BOSTON--(BUSINESS WIRE)-- Verastem, Inc. (Nasdaq:VSTM) (Verastem Oncology or the Company), a biopharmaceutical company focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients, today announced duvelisib (COPIKTRATM) has received orphan drug designation from the U.S. Food and Drug Administration for use in the treatment of T-Cell lymphoma. The designation was created to encourage the development of drugs that may provide significant benefit to patients suffering from rare diseases.
“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,” said Brian Stuglik, Chief Executive Officer of Verastem Oncology. “We look forward to sharing the results of our Phase 2 PRIMO study and efficiently advancing our development program in this indication.”
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 (FL) 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 (PTCL) and further characterize its efficacy and tolerability in this population.
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 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.
About Peripheral T-Cell Lymphoma
Peripheral T-cell lymphoma (PTCL) is a rare, aggressive type of non-Hodgkin lymphoma (NHL) that develops in mature white blood cells called “T cells” and “natural killer (NK) cells”1 which circulate with the lymphatic system.2 PTCL accounts for between 10-15% of all non-Hodgkin lymphomas (NHLs) and generally affects people aged 60 years and older.1 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.2 There is currently no established standard of care for patients with relapsed or refractory disease.1
SELECT IMPORTANT SAFETY INFORMATION
This does not include all information needed to use COPIKTRA™ (duvelisib) safety and effectively. See full Prescribing Information.
WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR COLITIS, CUTANEOUS REACTIONS, and PNEUMONITIS
See full Prescribing Information for complete boxed warning
- Fatal and/or serious infections occurred in 31% (4% fatal) of COPIKTRA-treated patients. Monitor for signs and symptoms of infection. Withhold COPIKTRA if infection is suspected.
- Fatal and/or serious diarrhea or colitis occurred in 18% (