MAJOR AMENDMENT leads to PDUFA extended to August 30th but approval comes on July 16th, 45 days earlier than scheduled. See the two PRs below.
Kadmon Announces U.S. FDA Has Extended the Review Period for Belumosudil in Chronic Graft-Versus-Host Disease
March 10, 2021 at 4:14 PM EST
NEW YORK, NY / ACCESSWIRE / March 10, 2021 / Kadmon Holdings, Inc. (NASDAQ:KDMN) today announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for belumosudil for the treatment of chronic graft-versus-host disease (cGVHD). In a notice received from the FDA on March 9, 2021, the Company was informed that the Prescription Drug User Fee Act (PDUFA) goal date for its Priority Review of belumosudil has been extended to August 30, 2021.
The FDA extended the PDUFA date to allow time to review additional information submitted by Kadmon in response to a recent FDA information request. The submission of the additional information has been determined by the FDA to constitute a major amendment to the NDA, resulting in an extension of the PDUFA date by three months.
"We remain confident in the data supporting our application for belumosudil in cGVHD and look forward to continuing to work closely with the FDA during the remainder of the review process," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We are committed to bringing belumosudil to market, once approved, to help meet the needs of patients living with cGVHD."
Belumosudil (KD025) is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. In November 2020, the U.S. Food and Drug Administration (FDA) accepted and granted Priority Review for the NDA for belumosudil for the treatment of patients with cGVHD. The NDA is being reviewed under the FDA's Real-Time Oncology Review (RTOR) and Project Orbis pilot programs. The FDA has granted Breakthrough Therapy Designation to belumosudil for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy as well as Orphan Drug Designation to belumosudil for the treatment of cGVHD.
cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD.
Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Kadmon's clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.
U.S. FDA Grants Full Approval of REZUROCK(TM) (belumosudil) for the Treatment of Patients with Chronic Graft-Versus-Host Disease (cGVHD)
July 16, 2021 at 2:20 PM EDT
- REZUROCK is approved for the treatment of adult and pediatric patients 12 years and older with cGVHD after failure of at least two prior lines of systemic therapy -
- Kadmon to Host Conference Call on Monday, July 19, 2021 at 8:00 a.m. ET -
NEW YORK, NY / ACCESSWIRE / July 16, 2021 / Kadmon Holdings, Inc. (Nasdaq:KDMN) today announced that the U.S. Food and Drug Administration (FDA) has approved REZUROCK™ (belumosudil) 200 mg once daily (QD) for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy. The FDA granted Breakthrough Therapy designation and Priority Review for REZUROCK and reviewed the New Drug Application (NDA) under the Real-Time Oncology Review (RTOR) pilot program. The FDA approved this NDA six weeks ahead of the Prescription Drug User Fee Act (PDUFA) goal date of August 30, 2021. REZUROCK is the first and only FDA-approved small molecule inhibitor of ROCK2, a signaling pathway that modulates inflammatory responses and fibrotic processes.
"REZUROCK represents a new treatment paradigm for thousands of cGVHD patients, including those with difficult-to-treat manifestations like fibrosis," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine at Harvard Medical School and Medical Director, Adult Stem Cell Transplantation Program at the Dana-Farber Cancer Institute. "REZUROCK has shown robust and durable responses across the spectrum of cGVHD and is safe and well tolerated, allowing patients to stay on therapy and achieve meaningful benefit from treatment."
The FDA approval of REZUROCK is based on safety and efficacy results from ROCKstar (KD025-213), a randomized, open-label, multicenter pivotal trial of REZUROCK in patients with cGVHD who had received two to five prior lines of systemic therapy. There were 65 patients treated with REZUROCK 200 mg taken orally QD. The median time from cGVHD diagnosis was 25.3 months and 48% of patients had four or more organs involved. Patients had cycled through a median of 3 prior lines of systemic therapy and 78% were refractory to their last therapy. REZUROCK 200 mg QD achieved an Overall Response Rate (ORR) of 75% through Cycle 7 Day 1 of treatment (95% Confidence Interval (CI): 63, 85), with 6% achieving a complete response and 69% achieving a partial response. The median time to first response was 1.8 months. Sixty-two percent (62%) of responders did not require new systemic therapy for at least 12 months following response. The median duration of response, calculated from first response to progression, death, or new systemic therapies for chronic GVHD, was 1.9 months. ORR results were supported by clinically meaningful improvement from baseline in the Lee Symptom Scale (LSS) score, a chronic GVHD symptom measurement, in 52% of patients through Cycle 7 Day 1 of treatment.
"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, and Research Director of the Long-Term Follow-Up Program at Fred Hutchinson.
REZUROCK has been well tolerated and adverse events have been consistent with those expected in patients with advanced cGVHD receiving corticosteroids and/or other immunosuppressants.
"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. "Thank you to the patients, their families and caregivers, who are the center of our focus in achieving this significant milestone. We have built a hematology/oncology-experienced commercial team and we look forward to rapid adoption of REZUROCK for patients in need."
REZUROCK is expected to be available in the United States by late August 2021.
Kadmon is committed to helping patients with treatment access and support. Kadmon ASSIST™ is a program designed to help and support REZUROCK patients and their caregivers throughout their treatment journey. This program provides reimbursement assistance and savings programs for eligible patients. For more information, please call 1-844-KADMON1 (1-844-523-6661), Monday-Friday, 8:00 a.m.to 8:00 p.m. ET.
The NDA for REZUROCK is part of Project Orbis, an initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology drugs among participating international health authorities.
Conference Call and Webcast
Kadmon will host a conference call on Monday, July 19, 2021 at 8:00 a.m. ET to discuss the FDA approval of REZUROCK.
To participate in the conference call, please dial (866) 762-3021 (domestic) or +1 (703) 925-2661 (international) and reference the conference ID: 5399837.
original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.