Kadmon's Chronic GVHD Drug Wins First FDA Approval for Company

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The U.S. Food and Drug Administration (FDA) comes through for blood cancer patients experiencing chronic graft-versus-host disease after a bone marrow transplant. On Friday, the agency approved belumosudil as a new treatment option for anyone over 12 with cGVHD after two prior lines of therapy have failed. 

Kadmon’s flagship drug will be marketed as Rezurock. It’s the first small molecule approved that inhibits ROCK2, an inflammatory response and fibrotic signaling pathway.  

Inhibiting this pathway helped a majority of the 65 patients treated with 200 mg of belumosudil a day achieve a response – 6% achieved a complete response and 69% achieved a partial response. In the 75% of patients with a response, no new therapies were needed in 62% of patients for at least 12 months. 

"Patients receiving REZUROCK reported significant improvements in cGVHD symptoms, showing that not only did treatment result in organ responses, but it also made people feel better. This is so important for a chronic disease with a high symptom burden," said Stephanie Lee, MD, MPH, Professor at the Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine. 

For many patients with blood cancers, a bone marrow transplant can be the answer for a complete cure. But that cure can come with a price, sometimes a fatal one.  

Up to 70% of transplant recipients develop acute GVHD within the first few months of treatment. Around 40% develop chronic GVHD 100 days or more after the transplant. The most effective bone marrow transplants are done with healthy donor stem cells. But when those donor cells mistakenly start attacking your own cells, that’s when GVHD becomes a serious problem. It’s estimated 14,000 patients in the US are currently living with cGVHD.  

"We are proud to introduce REZUROCK as a new treatment that uniquely addresses the underlying inflammatory and fibrotic pathophysiology of chronic GVHD," said Harlan W. Waksal, MD, President and CEO of Kadmon. 

Rezurock's target is unique at this point, but not the only option for the disease on the market. Its competition will include Incyte’s Jakafi (under FDA review for cGVHD indication with a target date of September 22) and Imbruvica, J&J and AbbVie’s drug approved back in 2017 as the first FDA-approved option for the treatment of cGVHD. 

Belumosudil had snagged Breakthrough Therapy Designation from the FDA back in October 2018. After submitting an NDA last September, the review was extended in March with a goal date for end of August. Reviewed under the pilot Real-Time Oncology Review program, the drug was approved six weeks ahead of schedule.  

The drug was granted Orphan Drug Designation for the treatment of systemic sclerosis last fall. 

Kadmon expects Rezurock will be available in the US by late August.  

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