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FDA Approves GlaxoSmithKline's Nucala To Treat Rare Blood Disorders

fda approved nucala 092820 lt

The U.S. Food and Drug Administration approved the first drug to treat adults and children aged 12 years and older with hypereosinophilic syndrome (HES) for six months or longer without another identifiable non-blood related cause of the disease.

GlaxoSmithKline's Nucala (mepolizumab) is the first FDA-approved drug in nearly 14 years to treat this group of rare and debilitating blood diseases and other rare conditions.

HES is a heterogeneous group of rare disorders associated with persistent eosinophilia (higher than normal levels of a type of disease-fighting white blood cell) with evidence of organ damage. Signs and symptoms vary significantly based on which parts of the body are affected.

Although any organ system can be involved in HES, the heart, central nervous system, skin, and respiratory tract are the most commonly affected.

The approval for Nucala was granted based on the results of a randomized, double-blind, multicenter, placebo-controlled trial in 108 patients with HES. In the study, patients were randomly assigned to receive Nucala or placebo by injection every four weeks. Fewer patients in the Nucala treatment group (28%) had HES flares compared to patients in the placebo group (56%), with a 50% relative reduction.

For the treatment of HES, Nucala received orphan drug designation, which provides incentives to assist and encourage drug development for rare diseases. Additionally, the application was granted fast track designation and priority review.

Nucala is also FDA-approved for patients aged 6 years and older with severe asthma with an eosinophilic phenotype and for adult patients with eosinophilic granulomatosis with polyangiitis, a rare autoimmune condition that causes blood vessel inflammation.

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