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FDA grants orphan drug designation to Yeliva for cholangiocarcinoma
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The FDA granted orphan drug designation to Yeliva for the treatment of cholangiocarcinoma, according to the drug’s manufacturer.
Yeliva (ABC294640, RedHill Biopharma) is a first-in-class, orally administered, sphingosine kinase-2 selective inhibitor with anticancer and anti-inflammatory activities.
The phase 1 study designed to evaluate Yeliva in three patients with advanced solid tumors met its primary and secondary endpoints. One patient achieved a sustained partial response (OS, 20.3 months) and two patients showed prolonged stable disease (OS, 17.6 months and 16.3 months).
Further, the study demonstrated the drug is well tolerated and can be safely administered to patients at doses that provide circulating drug levels that are predicted to have therapeutic activity.
“Cholangiocarcinoma is a cancer with a poor prognosis. Patients suffering from this disease have very few treatment options, and they are of limited efficacy,” Mark L. Levitt, MD, PhD, medical director of oncology at RedHill Biopharma, said in a company-issued release. “Based on promising preclinical data, as well as results from three previously treated cholangiocarcinoma patients who took part in the phase 1 study with Yeliva, we are hopeful that Yeliva could potentially provide a much-needed new treatment option for patients.”
Three additional trials of Yeliva are ongoing, including:
- a phase 2 study for the treatment of advanced hepatocellular carcinoma;
- a phase 1b/2 study for the treatment of refractory or relapsed multiple myeloma; and
- a phase 1/2 study for the treatment of refractory/relapsed diffuse large B-cell lymphoma and Kaposi sarcoma.
Further, this year the company plans to initiate a phase 1b study of Yeliva as a radioprotectant for the prevention of mucositis in patients with head and neck cancer undergoing therapeutic radiotherapy, as well as a phase 2 study of the drug to treat patients with moderate to severe ulcerative colitis.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.