after being fast tracked ABD-147 was designed orphan drug for the Treatment of Neuroendocrine Carcinoma (prostate included)

[Maxone73]

DLL3 is expressed by neuroendocrine cancers, including prostate cancer! They plan to start a phase 1 trial by the end of 2024 (on lung cancer, but it’s worth to keep an eye on it).

“ABD-147 is a next-generation precision radiopharmaceutical biologic therapy designed to deliver Actinium-225 (225Ac) to solid tumors expressing DLL3, a protein found on the surface of neuroendocrine tumors, but rarely expressed on the surface of normal cells or tissues.”

businesswire.com/news/home/...

[NickJoy]

Very good news. Thank you.

[TeleGuy]

I sure wish I understood what is “orphan” about this new drug. I have NE differentiation but strangely no DLL3 expression.

[Maxone73 in reply to TeleGuy]

here you go!

"The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including:

- Tax credits for qualified clinical trials

- Exemption from user fees

- Potential seven years of market exclusivity after approval"

which means that they will be helped in carrying on with the development part. Since NE tumors are rare, without support, there is a risk that no company would invest in developing treatments for such diseases.

[TeleGuy in reply to Maxone73]

Yes, I read that in the press release, but I thought that orphan drugs were old generic drugs that someone discovered were useful for something new.