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Karyopharm's (KPTI) Eltanexor Gets Orphan Drug Tag for MDS

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Karyopharm Therapeutics Inc. KPTI announced that the FDA has granted Orphan Drug designation to its novel oral, selective inhibitor of nuclear export compound, eltanexor, for the treatment of myelodysplastic syndromes (“MDS”).

The Orphan Drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes one that affects fewer than 200,000 individuals in the United States. The designation also includes incentives, including financial aid for clinical testing and seven-year marketing exclusivity upon regulatory approval.

MDS represents a group of diverse blood disorders that occur because of the bone marrow cells’ failure to mature into healthy blood cells.

An ongoing open-label phase I/II study is currently evaluating eltanexor as a single agent or in combination with approved as well as prospective investigational agents for addressing patients with various types of hematologic and solid-tumor cancers.

Karyopharm earlier announced positive data from a phase I study that investigated eltanexor as a single agent in patients with hypomethylating agent (“HMA”)-refractory MDS. Data from the same showed that treatment with eltanexor led to an overall response rate of 53% and median overall survival of 9.9 months. Currently there are no approved therapies to treat HMA- refractory MDS.

Per the company, an estimated 15,000 people in the United States are diagnosed with intermediate to high-risk MDS each year. Hence, if successfully developed and upon potential approval, eltanexor can become a new treatment option for the given patient population.

Several other drugmakers and big companies are engaged in developing their pipeline candidates and marketed drugs for treating MDS.

Pharma giants AbbVie ABBV and Roche RHHBY are jointly developing cancer drug Venclexta (called Venclyxto in Europe). In July 2021, the FDA granted a Breakthrough Therapy designation to Venclexta in combination with azacitidine for the potential treatment of adult patients with previously untreated intermediate-, high- and very high-risk MDS.

Both AbbVie and Roche jointly commercialize Venclexta in the United States while AbbVie markets it outside the country. AbbVie and Roche equally share all profits and losses related to the development and commercialization of Venclexta in the United States.

Small biotech, Geron Corporation GERN, is developing its lead pipeline candidate, imetelstat, for treating MDS. A phase III study, IMerge, is evaluating the candidate in patients with lower-risk MDS.

Geron is also developing imetelstat for treating relapsed/refractory myelofibrosis. Geron is planning to develop imetelstat in additional hematologic myeloid malignancies.


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