Pharos iBbio said Tuesday that PHI-101, its next-generation indigenous acute myeloid leukemia drug, has received orphan drug designation from the European Medicines Agency (EMA).
The designation is noteworthy, as it officially recognizes the innovative drug's potential and promise in developing treatments for rare and incurable diseases in Europe and solidifies its position as a next-generation treatment for acute myeloid leukemia, the company explained.
The EMA grants orphan drug status to drug candidates expected to provide significant therapeutic benefits in rare diseases with a prevalence of less than 50 per 100,000 people in Europe, for which there are no existing treatments or an urgent need for new treatment options.
As a result of PHI-101's orphan drug designation in Europe, Pharos iBio will receive several benefits, such as a shorter review period for new drug applications, consultation on clinical trials, reduced marketing authorization application fees and other development costs, and market exclusivity for 10 years after approval.
Accordingly, Pharos iBio noted that PHI-101 is well positioned for rapid approval and commercialization in the European market while also providing favorable conditions for global expansion.
PHI-101 is an innovative anticancer drug candidate derived from Pharos iBio's self-developed AI drug discovery platform, Chemiverse, and is a next-generation AML treatment that targets various resistance mutations in the FLT3 protein. It represents a new treatment option for patients with relapsed or refractory AML. It demonstrated high therapeutic efficacy in a global phase 1 clinical trial and cardiotoxicity safety with AI-based clinical prediction in non-clinical studies.
Acute myeloid leukemia (AML), the target disease of PHI-101, is rare and incurable in need of new therapies due to high relapse rates and drug resistance limitations of existing approved therapies. AML patients with FLT3 mutations have about twice the survival rate of those without FLT3 mutations and are at increased risk of relapse, creating an urgent need for innovative therapies.
Earlier, in 2019, the U.S. Food and Drug Administration (FDA) designated PHI-101 an orphan drug, and the Ministry of Food and Drug Safety (MFDS) designated it a development-stage orphan drug last year.
PHI-101's orphan drug designation from the European Medicines Agency is a significant achievement that officially recognizes its innovation and potential in the global marketplace, and this designation has further increased interest in PHI-101 globally,” Pharos iBio CEO Yoon Jeong-hyeok said. “We will continue to accelerate our development to bring new treatment options to patients with relapsed or refractory acute myeloid leukemia who are limited by existing therapies as soon as possible.”