The U.S. Food and Drug Administration approved Copiktra (duvelisib) capsules for the treatment of adults with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after a minimum of two prior therapies.
The medicine also received accelerated approval for follicular lymphoma patients whose disease returned or failed to respond to at least two prior systemic therapies. Continued approval for follicular lymphoma depends on clinical benefits in confirmatory trials.
Developed by Verastem Oncology, Copiktra the first approved dual inhibitor of PI3K-delta and and PI3K-gamma, two enzymes known to support the growth and survival of malignant immune B-cells.
CLL and SLL are common types of indolent, or slow-growing, non-Hodgkin’s lymphoma. They are essentially the same disease, differing in the primary cancer location. When the cancer cells are mainly in the bloodstream and the bone marrow, the disease is referred to as CLL, while SLL refers to when the cancer cells are mostly in the lymph nodes.
“With today’s FDA approval of Copiktra, Verastem Oncology is delivering upon our commitment to patients with cancer by bringing a new oral medicine to market,” Robert Forrester, the company’s president and CEO, said in a press release. “We are driven by the strength and courage of those battling cancer, and we are committed to advancing therapies such as Copkitra with the potential to make a significant impact for patients, their caregivers and physicians.”
Copiktra’s approval is based on clinical trial results from more than 400 adults — the Phase 2 DYNAMO study (NCT01882803) for indolent non-Hodgkin’s lymphoma, and the DUO Phase 3 clinical trial (NCT02004522) for CLL/SLL.
Among the 83 patients with follicular lymphoma, 42% achieved at least a partial reduction in their tumors. Response rates for SLL were 68%.
DUO compared the effectiveness of stand-alone therapy with 25 mg Copiktra versus Novartis‘ Arzerra (ofatumumab) in 319 patients with relapsed or refractory CLL/SLL following at least one prior therapy.
In a subset of heavily treated patients — those with at least two previous lines of therapy — progression-free survival results showed that Copiktra outperformed Arzerra, and it was on this point that approval was based. Specifically, these advanced patients treated with Copiktra remained progression free for a median of 16.4 months, compared to 9.1 months in those using Arzerra.
“We are immensely grateful to the many patients who participated in the duvelisib clinical trial program,” Forrester said.
“Copiktra is an important addition to the evolving treatment paradigm for patients with CLL/SLL and FL [follicular lymphoma],” said Ian Flinn, MD, PhD, the lead investigator of the DYNAMO and DUO studies.
FDA approval follows its April 2018 decision to grant Copiktra priority review as a potential treatment for the two lymphoma types.
“Each lymphoma patient’s experience is unique, and today’s approval offers patients with CLL/SLL and FL a new treatment option and a new opportunity for hope,” said Meghan Gutierrez, CEO of the Lymphoma Research Foundation. “We applaud the FDA, Verastem and the patients who participated in the clinical trials.”
The therapy will be available on the U.S. market immediately. Verastem will help patients access the medication through its personalized Verastem Cares program, which provide information and assistance to patients prescribed Copiktra.
“We are excited to offer a new treatment that can allow patients to manage their disease with an oral monotherapy,” said Joseph Lobacki, Verastem’s executive vice president and chief commercial officer.
“We continue to hear from physicians and patients that there is a great need for additional treatment options to fight chronic cancers such as CLL/SLL and FL,” said Lobacki, who added that the company has already assembled an experienced oncology commercial team and established its distribution network to prepare for Copiktra’s commercialization.
Verastem cautions that Copiktra treatment may cause four fatal and/or serious toxicities — infections, diarrhea or colitis, skin reactions, and pneumonitis. Full prescribing information for Copiktra is available here.
Verastem is implementing a risk evaluation and mitigation strategy offering dosing and safety information to support physicians’ management of patients on Copiktra.
The company’s management team recently hosted a conference call to discuss Copiktra’s approval. A replay of the webcast is now available on Verastem’s website.