Research & Development
US FDA Has Extends Review Period for Ruxolitinib in Acute Graft-Versus-Host Disease
8 February 2019 - - The US Food and Drug Administration has extended the review period for the supplemental New Drug Application for ruxolitinib (Jakafi) for the treatment of patients with acute graft-versus-host disease who have had an inadequate response to corticosteroids, US-based biopharmaceutical company Incyte (NASDAQ: INCY) said

The new Prescription Drug User Fee Act (PDUFA) target action date is May 24, 2019.

According to the company, the FDA extended the action date to allow time to review additional data submitted by Incyte in response to the FDA's information requests.

Submission of the additional information has been determined by the FDA to constitute a Major Amendment to the sNDA, resulting in an extension of the PDUFA goal date by three months.

The sNDA for ruxolitinib for the treatment of patients with acute GVHD was submitted in August 2018, and the FDA granted both Priority Review and Breakthrough Therapy Designation.

The FDA grants Priority Review to medicines that have the potential to provide significant improvements in the treatment of a serious disease. The FDA's Breakthrough Therapy Designation is designed to expedite the development and review of drugs for serious conditions that have shown encouraging early clinical results and may demonstrate substantial improvements over available medicines.

Additionally, the FDA granted ruxolitinib Orphan Drug Designation for the treatment of GVHD, a designation granted to investigational compounds intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people.

GVHD is a condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor), where the donated cells initiate an immune response and attack the transplant recipients organs, leading to significant morbidity and mortality.

There are two forms of GVHD, acute and chronic, which can affect multiple organ systems including the skin, gastrointestinal (digestive) tract and liver.

Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the US Food and Drug Administration for treatment of people with polycythemia vera who have had an inadequate response to or are intolerant of hydroxyurea.

Jakafi is also indicated for treatment of people with intermediate or high-risk myelofibrosis, including primary MF, post–polycythemia vera MF, and post–essential thrombocythemia MF.

It is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corp.

Jakavi is a registered trademark of Novartis AG in countries outside the United States. Ruxolitinib is also being evaluated in patients with acute and chronic GVHD who have an inadequate response to corticosteroids in the REACH2 and REACH3 clinical studies, respectively.

It is expected that these two pivotal studies will complete in 2019, and could support additional regulatory submissions, in the US by Incyte and ex-US by Novartis, in 2020.

Incyte is a Wilmington, Delaware-based biopharmaceutical company focused on the discovery, development and commercialization of proprietary therapeutics.


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