Shire Nabs Breakthrough Tags for Drug to Treat Rare Gastrointestinal Conditions

Published: Jun 15, 2016

Shire Nabs Breakthrough Tag for Drug to Treat Rare Gastrointestinal Conditions June 13, 2016
By Alex Keown, BioSpace.com Breaking News Staff

LEXINGTON, Mass. – Shire ’s rare disease pipeline is getting stronger. The U.S. Food and Drug Administration awarded Breakthrough Therapy designation to two of Shire’s investigational gastrointestinal drugs, the company announced this morning.

Shire’s SHP621 (budesonide oral suspension) for eosinophilic esophagitis and SHP625 (maralixibat) for progressive familial intrahepatic cholestasis type 2, will face an expedited regulatory review process due to the FDA designation. The FDA’s Breakthrough Therapy designation is granted to a treatment intended to treat a serious or life-threatening disease and preliminary clinical evidence submitted by a drug company indicates the drug “may demonstrate substantial improvement on one or more clinically significant endpoints over current standard of care.”

SHP621 BOS is an oral form of the asthma steroid budesonide, marketed as Pulmicort by AstraZeneca. Shire’s drug is formulated specifically for EoE. Shire said the oral version of its EOE treatment was more effective than an inhaled version. The FDA’s Breakthrough Therapy designation was based on Phase II data that showed a 12 week regimen of SHP621 BOS “significantly reduced both dysphagia symptoms and achieved higher proportion of subjects with histologic response,” in adolescent and adult patients with EoE. Eosinophilic esophagitis is a chronic and rare disease that stems from an elevated number of eosinophils, a type of white blood cell, which infiltrate the walls of the esophagus and may lead to difficulty in swallowing. The disease affects between 15 and 55 people per 100,000 in the western world, Ireland-based Shire said in a statement.

Maralixibat, an ASBT inhibitors, is being evaluated in several rare cholestatic liver diseases for both pediatric and adult populations. ASBT (apical sodium-dependent bile acid transporter) inhibitors are designed to block bile acid reabsorption in the ileum and increases fecal bile acid excretion. Shire’s Maralixibat is being developed as an oral treatment for pediatric use. The Breakthrough Therapy designation was supported by the interim results of Shire’s Phase II single-arm, open-label study in pediatric patients with PFIC. Maralixibat was granted orphan designation by both the FDA and the European Medicines Agency. PFIC refers to a group of autosomal-recessive liver disorders of childhood that disrupt bile formation and present with cholestasis. PFIC is estimated to affect one in 50,000 to one in 100,000 births.

“Receiving Breakthrough Therapy Designation on two pipeline products this past week reflects the potential of our strong and innovative pipeline of more than 60 programs,” Flemming Ornskov, Shire’s chief executive officer, said in a statement. “Shire is committed to bringing innovation to the rare and specialty areas we focus on. We persevere to see compounds through the many stages of development through their challenges and successes, and always keep patients with unmet needs top of mind.” Shire’s stock is up slightly this morning, currently trading at $177.68 per share.

Thanks in part to its recent acquisition of Baxalta, Shire has one of the largest rare disease pipelines in the world. Shire's top drug is Vyvanse, for the treatment of attention deficit hyperactivity disorder (ADHA) and for moderate-to-severe Binge Eating Disorder. Another rare disease drug in Shire’s arsenal is Cinryze for the treatment of hereditary angioedema, which is expected to generate $765 million. In October, Cinryze received Fast Track Designation from the U.S. Food and Drug Administration intravenous administration in subjects with Antibody Mediated Rejection in renal transplant recipients.

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