Rare diseases are, by their nature, rare—and because of this, it can be difficult to spur drug development. The FDA worked to overcome this through the creation of the…
According to an article from Cancer Network, the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug designation to second-line GNS561 (ezurpimtrostrat) for cholangiocarcinoma (bile duct cancer). In…
In Europe, Orphan Drug designation is granted to therapies intended to treat, prevent, or diagnose a rare, life-threatening, or debilitating disease. For the purpose of this status, rare conditions…
Nelson Ambrogio had always looked up to his older brother, Daniel. He has many positive memories of growing up with him. They both shared a passion for soccer, and Nelson…
In late February 2022, biopharmaceutical company Trascenta Holding Limited ("Trascenta") provided an update on its clinical program for TST001, a monoclonal antibody therapy designed for those with locally advanced or…
According to a recent article, researchers have found that adding durvalumab to chemotherapy treatments results in prolonged survival rates in patients with advanced biliary tract cancer. Biliary Tract Cancer Biliary…
Funding is a problem that many rare diseases face when it comes to research. Cholangiocarcinoma, also known as bile duct cancer, is no exception. Because of this, it is often…
The FDA has recently approved a new diagnostic tool to help determine which IDH1-mutated cholangiocarcinoma patients may be a good fit for the novel IDH1 inhibitor called ivosidenib. The new…
According to a recent article at Pharmacy Times, Infigratinib has been granted accelerated approval for the treatment of those with metastatic cholangiocarcinoma. The treatment is for adults whose cholangiocarcinoma is…
Updates from the Phase 3 clinical trial called FIGHT-302 were just released at the 2021 European Society for Medical Oncology World Congress on Gastrointestinal Cancer. The trial is comparing two…
A survey of oncologists has found that more education is clearly needed regarding cholangiocarcinoma. Specifically, more education would be beneficial regarding testing and targeting the mutations which cause the condition…
According to MPR, the FDA recently granted Orphan Drug designation to CPI-613 (devimistat), an investigational treatment designed for patients with biliary cancer. Altogether, "biliary cancer" can consist of bile…
As reported in Fierce Pharma; BridgeBio has earned FDA approval for their new targeted cancer treatment, Truseltiq. The approval is the biotech company’s first ever approval for a cancer drug,…
Patient Worthy was a media partner at this year's NORD Living Rare, Living Stronger Patient and Family Forum. This is an annual event hosted by the National Organization for Rare…
In late May 2021, the FDA granted accelerated approval to Truseltiq (infigratinib). As reported by Cure, this therapy is designed for adult patients with unresectable locally advanced, previously treated, or…
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