A Pittsburg-based drug development company has received U.S. Food and Drug Administration (FDA) approval for phase 3 clinical trials of a product to treat severe, persistent pain. The medication, T-121, is being developed by Thar Pharmaceuticals and is expected to enter the market by 2019. T-121 is an oral version of Novartis’ intravenous-only zoledronic acid, which is sold under the brand name Zometra. T-121 will be intended for patients suffering from complex regional pain syndrome/reflex sympathetic dystrophy (CRPS/RSD), a chronic pain condition often brought on by some sort of trauma. About 70,000 people across the U.S. experience pain from CRPS/RSD, which can become chronic over time and become a disabling condition.
Treatments for CRPS/RSD.
There are many different types of treatments for CRPS and new ones come about relatively frequently, although what works for one does not usually work for another, making treating the condition all the more difficult. Generally, the earlier CRPS is caught and treated correctly, the greater the chance that the condition will respond to medical treatment. Although most doctors agree that a combination of diet, exercise, physical therapy, and medication is the best treatment of CRPS for most patients, exactly what that combination may be and which medications work best is a highly debated issue among pain management doctors. There are no FDA-approved treatments for the pain of CRPS/RSD. Thar Pharmaceuticals developed the drug through the FDA’s orphan disease program, which allows for expedited review, tax credits and other competitive advantages for medications that help fewer than 200,000 people.
CRPS/RSD affects fewer than 200,000 patients in the U.S. each year, according to the National Organization for Rare Disorders.
Thar Pharmaceuticals Inc. has received Food and Drug Administration approval for phase 3 clinical trials of its core product, dubbed T-121. Marketing of the oral drug, which will be used to treat severe, persistent pain, is expected by 2019, according to President and CEO Raymond Houck.“Getting to phase 3 is a big deal,” said Mr. Houck, 58.Phase 3 trials last up to four years. The test is intended to gauge a drug compound’s effectiveness, according to the FDA. Only 25 percent to 30 percent of drugs reach phase 3.Thar, which was founded in 2008 and employs seven people, is targeting complex regional pain syndrome, a condition that usually follows a bone fracture, serious burn or other trauma. About 70,000 people nationwide experience such pain, which can abate over time or become a disabling condition.There are no Food and Drug Administration-approved treatments for the pain. Thar developed the drug through the FDA’s orphan disease program, which allows for expedited review, tax credits and other competitive advantages for medications that help fewer than 200,000 people. Historically, big drug companies have shunned diseases that affect few people, focusing instead on making the next blockbuster statin, a medication taken by millions of people for high cholesterol.But Big Pharma has been increasingly paying attention to orphan disease treatments because of the potentially big paybacks for new prescription medications. Thar is not yet profitable and funding for operations has come from private investors.Thar’s focus is converting intravenously administered medications into ones that can be taken orally, preferred by health insurers because oral drugs are less costly to administer. Patients also tend to prefer oral drugs because administration is noninvasive.Thar’s T-121 is an oral version of Novartis’ intravenous-only zoledronic acid, which is sold under the Zometra brand name and prescribed to prevent or treat weakened bones and other conditions. Zometra’s safety record is expected to further speed FDA review of Thar’s oral version, Mr. Houck said.