FDA Grants Orphan Drug Designation to ABD-147 in Neuroendocrine Carcinoma

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• The FDA has granted orphan drug designation to ABD-147 for the treatment of neuroendocrine carcinoma.
• ABD-147 is a next-generation precision radiopharmaceutical biologic therapy.
• A first-in-human study will evaluate ABD-147 in patients with small cell lung cancer (SCLC) or large cell neuroendocrine carcinoma (LCNEC) who previously received platinum-based therapy.

ABD-147, a next-generation precision radiopharmaceutical, has been granted orphan drug designation by the FDA for the treatment of neuroendocrine carcinoma.¹

With this designation, Abdera Therapeutics, the sponsor, can be eligible for incentives like tax credits for clinical trials, exemption from user fees, and a potential 7 years of market exclusivity.

“Neuroendocrine carcinomas, including SCLC and LCNEC, are aggressive and challenging to treat effectively with current systemic therapies,” said Philippe Bishop, MD, chief medical officer of Abdera Therapeutics, in a press release. “By delivering a potent radioisotope to neuroendocrine tumors expressing DLL3 with custom-engineered [pharmacokinetic] properties, we believe ABD-147 has the potential to become a best-in-class DLL3-targeting treatment for aggressive neuroendocrine tumors.”

In July 2024, the FDA also granted fast track designation to ABD-147 in extensive-stage SCLC.² The fast track designation is intended to facilitate the development and review of drugs that treat serious conditions and fill unmet needs. The designation provides the sponsor with more frequent communication with the FDA as well as eligibility for accelerated approval and priority review.

This year, Abdera will begin a phase 1, first-in-human study evaluating ABD-147 in LCNEC and SCLC that has been previously treated with platinum-based therapy.¹

ABD-147 is designed to deliver actininum-225 to DLL3-expressing tumors. DLL3 is upregulated in some high-grade neuroendocrine carcinomas, including SCLC, while it is typically absent on the surface of nonmalignant cells, making it an attractive target for radiotherapy.

REFERENCES:

1. Abdera Therapeutics announces FDA orphan drug designation for ABD-147 for the treatment of neuroendocrine carcinoma. News release. Abdera Therapeutics. September 5, 2024. Accessed September 10, 2024. https://tinyurl.com/fccse5a7

2. Abdera Therapeutics announces FDA fast track designation for ABD-147, a next-generation precision radiopharmaceutical therapy, for the treatment of patients with extensive-stage small cell lung cancer. News release. Abdera Therapeutics. June 27, 2024. Accessed September 10, 2024. https://tinyurl.com/3k2y8ece