IDEAYA: Darovasertib Gets Orphan Drug Designation For Uveal Melanoma

(RTTNews) - IDEAYA Biosciences, Inc. (IDYA), a synthetic lethality focused precision medicine oncology company, Monday announced that the U.S. Food and Drug Administration has granted orphan-drug designation to darovasertib, a potential first-in-class protein kinase C or PKC inhibitor, for the treatment of uveal melanoma.

The company said it is currently evaluating the synthetic lethal combination of darovasertib, a PKC inhibitor, and crizotinib, a cMET inhibitor, in patients with metastatic uveal melanoma or MUM and in patients with GNAQ or GNA11 mutant solid tumors, in an ongoing Phase 1/2 clinical trial (NCT03947385) pursuant to a clinical trial collaboration and supply agreement with Pfizer.

Orphan-drug designation or 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 U.S.

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.

Matthew Maurer, Vice President, Head of Clinical Oncology and Medical Affairs, at IDEAYA, said, "We are excited to advance darovasertib towards a potential registration-enabling trial in metastatic uveal melanoma, and the orphan-drug designation is an important step towards our goal to bring this novel therapy to patients."

As of May 1, 2022, Darovasertib has been evaluated in over 200 patients, including 74 patients in combination with crizotinib. The company is targeting 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.