First bone marrow disorder treatment approved by the FDA after nearly a decade

By Vassia Barba contact

- Last updated on GMT

(Image: Getty/YanaVasileva)
(Image: Getty/YanaVasileva)

Related tags: Bristol-myers squibb, Bms, Celgene, Food and drug administration, FDA approval

Celgene’s Inrebic becomes the first treatment for myelofibrosis to be approved by the US FDA since 2011 after having received a priority review and orphan drug designation.

Inrebic (fedratinib), is a small molecule oral kinase inhibitor targeting Janus associated kinase 2 and FMS-like tyrosine kinase 3, indicated for the treatment of adult patients with myelofibrosis.

Until today, the only available treatment for myelofibrosis was Jakafi (ruxolitinib), which was developed by Incyte in collaboration with Novartis and approved​ by the US Food and Drug Administration (FDA) in 2011.

In Phase III clinical studies 37% of patients treated with Inrebic experienced reduction of spleen volume, while 40% of patients reported improved symptoms.

“The approval provides another treatment option for newly-diagnosed patients, as well as for patients who have experienced failure with ruxolitinib due to lack of tolerance or response,” ​commented Paul Jardina, CEO of Onco360, a specialty pharmacy network partner which will distribute​ the product.

Myelofibrosis is a rare, chronic bone marrow disorder that disrupts the body’s normal production of blood cells, potentially leading to anemia, weakness, fatigue and enlargement of the spleen and liver.

On average, patients with myelofibrosis survive five to six years following their initial diagnosis, while in the absence of treatment, up to 12% will develop acute myeloid leukemia.

The disease affects 1.5 of every 100,000 people each year in the US, while nearly 18,000 people are currently living with the disease.

The approval for Inrebic was granted to Impact Biomedicines, a wholly-owned subsidiary of Celgene, after the drug had received priority review and orphan drug designation.

Celgene was acquired​ by Bristol-Myers Squibb earlier this year in a $74bn (€65.2bn) deal, with the merger expected to be complete by September, 2019.

A broken regulatory deadlock

Fedratinib was developed by TargeGen, which was acquired​ by Sanofi in 2010. Sanofi was forced to stop the clinical trials​ for the product in 2013, after the FDA placed a hold on them due to cases of Wernicke’s encephalopathy observed in eight patients.

With the funding support of Medicxi, start-up Impact Biosciences, which was formed in 2016,  acquired the development rights for fedratinib from Sanofi and restarted the clinical trials, after the FDA lifted the hold in 2017.

The development of fedratinib was then completed by Celgene, after the latter acquired​ Impact for $1.1bn upfront, in January 2018.

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