The Food and Drug Administration (FDA) has approved PALSONIFY™ (paltusotine), the first once-daily oral medication for the first-line treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option.

Paltusotine becomes the first oral, nonpeptide somatostatin receptor type 2 agonist available for patients with acromegaly, who previously relied primarily on monthly injectable treatments. The approval represents a paradigm shift in managing this rare endocrine disorder.

The FDA based its approval on data from 2 phase 3 trials: PATHFNDR-1 and PATHFNDR-2. PATHFNDR-1 showed paltusotine maintained insulin-like growth factor-1 (IGF-1) levels and symptom control in patients transitioning from monthly injectable medications. PATHFNDR-2 demonstrated rapid IGF-1 reduction and symptom improvement in treatment-naive patients.

IGF-1 serves as the primary biomarker physicians use to monitor acromegaly treatment response.

Acromegaly results from excess growth hormone production, typically caused by pituitary adenomas.

The FDA had granted paltusotine Orphan Drug Designation in July 2020, a status reserved for treatments addressing rare diseases. This designation provided regulatory incentives including market exclusivity and tax credits for clinical development costs.

"With the FDA approval of our lead therapy Palsonify, today marks a new era for those living with acromegaly and also for Crinetics as a company," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "We are very pleased to be fulfilling our commitment to transforming patient lives. This approval is the first to come from our deep pipeline of first-in-class, small molecule drugs."

Source: Crinetics Pharmaceuticals