Lundabaserade Ablivas huvudkandidat, KL1333, genomgår en global, randomiserad, placebokontrollerad och potentiellt registreringsgrundade fas II-studie, FALCON. Studien utvärderar säkerheten och effekten av kandidaten hos patienter med primär mitokondriell sjukdom, ett tillstånd för vilket det inte finns några tillgängliga behandlingar.

Tidigare i år doserades den första patienten i studien. Studien kommer initialt att omfatta 40 patienter från sex länder. Interimsresultat väntas runt halvårsskiftet 2024. Läs mer.

Fast Track-status för KL1333

Amerikanska Food and Drug Administration (FDA) beviljade nyligen KL1333 Fast Track–beteckning, vilket understryker behovet av nya och effektiva behandlingar för denna patientgrupp.

Detta innebär att Abliva kommer att kunna interagera oftare med tillsynsmyndigheter. Möten, skriftlig kommunikationoch kontinuerlig återkoppling på Ablivas New Drug Application (NDA) skulle underlätta den regulatoriska processen.

Läs mer om Fast Track och andra regulatoriska beteckningar som syftar till att effektivisera läkemedelsutvecklingen i vår artikelserie om läkemedelsutveckling.

Vd kommenterar

BioStock kontaktade Ablivas vd Ellen K. Donnelly för att få veta mer om nyheten.

What is Fast Track designation?

– Fast track designation is a tool offered by the FDA to companies who are developing a medicine for a serious condition which addresses a high unmet medical need.  A company needs to apply for the designation and the FDA reviews the applications to determine if the program qualifies for the designation.

– Their designation is an important external confirmation of our business strategy and target product profile for our lead compound. The regulator of the world’s most valuable pharmaceutical market has confirmed that our target population do suffer from a serious condition which is key component of future pricing possibilities. FDA, having access to all our study data, has also confirmed that our results so far demonstrate that our product can address a high unmet medical need.

What does this designation mean for Abliva?

– Fast track designation will, in short, facilitate our interactions with the FDA. The designation allows us to request more frequent meetings with the FDA to discuss our program, our study and our path to the market. This is important as it will allow us to gain clarity quickly on any questions that arise, or simply ensure we have the necessary data in the package that will be reviewed for marketing approval. The designation also allows for rolling review of our NDA submission, which means that it can be reviewed in pieces versus waiting until the entire package is completed. All of these benefits are designed to shorten the time to drug approval and the waiting patients. 

Could you describe how this will impact the company’s regulatory process?

– I do not expect that the designation will impact our regulatory processes, as Abliva has always had a strategy of meeting with the regulators ‘early and often’ and we have a fabulous team of regulatory experts assisting us with our regulatory strategy. We do, however, hope that the ability to request additional meetings and the potential for rolling review of the NDA will shorten our time to market, addressing the high unmet medical need in primary mitochondrial disease. 

Interim results are expected by mid-2024. What are you focused on now?

– The team is currently focused on recruiting the patient cohort that will contribute six month data to the interim analysis. We have a fantastic group of sites involved in the study across six countries, and they are all working aggressively to identify patients who have the profile required to participate in the study. Recruiting rare disease patients is always challenging, and I am thrilled that we are on track with our recruitment and the timeline to the interim analysis.