PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company and leader in the field of biopolymer-based drugs, focused on developing treatments for metabolic and specialty cardiopulmonary disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vasomera (PB1046) Injection for the treatment of cardiomyopathy associated with dystrophinopathies: Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and X-linked dilated cardiomyopathy (XL-dCMP).
Dystrophinopathies are rare heterogeneous X-linked genetic disorders characterized by progressive muscle tissue degeneration caused by mutations in the dystrophin gene. As current treatment has focused on prevention of skeletal muscle loss and preservation of respiratory function, cardiomyopathy has emerged as a leading cause of death.
PhaseBio plans to initiate a Phase 2a study of PB1046 in the first quarter of 2016. The study will be conducted in two parts. Part One will be a double-blind, placebo-controlled, sequential dose escalation study to assess safety, tolerability, pharmacokinetics (PK) and explore pharmacodynamic (PD) response following four weeks of dosing with PB1046 in approximately 20 patients with stable New York Heart Association (NYHA) Class II/III heart failure with reduced ejection fraction. Two doses will be selected to move forward to Part Two, which will assess the safety, PK and explore PD response in approximately 20 patients with DMD or BMD who have evidence of cardiac dysfunction.
"Orphan drug designation is an important regulatory milestone for PB1046 and PhaseBio, and highlights the need for therapies to treat cardiomyopathy associated with dystrophinopathies, a life-threatening condition with no adequate therapy options," said Jonathan P. Mow, Chief Executive Officer of PhaseBio. "We are on track to advance PB1046 into the Phase 2a study early next year and look forward to continuing to work with the FDA to meet this critical patient need."
The Orphan Drug Designation program is intended to encourage companies to develop therapeutics for diseases that affect fewer than 200,000 individuals in the U.S. Orphan drug designation will provide PB1046 with seven years of marketing exclusivity if approved by the FDA for the treatment of dystrophic cardiomyopathy. Prior to FDA approval, orphan drug designation provides incentives for sponsors, including tax credits for clinical research expenses, the opportunity to obtain government grant funding to support clinical research and an exemption from FDA user fees.
PB1046 has also received orphan drug designation for the treatment of World Health Organization (WHO) group I Pulmonary Arterial Hypertension.
