FDA approves multiple myeloma therapy Xpovio despite safety concerns
pharmafile | July 4, 2019 | News story | Sales and Marketing | FDA, Karyopharm, Xpovio, multiple myeloma, pharma
The FDA has made the controversial decision to approve the nuclear export inhibitor Xpovio (selinexor) from Karyopharm Therapeutics, in combination with dexamethasone, in the treatment of a sub-population of relapsed or refractory multiple myeloma (RRMM) patients, despite an advisory panel recommending against approval earlier this year.
The US agency chose to approve the drug under its accelerated pathway for adult RRMM patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody.
This was despite a panel in February voting 5-8 against recommending the drug due to concerns over its risks. However, the approval was in part driven by the unmet need for treatment faced by patients with the condition. In light of this, Xpovio’s label includes a warning to doctors to reduce the prescribed dosage in the event of certain side-effects.
The approval was based on Phase 2 data which showed that Xpovio had an overall response rate of 25.3% in a subgroup of 83 patients whose disease was refractory to bortezomib, carfilzomib, lenalidomide, pomalidomide, and daratumumab. Additionally, median time to response was four weeks and median duration of response was 3.8 months.
However, 27% of the 202 participants across the whole trial were forced to discontinue treatment due to adverse reactions, while the rate of fatal adverse reactions was 8.9%.
“Despite recent advances in the treatment of multiple myeloma, almost all our patients will develop disease that is resistant to the five most commonly used anti-myeloma drugs we currently have available, and the prognosis for this patient population is particularly poor,” commented Dr Paul Richardson, Clinical Program Leader and Director of Clinical Research, Jerome Lipper Multiple Myeloma Center at the Dana-Farber Cancer Institute. “The accelerated approval of oral XPOVIO marks an important advance in the treatment paradigm for patients with relapsed refractory multiple myeloma, and in my view, is an important addition to our therapeutic armamentarium.”
Dr Michael G Kauffman, Chief Executive Officer of Karyopharm, also remarked: “Having worked on novel drugs in myeloma beginning with Velcade in the year 2000, I have been thrilled to see such exciting progress overall in the field where there are substantial increases in patients’ duration and quality of life. The accelerated approval of oral Xpovio targeting XPO1 represents the first approval against a new target in myeloma since 2015, and we look forward to advancing the further clinical development of Xpovio.”
Matt Fellows
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