FDA Grants Orphan Drug Designation to Cellenkos' CK0804 Treg Therapy for Treatment of Myelofibrosis

Treg therapeutic designed to preferentially traffic towards its ligand CXCL12 over expressed in the bone marrow and spleen in myelofibrosis. -- CK0804 engages with antigen presenting cells within ...
Targeted Oncology

FDA Grants Orphan Drug Designation to CK0804 in Myelofibrosis

CK0804, a novel Treg therapy, gains FDA orphan drug status for myelofibrosis, promising new hope for patients with limited treatment options. CK0804, an investigational allogeneic regulatory T cell ...

Syndax and World Orphan Drug Alliance to Launch a Multi-Regional Managed Access Program, Expanding Access to Revuforj® (revumenib) Outside the U.S.

Program will expand access in certain regions where Revuforj is not available commercially, as permitted by local regulations -- Program will ...

FDA and EMA Grant Orphan Drug Designation for Curadel’s CPI-008, a Targeted Zwitterionic Imaging Drug for Pancreatic Cancer

Curadel's novel targeted zwitterionic surgical imaging drug for pancreatic cancer has been granted Orphan Drug Designation by the FDA and the EMA.

Swedish Orphan Biovitrum AB: Sobi to advance Gamifant (emapalumab) in interferon-gamma-driven sepsis (IDS) based on EMBRACE topline data

Sobi (STO: SOBI) today announced that topline results from the Phase 2a EMBRACE study evaluating Gamifant (emapalumab) for interferon-gamma (IFN?)-driven ...
Dhar Mann on MSN

1,000 lives saved by an orphan teen hacker?

After being abandoned by his parents and surviving the foster system, a brilliant teen hacker is lured into cybercrime by a ...
pharmaphorum

Orphan drugs' financial success raises questions

R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. However, Ben Hargreaves finds that the ...
pharmaphorum

Orphan medicines launch excellence: sustaining launch success

Launching orphan medicines excellently will become even more important over the next five years as more patients with rare diseases have pharmacotherapies available. There are also a growing number of ...
Nebraska TV

Minden cook-off aims to raise funds

The Zambian Mission Project Chili & Cinnamon Roll Cook-Off is set to take place on Sunday, Feb. 2, 2026, from 11:30 a.m. to 2 ...

SN BioScience received US FDA Orphan Drug Designation for its Nano Anti-Cancer Drug 'SNB-101' on Gastric Cancer.

SNB-101 previously received ODDs from the US FDA for small cell lung cancer in July 2023 and pancreatic cancer in February 2024. By receiving an ODD for gastric cancer this time, SN Bioscience expects ...

Curadel Pharma: FDA and EMA Grant Orphan Drug Designation for Curadel's CPI-008, a Targeted Zwitterionic Imaging Drug for Pancreatic Cancer

US and EU regulatory designations provide critical development resources to advance novel tumor detection technology Curadel Pharma, a pioneer in zwitterionicity and innovator in advanced radiotherapi ...

FibroGen Rebrands as Kyntra Bio to Reflect a New Era of Focus and Momentum

Name change to Kyntra Bio reflects the newly sharpened focus of the Company on novel therapies with potential for outsized impact in oncology and ...