The U.S. Food and Drug Administration approved Crinetics Pharmaceuticals’ drug to treat a rare hormonal disorder on Thursday, making it the first once-daily oral pill available for patients in the country.
The drug, Palsonify, chemically known as paltusotine, was approved to treat acromegaly, a rare condition in which the pituitary gland in the brain produces excess growth hormone, causing the bones, organs and other tissues to grow bigger, caused due to a tumor on the pituitary gland.
Treatment options for acromegaly include surgery to remove pituitary tumors, radiation therapy and medications such as somatostatin analogs that reduce growth hormone levels and may shrink tumors.
The current standard-of-care involves monthly injections of drugs such as octreotide or lanreotide.
Palsonify, to be administered once daily, will be available from early October.
Crinetics said it is working closely with payers, healthcare providers and patient advocacy organizations to support those who may benefit from this treatment.
The approval was backed by data from two late-stage studies that tested the drug’s safety and efficacy in previously treated and medically untreated adults.
In the studies, paltusotine, maintained IGF-1 levels – a protein that plays a role in moderating the growth hormone – in patients with acromegaly who switched from monthly injectable medications. It also rapidly decreased IGF-1 levels in medically untreated acromegaly patients.
New patients, who have not been on previous therapy as well as patients who have had issues with the long-acting injections will gravitate towards Palsonify, said Jill Sisco, president of Acromegaly Community a patient support group.
Another FDA-approved oral treatment for acromegaly is Chiesi’s Mycapssa, a twice daily long-term maintenance treatment for patients who have responded to octreotide or lanreotide.
Other injectable treatment options include Pfizer’s (PFE.N), opens new tab Somavert, Novartis'(NOVN.S), opens new tab Sandostatin and Signifor.
Analysts at Piper Sandler estimate peak sales of Palsonify could reach $1.5 billion by 2030.
