Ideaya Biosciences yesterday announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Darovasertib, a potential first-in-class Protein Kinase C (PKC) inhibitor, for the treatment of uveal melanoma.
In a statement, Ideaya said that it is currently evaluating the synthetic lethal combination of darovasertib, a PKC inhibitor, and crizotinib, a cMET inhibitor, in patients with Metastatic Uveal Melanoma (MUM), and in patients with GNAQ or GNA11 mutant solid tumours, in an ongoing phase-I/II clinical trial (NCT03947385) pursuant to a clinical trial collaboration and supply agreement with Pfizer.
ODD is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the US. Under the ODD, Ideaya may be entitled to certain tax credits, exemption from user fees, and seven years of statutory marketing exclusivity, subject to FDA approval of a marketing application for Darovasertib as a designated orphan-drug product, the statement added.
As of 1st May, 2022, Darovasertib has been evaluated in more tha 200 patients, including 74 patients in combination with Crizotinib. The company is targetting a clinical data update for Darovasertib and Crizotinib combination in mid-2022, including tolerability and clinical efficacy. Ideaya is also planning to seek FDA regulatory guidance for a potential registration-enabling trial design to evaluate the Darovasertib and Crizotinib combination in MUM in mid-2022. The company is pre-clinically evaluating potential expansion opportunities for Darovasertib in other oncology indications, including in additional cMET-driven tumours, such as hepatocellular carcinoma and non-small cell lung cancer, and in KRAS G12C non-small cell lung cancer.