Shares of Verastem, Inc. (VSTM) saw a sharp rise in volume last week after the company announced that its drug targeting the treatment of T-cell lymphoma, duvelisib (COPIKTRA), had earned orphan drug designation from the U.S. Food and Drug Administration. The designation was created to encourage the development of drugs that may provide significant benefit to patients suffering from rare diseases. Share volume increased by 2X-3X normal trading activity after the news was announced.
Verastem is a biopharmaceutical company focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients. The company previously announced that COPIKTRA had received Fast Track status to treat Peripheral T-cell lymphoma.
Verastem Drug On Track For Expedited Approval
Commenting on the new designation for COPIKTRA, Brian Stuglik, Chief Executive Officer of Verastem Oncology said,
“Receiving orphan drug designation for T-Cell Lymphoma, in addition to the previously-granted Fast Track status, for Peripheral T-Cell lymphoma, marks another important regulatory milestone to bring COPIKTRA to patients who are faced with this aggressive type of disease with limited therapeutic options,” He added, “We look forward to sharing the results of our Phase 2 PRIMO study and efficiently advancing our development program in this indication.”
COPIKTRA is already approved in the United States for the treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least 2 prior therapies and accelerated approval in follicular lymphoma (FL) after at least 2 prior systemic therapies. The approval of COPIKTRA to treat T-cell lymphoma would be an additional indication for the drug.
According to the company, the ongoing Phase 2 PRIMO study will provide guidance on a duvelisib monotherapy dosing regimen in patients with relapsed or refractory peripheral T-cell lymphoma (PTCL) and further characterize its efficacy and tolerability in this population.
Orphan Drug Advantages Provided To COPIKTRA
The Orphan Drug status awarded to Verastem’s COPIKTRA can help to carve a niche for the drug after approval. In the U.S., under the Orphan Drug Act, the FDA’s Office of Orphan Products Development (OOPD) grants orphan drug status to a drug or biologic intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders, which is generally a disease that affects fewer than 200,000 individuals in the U.S. and that are expected to provide a significant therapeutic advantage over existing treatments. Orphan designation further qualifies a company for benefits that apply across all stages of drug development, including an accelerated approval process, seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and a waiver of certain administrative fees.
If approved, COPIKTRA will add to the company’s already approved product list, with its first FDA approved product now available for the treatment of patients with certain types of indolent non-Hodgkin’s lymphoma (iNHL). The Verastem pipeline is comprised of product candidates that seek to treat cancer by modulating the local tumor microenvironment.
Shares of Verastem have been consolidating at around the $1.20 level and settled Friday at $1.19 per share. In June, Verastem said it is reiterating its previously issued financial guidance for the full year 2019, saying that it anticipates revenue from sales of Copiktra to be in the range of $10 to $12 million based on revenue to date, current run rates and near-term expectations.