The U.S. Food and Drug Administration has granted an orphan drug designation to Emtora Biosciences for its core therapeutic in treating familial adenomatous polyposis, or FAP.
The federal recognition is a significant milestone in the San Antonio-based biotech’s bid to expedite commercialization of its initial drug, eRapa, for use against the rare cancerous disease.
In February, Emtora Biosciences changed its name — it was Rapamycin Holdings Inc. — and new CEO Carole Spangler Vaughn told me one of her first priorities was to shift the company’s market strategy by seeking the orphan drug designation.
“There is no drug for this. It’s a hideous disease, but it’s a perfect disease for us for a number of reasons,” she said. “That opens a lot of doors with faster approval. There are fees, which are waved. There are tax incentives and market exclusivity for the active ingredient. It’s a great way to get in.”
Emtora plans to begin a phase 2a clinical trial in patients with FAP in late 2019. That will follow a phase 1b safety and dose escalation study in early-stage prostate cancer patients that will conclude this summer.
“FAP is a devastating disease for which no current therapeutic options exist, and eRapa has the potential to become a life-changing therapy for patients with this disease,” Spangler Vaughn said.
The timing of the designation is critical for other reasons, too. It comes after Emtora recently closed on a $2 million convertible note and as the company prepares to launch a series B funding round.
The FDA's Office of Orphan Drug Products grants orphan status to advance the development of therapies for treating rare diseases or conditions affecting fewer than 200,000 people in the U.S.