Benzinga | Sep 9, 2021 07:24AM EDT

Freeline Announces Orphan Drug Designations for FLT201 for the Treatment of Gaucher Disease

Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage biotechnology company developing transformative AAV-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that both the U.S. Food and Drug Administration ("FDA") and European Commission ("EC") have granted Orphan Drug Designation ("ODD") for FLT201 for the treatment of Gaucher disease.

"Effectively advancing our core programs is priority one in our refreshed strategic plan," said Michael Parini, Chief Executive Officer of Freeline. "Receiving orphan drug designation for FLT201 is an important regulatory milestone in delivering on the promise of our Gaucher program to address the urgent, unmet need of patients living with Gaucher disease Type 1."

"Our robust preclinical data suggest that the proprietary engineered, more stable GCase variant in FLT201 has the potential to deliver sustained levels of ?-glucocerebrosidase and penetrate target tissues in Gaucher disease. We believe our approach may offer significant improvements in clinical outcomes over theexisting standard of care, particularly in terms of GCase uptake into difficult-to-reach tissues," said Mr. Parini.

"The Company remains on track to initiate our Phase 1/2 dose-finding trial in Gaucher disease Type 1 before year end," continued Mr. Parini. "We are enthusiastic about advancing this program, and we plan to share clinical data from the dose-finding trial at the right time in 2022."

Orphan Drug Designation in the United States is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 individuals in the United States. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax credits, FDA user-fee benefits and seven years of market exclusivity in the U.S. following marketing approval.

Orphan Drug Designation in the European Union ("EU") is granted by the European Commission based on a positive opinion issued by the European Medicines Agency ("EMA") Committee for Orphan Medicinal Products ("COMP"). It is assigned to medicines intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating and affects fewer than five in 10,000 people in the EU. The designation could allow regulatory and financial incentives for companies to develop and market therapies, including market exclusivity, protocol assistance and fee reductions.