Deciphera Pharmaceuticals Wins Early FDA Nod for GI Cancer Treatment

Xconomy Boston — 

Some patients with a deadly type of tumor that forms in the gastrointestinal tract don’t respond to existing targeted therapies. On Friday the FDA approved Deciphera Pharmaceuticals drug ripretinib (Qinlock), a new treatment intended for patients with a gastrointestinal stromal tumor (GIST) who have failed at least three previous treatments.

Deciphera (NASDAQ: DCPH) said the agency’s approval, the company’s first, arrived three months earlier than expected. The Waltham, MA-based biotech announced plans to seek FDA approval of the cancer medicine last August. That application was accepted in February under priority review, setting up the drug for a decision by Aug. 13. Drug decisions can come sooner than the target date, and lately the FDA has been making a lot of early rulings for cancer drugs.

The company submitted ripretinib for regulatory review after the drug hit its main goal in a Phase 3 trial, INVICTUS, which enrolled 129 patients with gastrointestinal stromal tumors (GIST) who have failed at least three treatments.

About 4,000 to 6,000 adults in the US are diagnosed with GIST yearly, the company says. The tumors can start anywhere along the gastrointestinal tract, but most commonly occur in the stomach, small intestine, and large intestine.

Although the FDA has approved four targeted GIST therapies over the past 20 years, some patients’ tumors don’t respond to those treatments, according to a statement from Richard Pazdur, head of the FDA’s Oncology Center of Excellence and acting director of the agency’s Office of Oncologic Diseases.

Treatment for GIST starts with surgery. But if that doesn’t appropriately address the rare cancer, multiple kinase inhibitors are available, including imatinib (Gleevec), sunitinib (Sutent), and regorafenib (Stivarga). Patients who have been treated with three or more kinase inhibitors, including imatinib, are eligible to receive ripretinib.

In a statement from Deciphera about the drug’s approval, Margaret von Mehren, chief of sarcoma oncology and associate director for clinical research at the Fox Chase Cancer Center in Philadelphia, said the cancer is typically driven by a spectrum of mutations and secondary mutations typically prompt tumor progression after the cancer initially responds to kinase inhibitors.

Deciphera’s kinase “switch control” technology facilitates the drug’s ability to block multiple mutations that drive the disease, said the company’s chief medical officer, Matt Sherman, speaking on a conference call Friday afternoon.

Dan Martin, the company’s chief commercial officer, said the list price for a 30-day supply of the drug will be $32,000. In INVICTUS, ripretinib kept tumors from spreading—a measurement referred to progression-free survival—for a median of 6.3 months compared to a median of about one month for the patients who received a placebo.

Deciphera said ripretinib would become commercially available in the US next week. The company plans to ask European regulators to review the drug in the second half of this year.

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