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AstraZeneca's Rare Pediatric Genetic Disorder Drug Granted Breakthrough Therapy Designation

Shanthi Rexaline

AstraZeneca plc (NYSE: AZN) shares, which have gained about 9 percent year-to-date, were seeing incremental upside Monday in the wake of a company announcement. 

The shares also received a shot in the arm from a Cowen upgrade from Market Perform to Outperform.

What Happened

U.K.-based AstraZeneca and U.S. pharma giant Merck & Co., Inc. (NYSE: MRK) announced the FDA has granted Breakthrough Therapy Designation for their investigational MEK1/2 inhibitor selumetinib.

Selumetinib, a potentially new medicine, is being evaluated in pediatric patients ages 3 and older for neurofibromatosis type 1 symptomatic and/or progressive, inoperable plexiform neurofibroma — a rare incurable genetic condition.

Why It's Important

NF1-related plexiform neurofibromas have no approved medications. 

The BTD, according to AstraZeneca, is an acknowledgment of the significant unmet need and the potential benefit of selumetinib in this setting, supporting a potential expedited review.

AstraZeneca shares were trading up 2.18 percent at $41.31 at the time of publication Monday, while Merck shares were higher by 0.14 percent at $83.32. 

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