Carmel biotech company lands key designation for drug candidate

CARMEL, Ind. (Inside INdiana Business) — A Carmel-based biopharmaceutical company has received an Orphan Drug designation from the U.S. Food and Drug Administration for its lead therapeutic product candidate. The drug, MBX 2109, is being developed for the treatment of hypoparathyroidism.

The Orphan Drug designation, the company says, is designed to support the development of medicines that address diseases or conditions that affect fewer than 200,000 people in the country. It qualifies MBX for incentives such as tax credits for clinical testing, prescription drug user fee exemptions and marketing exclusivity for seven years once the drug receives FDA approval.

Hypoparathyroidism is a rare endocrine disorder that decreases calcium and increases phosphorus levels in the blood. MBX says about 200,000 people around the world have the disorder, which can cause problems in the kidneys and bones.

“This is an exciting achievement for MBX and underscores the significant need to expand treatment options for hypoparathyroidism,” MBX Chief Executive Officer Kent Hawryluk said in written remarks. “We believe MBX 2109 has the potential to overcome limitations of the current standard of care with fewer administrations necessary due to its long-acting profile. Our goal is to improve the outcomes and quality of life for patients with this chronic condition.”

MBX 2109 is currently being evaluated in a Phase 1 clinical trial.

The designation was awarded nearly two years to the day after MBX Biosciences closed on a $35 million Series A round of funding. Shortly after the funding round closed, the company was spotlighted in our Life Sciences INdiana newsletter.