Celgene’s Inrebic becomes second FDA-approved therapy for rare bone marrow disorder
Celgene is celebrating the approval in the US of its Janus kinase (JAK) 2 inhibitor Inrebic (fedratinib) in the treatment of intermediate-2 or high-risk primary or secondary myelofibrosis, a rare disorder of the bone marrow.
A chronic condition, myelofibrosis is characterised by the formation of scar tissue in the bone marrow, disrupting the production of blood cells and leading to severe anaemia and the enlargement of the body’s organs.
The FDA’s decision to approve the drug means that it is the first drug approved in the US for the treatment of this condition since the authorisation of Jakafi in 2011. It was based on data drawn from 289 participants, each randomised to receive either a 400mg or 500mg daily dose of Inrebic or placebo.
It was found that 35 of the 96 patients receiving 400mg of Inrebic experienced a “significant therapeutic effect” of greater than or equal to a 35% reduction from baseline in spleen volume after 26 weeks of treatment, while 36 patients saw an improvement of 50% or more in myelofibrosis-related symptoms.
“Prior to today, there was one FDA-approved drug to treat patients with myelofibrosis, a rare bone marrow disorder. Our approval today provides another option for patients,” said Dr Richard Pazdur, Director of the FDA’s Oncology Center of Excellence and acting Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “The FDA is committed to encouraging the development of treatments for patients with rare diseases and providing alternative options, as not all patients respond in the same way.”
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