Research & Development
Verastem Oncology Receives Orphan Drug Designation from FDA for Copiktra for the Treatment of T-Cell Lymphoma
7 October 2019 - - US-based biopharmaceutical company Verastem, Inc.'s (NASDAQ: VSTM) duvelisib (Copiktra) has received orphan drug designation from the US Food and Drug Administration for use in the treatment of T-Cell lymphoma, the company said.

The designation was created to encourage the development of drugs that may provide significant benefit to patients suffering from rare diseases.

Copiktra is 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 after at least 2 prior systemic therapies.

Copiktra is not currently approved for the treatment of T-cell lymphoma. The company's ongoing Phase 2 PRIMO study will provide guidance on a duvelisib monotherapy dosing regimen in patients with relapsed or refractory peripheral T-cell lymphoma and further characterize its efficacy and tolerability in this population.

In the US, under the Orphan Drug Act, the FDA's Office of Orphan Products Development 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 US and that are expected to provide a significant therapeutic advantage over existing treatments.

Orphan designation 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 US clinical trials, eligibility for orphan drug grants, and a waiver of certain administrative fees.

Peripheral T-cell lymphoma is a rare, aggressive type of non-Hodgkin lymphoma that develops in mature white blood cells called "T cells" and "natural killer cells"1 which circulate with the lymphatic system.2 PTCL accounts for between 10-15% of all non-Hodgkin lymphomas and generally affects people aged 60 years and older.

Although there are many different subtypes of peripheral T-cell lymphoma, they often present in a similar way, with widespread, enlarged, painless lymph nodes in the neck, armpit or groin.

There is currently no established standard of care for patients with relapsed or refractory disease.

Copiktra is an oral inhibitor of phosphoinositide 3-kinase, and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells.

PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.

Copiktra is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma after at least two prior systemic therapies.

Copiktra is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma, for which it has received Fast Track status, and is being investigated in combination with other agents through investigator-sponsored studies.

Verastem Oncology (NASDAQ: VSTM) is a commercial biopharmaceutical company committed to the development and commercialization of medicines to improve the lives of patients diagnosed with cancer.
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