Ironwood Pharma’s Sickle Cell Drug Gets Orphan Drug Designation

Ironwood Pharmaceuticals, headquartered in Cambridge, Massachusetts, announced the U.S. Food and Drug Administration (FDA) had granted its olinciguat (IW-1701) Orphan Drug Designation for sickle cell disease.

The FDA’s Office of Orphan Drug Products grants orphan status to drugs designed to treat diseases that affect fewer than 200,000 people in the U.S. It offers several benefits, including seven years of market exclusively after FDA approval, prescription drug user fee waivers and tax credits for qualified clinical trials.

Olinciguat is an oral soluble guanlylate cyclase (sGC) stimulator. The company is currently enrolling patients in its Phase II STRONG-SCD clinical trial. The company expects to enroll about 80 patients in the trial, which will evaluate the safety, tolerability, pharmaocokinetics and pharmacodynamics of the drug in sickle cell disease patients.

“There is an urgent need for new, innovative treatments for patients with sickle cell disease, a debilitating and potentially fatal inherited blood disorder that causes painful crises, organ damage and other serious complications,” said Christopher Wright, Ironwood’s senior vice president of global development and chief development officer, in a statement. “The orphan drug designation adds momentum to our clinical program investigating olinciguat, which has the potential to improve multiple aspects of sickle cell disease pathophysiology. The designation is also an important milestone in Ironwood’s evolution as we advance our pipeline of SGC stimulators focused on the treatment of serious and orphan diseases.”

In early May, Ironwood announced it will split into two independent companies. They will be Ironwood and what was being called R&D Co, although presumably a more appropriate name will be chosen. The idea is to separate its sGC business from its commercial and gastrointestinal business.

The split is expected to be finished in the first half of next year, and is designed to “unlock value, increase operational performance and strategic flexibility, and tailor capital structure for each business.”

When announced, company shareholders were not enthusiastic. Shares dropped more than 3 percent in early trading and as much in premarket trading as 6 percent.

Ironwood indicated it believes the separation would lead to it becoming profitable. It plans to build on its commercial success to accelerate the growth of its in-market product while advancing development programs for gastrointestinal disease, uncontrolled gout, and abdominal pain.

The R&D company will focus on the sGC pipeline, which includes olinciguat. The other compound in this pipeline includes its Phase II praliciguat being developed for cardiovascular disorders, diabetic nephropathies, heart failure, and hypertension in diabetes.

In a statement at the time, Peter Hecht, the company’s chief executive officer, said, “Since our founding 20 years ago, we have been driven by a simple mission: create and commercialize innovative drugs that can change patients’ lives and generate value for our shareholders. Ironwood today markets three commercial medicines, including Linzess, a category leader, and is advancing a deep pipeline of drug candidates targeting severe and high unmet need diseases.”

Linzess and Constella are prescribed to treat adults with irritable bowel syndrome (IBS) with constipation, and Duzallo and Zurampic are prescribed to treat gout. It also has IW-3718, being developed for persistent gastroesophageal reflux disease (GERD), with Phase III trials expected to start in this year’s third quarter. It also has linaclotide delayed release, which is being tested to treat abdominal pain associated with all forms of IBS.