August 31, 2020: “Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co., Ltd., based in Tokyo, Japan, announces that the US FDA has granted orphan drug designation for the company’s novel, orally administered non-peptidomimetic antagonist of the cellular and X-linked inhibitors of apoptosis proteins (cIAP1/2 and XIAP), tolinapant (formerly known as ASTX660), for the treatment of T-cell lymphoma.
Orphan drug designation is granted to drugs intended to treat a rare disease or condition.
In the US an orphan disease is defined as one affecting fewer than 200,000 people. The designation provides for seven years of marketing exclusivity in the US following product approval, as well as certain tax incentives and grants.
“T-cell lymphoma remains a challenging disease area with poor prognosis and high unmet medical need,” said Mohammad Azab, MD, Astex Pharmaceuticals’ president and chief medical officer.
“Astex’s development of tolinapant is aimed at bringing a new therapeutic option for patients with this type of cancer.
We are delighted that the FDA has granted the orphan drug designation which recognizes the continued need for development of new treatments for patients with rare diseases such as peripheral T-cell lymphoma and cutaneous T-cell lymphoma.”
ASTX660 is an investigational compound and is not currently approved in any country.
Tolinapant (ASTX660)
Tolinapant is a novel, orally administered, non-peptidomimetic antagonist of the cellular and X-linked inhibitors of apoptosis proteins (cIAP1/2 and XIAP).
Inhibitors of apoptosis proteins (IAPs) are frequently overexpressed in tumor cells and contribute to tumor cell survival and chemo-resistance.
By inhibiting IAPs, tolinapant promotes cell death. Tolinapant also acts via a newly described immunomodulatory mechanism which works to enhance an anti-tumor immune response in T-cell lymphomas.
Tolinapant was designed using Astex’s fragment-based drug design technology.”
https://astx.com/astex-pharmaceuticals-announces-that-tolinapant-astx660-a-novel-antagonist-of-cellular-and-x-linked-inhibitors-of-apoptosis-proteins-has-been-granted-orphan-drug-designation-for-the-treatment-of-t/
