Kadmon bags a quick win for its graft-versus-host disease drug ahead of August PDUFA date
About a year and a half after Kadmon trumpeted “knock-your-socks-off” results for its chronic graft-versus-host disease (GVHD) treatment, the FDA has followed through with a speedy approval six weeks before the agency was supposed to announce a decision.
The FDA has approved Rezurock (belumosudil) as a third-line treatment in adult and pediatric patients 12 years and older, based on pivotal results from the ROCKstar trial showing a 75% overall response rate and a 69% partial response rate.
Like the acute form of the disease, chronic GVHD can occur after am stem cell transplant when immune cells from the donor attack the recipient, causing inflammation and fibrosis in tissues like the skin, eyes, joints, liver and lungs. The condition affects about 14,000 patients in the US, according to Kadmon. Rezurock is designed to tamp down that inflammatory response by blocking Rho-associated coiled-coil kinase 2 (ROCK2). It’s now the first approved therapy targeting that protein.
Kadmon’s stock $KDMN jumped on the news, up 20.56% late Friday afternoon.
Of the 65 patients in the ROCKstar study given 200mg of Rezurock twice daily, just under half of them had four or more organs affected by the disease.
“We believe that this is the most advanced, most severe population ever studied in this disease,” Sanjay Aggarwal, SVP of clinical development, said back in May 2020.
Six percent of patients achieved a complete response, Kadmon said, and 62% of responders didn’t require new systemic therapy for at least 12 months. The median time to first response was 1.8 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,” said Stephanie Lee, research director of the long-term follow-up program at the Fred Hutchinson Cancer Research Center.
Upon releasing the first glimpse at pivotal data back in 2019, Kadmon framed their drug as one of a growing number of options patients might use, noting that many of the people in this trial had didn’t respond to Incyte’s Jakafi or AbbVie and J&J’s Imbruvica.
The company was founded by Sam Waksal, the entrepreneur who was convicted of insider trading in 2003. He founded the company after his release, then handed the reins to his brother Harlan before its public debut as his sentence barred him from heading a public company.