Verastem, Inc. (VSTM - Free Report) announced that the FDA has granted orphan drug designation to Copiktra (duvelisib) for the treatment of T-Cell lymphoma. The drug already enjoys Fast Track designation for this indication.
Notably, orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. This designation also makes the company entitled to certain other benefits, including tax credits related to clinical trial expenses and an exemption from FDA user fee.
Please note that Copiktra is already approved as third or later-line treatment for chronic lymphocytic leukemia/small lymphocytic lymphoma and follicular lymphoma.
A phase II study — PRIMO — is evaluating Copiktra as monotherapy in patients with relapsed or refractory peripheral T-cell lymphoma ("PTCL"). In June, the company presented data from phased I study, which evaluated twice-daily 25mg or 75mg doses of Copiktra monotherapy or lead-in monotherapy to a combination regimen with romidepsin or Takeda's Velcade (bortezomib) in PTCL patients. Data from the phase I study demonstrated that Copiktra achieved compelling clinical activity.
Apart from PTCL, Verastem is developing the drug in non-Hodgkin lymphoma, lung cancer, mesothelioma, ovarian cancer and pancreatic cancer.
In 2016, Verastem had in-licensed Copiktra from Infinity Pharmaceuticals (INFI - Free Report) . Last year, Verastem entered into an exclusive collaboration and license agreement with China’s CSPC Pharmaceutical Group to develop and commercialize Copiktra for treating different types of cancer indications in China. In July, Verastem signed an exclusive license agreement with Sanofi (SNY - Free Report) for development and commercialization of Copiktra in Russia and CIS, Turkey, the Middle East and Africa.
Furthermore, Verastem is developing an oral small molecule kinase inhibitor, defactinib, as treatment for ovarian cancer, pancreatic cancer, mesothelioma, non-small cell lung cancer and other solid tumors. A phase Ib study is evaluating the candidate in combination with Merck's (MRK - Free Report) PD-1 inhibitor Keytruda as well as Eli Lilly’s Gemzar for treating patients with advanced pancreatic cancer.
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