Bortezomib Receives New FDA Indication for Retreatment of Patients with Myeloma

September 2014, Vol 5, No 7 - FDA Approvals, News & Updates


Bortezomib (Velcade; Millennium/Takeda Oncology), which is already approved by the FDA as an intravenous (IV) or subcutaneous treatment for patients with multiple myeloma, recently received a supplemental new indication for the retreatment of adults with myeloma whose disease had previously responded to bortezomib therapy and had relapsed (ie, progressed) at least 6 months after the last treatment with bortezomib.

Retreatment with bortezomib can be restarted at the last dose the patient had tolerated previously. The new indication includes a labeling update with dosing guidelines and safety and efficacy data related to the use of bortezomib as a single agent or in combination with dexamethasone.

“For the past 11 years, Velcade has played an important role as the only therapy proven to extend overall survival for patients with newly diagnosed and relapsed multiple myeloma,” said Michael Vasconcelles, MD, Global Head, Oncology Therapeutic Area Unit, Takeda. “With these newly approved dosing guidelines, physicians will be able to provide their patients, who have previously received Velcade, with an effective treatment extending Velcade use across the continuum of care of multiple myeloma.”

The new supplemental indication was based on the results from a phase 2 clinical trial, RETRIEVE, as well as other supportive data. The single-arm, open-label, international RETRIEVE trial included 130 patients with myeloma (aged ?18 years) who had previously responded to bortezomib-based therapy and whose disease relapsed at least 6 months after previous therapy.

The overall response rate was 38.5%; 1 patient achieved a complete response and 49 patients achieved a partial response. The median duration of response was 6.5 months (range, 0.6-19.3 months).

All patients had previously received a treatment regimen with bortezomib, with a median of 2 previous lines of therapy (range, 1-7), and had previously demonstrated a partial response or better. A total of 94 patients received dexamethasone in combination with bortezomib. RETRIEVE met its primary end point of best confirmed response to retreatment.

The safety of retreatment with bortezomib was consistent with the known safety profile of IV bortezomib in patients with relapsed myeloma; no cumulative toxicities were reported with retreatment.

The most common adverse event was thrombocytopenia, which was reported in 52% of patients. The incidence of grade ?3 thrombocytopenia was 24%. Peripheral neuropathy was observed in 28% of patients, and the incidence of grade ?3 peripheral neuropathy was 6%. The incidence of serious adverse reactions was 12.3%. The most common serious adverse reactions were thrombocytopenia (3.8%), diarrhea (2.3%), herpes zoster (1.5%), and pneumonia (1.5%). Overall, 13% of patients discontinued bortezomib retreatment because of adverse events. (August 8, 2014)