Novartis Announces NMPA Approval of Ruxolitinib Tablets for Acute Graft-versus-Host Disease in China

Recently, Novartis announced that its JAK inhibitor Jakavi® (Ruxolitinib Phosphate Tablets) has been approved by the National Medical Products Administration (NMPA) of China for the treatment of acute graft-versus-host disease (acute GVHD) in patients aged 12 years and above who have had an inadequate response to corticosteroids or other systemic therapies. This is currently the first and only drug approved in China for the treatment of this condition. Previously, Jakavi® was approved in China in 2017 for adult patients with intermediate or high-risk primary myelofibrosis (PMF) (also known as chronic idiopathic myelofibrosis), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF), to treat disease-related splenomegaly or disease-related symptoms. The approval of this new indication will further expand the beneficiary population of Jakavi®, benefiting more patients in China.

Graft-versus-host disease (GVHD) is a fatal complication following allogeneic hematopoietic stem cell transplantation. The donor's immune cells (graft) recognize the recipient’s body (host) as foreign and subsequently attack the host's organs and tissues, leading to a series of pathological changes and even death, significantly affecting the long-term survival rate and quality of life for post-transplant patients. GVHD can generally be divided into acute and chronic forms, with the incidence of acute GVHD reaching 30%-60%, and the moderate to severe cases accounting for 13%-47%. Acute GVHD typically occurs within 100 days post-transplant, with common clinical symptoms including characteristic rashes, abdominal pain with diarrhea, and elevated serum bilirubin levels. Patients with moderate to severe acute GVHD experience a range of complications, impacting their non-relapse mortality rate.

Glucocorticoids (hereinafter referred to as hormones) are the standard first-line treatment for acute GVHD recommended by guidelines both in China and internationally. However, approximately 50% of patients still fail to achieve sustained remission. For steroid-refractory patients receiving conventional treatment, the 6-month survival rate is only about 50%, with less than 30% surviving beyond 2 years. Meanwhile, there is currently no internationally standardized treatment regimen for steroid-refractory acute GVHD, and its treatment remains one of the severe challenges faced by allogeneic hematopoietic stem cell transplant patients and clinicians.

In the international multicenter, randomized, Phase III clinical trial REACH2, Jakavi® achieved breakthrough results and became the first new drug proven to be superior to Best Available Therapy (BAT) in a Phase III trial for patients with steroid-refractory acute GVHD. Compared with BAT, Jakavi® provided rapid and durable relief for patients with steroid-refractory acute GVHD, with a 28-day overall response rate of 62%, approximately 1.6 times that of the BAT group, and a 56-day sustained overall response rate of 40%, about twice that of the BAT group. It significantly extended patient survival, with a median overall survival of 11.1 months vs. 6.5 months for BAT, allowed faster tapering of steroids, and improved quality of life. Meanwhile, the drug was well-tolerated by patients, and its safety profile was consistent with previous observations in other Jakavi® studies. Supported by high-quality evidence-based medical evidence, Jakavi® received a Category 1 recommendation from the NCCN guidelines for the treatment of steroid-refractory acute GVHD.

Editor: Liuli

Disclaimer: This article is a reprint from Yaozhi.com. The images and text are the property of the original author, and the purpose of the reprint is to convey more information. It does not represent the views of this platform. If there are any issues regarding the content, copyright, or other aspects, please leave a message on this platform, and we will address it promptly.