FDA Gives PulmoSIM’s PT001 Orphan Drug Designation for PAH

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PT001, an investigational treatment for pulmonary arterial hypertension (PAH).

The FDA gives this designation to therapies with the potential to treat rare disorders, defined as those that affect fewer than 200,000 people in the U.S. The designation gives PT001’s developer, PulmoSIM Therapeutics, access to certain waived fees, tax credits in clinical research expenses, regulatory assistance, as well as the potential for seven years of marketing exclusivity if the therapy is approved.

“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,” Jo Varshney, PhD, CEO and founder of PulmoSIM, said in a press release.

PulmoSIM, which launched earlier this year, is the pharmaceutical subsidiary of VeriSIM Life. The parent company also owns BIOiSIM, a platform that uses artificial intelligence to compute simulations of human biology to predict which compounds will most likely benefit people with diseases like PAH.

The use of BIOiSIM will allow PulmoSIM to fast-track the development of therapies for rare diseases, according to Varshney.

PT001 “targets multiple responsible pathways in PAH” and is designed to more effectively alter the underlying biology that drives the disease, according to PulmoSIM.

“There still remains a high unmet need for more effective and less-burdensome treatment options for patients living with PAH,” Varshney said.

Vivek Gupta, PhD, scientific founder at PulmoSIM, said that the results of preclinical testing of PT001 in models of PAH “have been very encouraging.”

“There is an urgent need for new therapies that have the potential to address the underlying cause of PAH, and we are committed to advancing PT001 as a novel approach to treating this debilitating disease … 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,” Gupta said.

PulmoSIM is continuing to work with its partners and advisors to move PT001 into proof-of-concept clinical trials to test the medication’s efficacy.

A Conversation With Rare Disease Advocates