The US Food and Drug Administration (FDA) has approved orphan drug designation for PT001, an investigational treatment from PulmoSim for pulmonary arterial hypertension (PAH) which targets several responsible pathways in an aim to provide a more effective treatment.
Orphan drug designation is used by the FDA’s Office of Orphan Products Development (OOPD) to support the development of treatments for disorders affecting fewer than 200,000 people in the US. Orphan drug designation status will provide eligibility for 7 years of US marketing exclusivity for PulmoSim if PT001 is approved. Other benefits include the waiver of Prescription Drug User Fee Act (PDUFA) filing fees and tax credits in clinical research expenditures along with regulatory and strategic assistance.
Jo Varshney, DVM, PhD, chief executive officer and founder of PulmoSim, said, “we’re thrilled that the FDA has granted this designation for PT001 and look forward to advancing this drug candidate into a clinical trial program very soon.”
PAH is a progressive and fatal disorder affecting 15-50 persons per million within the US and Europe. The average life expectancy after diagnosis is less than 3 years and all currently approved treatments are incapable of targeting the core condition, thus making it a “rare disease epidemic,” according to a statement from PulmoSIM
“The preclinical results in this indication have been very encouraging,” said Vivek Gupta, PhD, scientific founder at PulmoSIM. “The development of this new therapy will directly improve the standard of care for PAH patients by reducing morbidity and enhancing the quality of life at a reasonable cost.”
A proof-of-concept clinical efficacy trial is currently in progress for PT001.
PulmoSIM Therapeutics is the pharmaceutical subsidiary of VeriSIM Life, Inc., focused on “developing breakthrough therapies for rare and progressive respiratory diseases to improve patient outcomes and quality of life,” the company’s statement said.
PulmoSim Therapeutics granted orphan drug designation for PT001 by the FDA to treat pulmonary arterial hypertension. News release. PulmoSim Therapeutics; June 15, 2021.