FDA Grants Orphan Drug Status to BEA-17 for Glioblastoma Treatment


After more than 15 years with no new treatments for glioblastoma, a novel LSD1 small molecule has been granted orphan drug designation by the FDA.

The FDA has granted orphan drug designation to BEA-17 for the treatment of patients with glioblastoma (GBM).1

BEA-17, a first-in-class LSD1 inhibitor, is designed to address LSD1 overexpression found in GBM. To date, there are no FDA-approved therapies that can target LSD1, and BEA-17 is not approved in any country.

In preclinical models, BEA-17 demonstrated in vivo potentiation of immune-modulating agents in multiple cancer types, including the standard-of-care combination of temozolomide (Temodar) and radiation in GBM models. In pharmacokinetic analyses, BEA-17 showed that ability to penetrate the blood-brain barrier as well as good oral availability. In-human studies have not been conducted yet.

The development of BEA-17 is the product of a grant awarded by SweLife and Medtech4Health in 2019 and was based on the lack of new therapies for GBM over a 15-year period.2 At the time the award was granted, Per Källblad, chief executive officer of Beactica Therapeutics AB, made a statement about the grant in a press release. “We are delighted about the recognition that this important project continues to receive. It is clear that BEA-17 is a promising compound on its own, but a combination therapy may create the synergy needed to maximize the clinical outcome in glioblastoma.”

With an orphan drug designation, the developers of BEA-17 will be eligible for federal grants, and tax credits that will help them to conduct qualified clinical trials. The company will also be eligible for prescription drug user fee exemptions, and a 7-year marketing exclusivity, given BEA-17 is later granted approval by the FDA.1

"The FDA's decision to grant orphan drug status to BEA-17 marks an important milestone for the program and highlights the significant need for novel therapies to treat these devastating brain tumors." said Källblad, in a separate press release. "The designation will facilitate our development of this agent which we believe has the potential to serve as a much-needed therapeutic option for patients affected by glioblastoma."


1. Beactica Therapeutics receives FDA Orphan Drug Designation for BEA-17 for the treatment of glioblastoma. News release. Beactica Therapeutics. February 1, 2023. Accessed February 1, 2023. https://bit.ly/3XWfGWI

2. Beactica wins grant to develop combination therapy for glioblastoma. News release. Beactica Therapeutics. June 18, 2019. Accessed February 1, 2023. https://bit.ly/3l1D0E9

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