Karyopharm Therapeutics Inc. (NASDAQ: KPTI) has announced that the FDA had granted its novel oral, Selective Inhibitor of Nuclear Exports (SINE) compound, eltanexor, an orphan drug designation for myelodysplastic syndromes (MDS) treatment. MDS is a collection of diseases associated with ineffective blood components production because of poor bone marrow function and has a risk of progressing to acute myeloid leukemia.
Karyopharm evaluating eltanexor as monotherapy in solid tumors
In an active open-label Phase 1/2 trial, Karyopharm is testing eltanexor as a monotherapy or with authorized and experimental treatments in patients with a variety of solid tumors and hematologic malignancies (KCP-8602-801; NCT02649790). Karyopharm previously announced encouraging results from an investigator-funded Phase 1 research testing eltanexor as a single agent in patients with a hypomethylating agent (HMA)-refractory MDS eltanexor showed a 53 percent overall response rate with 9.9 months median overall survival. This compares positively to previous HMA-refractory MDS patients’ survival rates of four to six months.
CEO of Karyopharm Richard Paulson stated, “We are pleased to receive the FDA’s orphan drug designation for eltanexor in MDS and believe it reinforces eltanexor’s potential to improve clinical outcomes for patients with HMA-refractory MDS. We are focused on advancing our ongoing clinical trials and remain steadfast in our commitment to bringing this new treatment option to patients and their families.”
US diagnoses 15,000 people with high-risk MDS yearly
Around 15,000 persons in the United States are identified with intermediate-to-high risk MDS each year. HMAs are the existing standard of therapy for recently diagnosed, high-risk MDS patients. Interestingly, only 40-60% of the patients, on the other hand, respond, and these responses often last less than 2 years. HMA-refractory illness has a poor outcome, with a median overall survival of 4 to 6 months. For HMA-refractory MDS, there are presently no authorized treatments.
Orphan drug designation is given to promote the development of drugs targeting conditions with less than 200,000 US patients. The designation qualifies the company for some incentives applying across all phases of development, including a possible 7-years market exclusivity after marketing approval.