Nucala Wins New FDA Indication to Treat Rare Autoimmune Disease

Modesto Morganelli
Dicembre 13, 2017

GlaxoSmithKline plc (GSK, GSK.L) announced that the US Food and Drug Administration has approved Nucala or mepolizumab as the first targeted treatment for eosinophilic granulomatosis with polyangiitis or EGPA, previously known as Churg-Strauss syndrome. EGPA can result in damage to lungs, sinuses, skin, heart, gastrointestinal tract, nerves and other organs.

"Prior to today's action, patients with this challenging, rare disease did not have an FDA-approved treatment option", said Badrul Chowdhury, MD, PhD, director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA Center for Drug Evaluation and Research. "It's notable that patients taking Nucala in clinical trials reported a significant improvement in their symptoms".

"Following physician and patient experience with Nucala in severe eosinophilic asthma, we are thrilled that the FDA has expanded the use of this medicine to patients with EGPA, another eosinophil-driven disease, enabling GSK to make it available to patients", Dube said. The global incidence is generally reported to be in the range of 1-4 per million, with an estimated prevalence of approximately 14-45 per million. Orphan drug designation provides incentives to assist and encourage the development of drugs for rare diseases. Mepolizumab is an interleukin-5 antagonist monoclonal antibody (IgG1 kappa) produced by recombinant DNA technology in Chinese hamster ovary cells.

The study evaluated the efficacy and safety of 300mg of mepolizumab administered subcutaneously every four weeks versus placebo as additional therapy to standard of care in 136 patients with relapsing and or refractory EGPA. Disease remission while on an OCS dose of ≤4mg of prednisone was considered the primary efficacy outcome. The investigators found that patients treated with mepolizumab had a higher remission rate compared with the placebo group. In addition, significantly more patients who received 300 mg of mepolizumab achieved remission within the first 24 weeks and remained in remission for the remainder of the 52-week study treatment period compared with patients who received the placebo.

Patients receiving 300 mg of mepolizumab achieved a significantly greater accrued time in remission compared with placebo.

Common adverse events associated with mepolizumab include headache, injection site reaction, back pain, and fatigue.

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