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Pila Pharma applies for Orphan Drug Designation in the US

The company has submitted a request for an Orphan Drug Designation to the U.S. Food and Drug Administration (FDA) for the company’s development candidate XEN-D0501.

The aim is to develop a novel treatment of a rare disease associated with pain due to neurogenic inflammation.

“I’m really pleased, that we can now publically share what we have been working on “under cover” for the last months! This submission marks a new phase in Pila Pharma’s history with the active exploration of alternative indications in which XEN-D0501 or its analogues can be useful. We have had our eyes on several other indications for a while, including this specific rare disease, but it is only now that we have the expertise, resources and collaboration agreements that allow us to start new projects. We have previously said that XEN-D0501 has the potential to be useful as a treatment for other diseases where neurogenic inflammation plays a role, and we will continue on that path to eventually develop a pipeline with a focus on TRP as a platform technology,” says Pila Pharma CEO Dorte X. Gram.



Pila Pharma’s development candidate XEN-D0501 is under development for the treatment of type 2 diabetes. Preparations are ongoing according to plan for future clinical trials in phase 2b. The company’s drug candidate, XEN-D0501, inhibits the activity of the body’s TRPV1 receptors. TRPV1 has several functions in the body and among other things, the discovery of its role in pain has been awarded the 2021 Nobel Prize in Medicine.

Read more: Pila Pharma’s leading principle awarded with a Nobel Prize

Clinical phase 2/3 studies

Provided that the Orphan Drug Designation is designated by the FDA, Pila Pharma intends to seek funding to initiate clinical phase 2/3 studies to test the safety and efficacy of XEN-D0501 in the rare disease with the aim of registering a product with orphan drug status.

An approval of the request for Orphan Drug Designation would mean that Pila Pharma would receive market exclusivity including other benefits for XEN-D0501 for the treatment of that specific disease in the USA for seven years following registration.

Photo of Dorte X. Gram: Peter Brinch