team decided to take over some tasks performed by external vendors. “Monitoring of data and medical reviews were then done internally,” says Dmitrii. “It has been a lot of additional work, but it eventually turned out to be of great value.”
On September 21, 2021, Octapharma was finally able to submit the interim analysis of 185 patients and requested a pre-BLA meeting with the FDA to ask for the study to be concluded early. Five months later, on February 22, 2022, the FDA agreed to end the study based on the favourable interim analysis data. The BLA was submitted on July 28, 2022, and final FDA approval is now expected in July 2023.
“We are really happy about the positive outcome of the interim analysis and fully prepared to take on the challenges that lie ahead of us in the coming months,” says Doris Hinterberger, Senior Global Clinical Project Manager, who took the lead in preparing documents for FDA submission.
“I am truly inspired by my team who produced their best work at a time when we were facing the COVID-19 pandemic and all that that entailed,” says Doris. “I’m sure we succeeded because we believed in the project and in ourselves, and because we come to work each day motivated to support our patients.”
Octapharma’s Clinical R&D team initiated the development of the LEX-209 clinical trial.
Octapharma submitted the interim analysis of 185 patients and requested a pre-biologics licence application (pre‑BLA) meeting with the FDA to ask for the study to be concluded.
The FDA agreed to end the study based on the favourable interim analysis data.
The BLA was submitted.
Final FDA approval expected.
Diseases & therapies