FDA Approves GSK’s New Drug for Rare Blood Disorders

On Sept. 25, 2020, FDA announced the approval of GlaxoSmithKline’s (GSK’s) Nucala (mepolizumab) for treating a group of rare blood disorders known as hypereosinophilic syndrome (HES), representing the first drug to treat this condition in nearly 14 years, according to an agency press release.

Nucala is specifically indicated for adults and children 12 years and older who have HES for six months or longer without another identifiable non-blood related cause of the disease.

HES is a heterogeneous group of rare disorders associated with persistent eosinophilia with evidence of organ damage. Skin rashes, itching, asthma, difficulty breathing, abdominal pain, vomiting, diarrhea, arthritis, muscle inflammation, congestive heart failure, deep venous thrombosis, and anemia are among the symptoms of HES. Nucala received orphan drug designation for the HES indication and was granted fast track designation and priority review.

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

“Today’s approval marks the first time in over a decade that there is a new FDA-approved treatment option for patients with hypereosinophilic syndrome,” said Ann Farrell, MD, director of the Division of Nonmalignant Hematology in FDA’s Center for Drug Evaluation and Research, in the press release. “FDA is committed to helping develop safe and effective treatment options for this group of rare and debilitating blood diseases and other rare conditions.”

Source: FDA​